Study of Pomalidomide (CC-4047) to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Effectiveness for Patients With Systemic Sclerosis With Interstitial Lung Disease
Study Details
Study Description
Brief Summary
The primary objective of this study is to evaluate the safety, tolerability, and efficacy of pomalidomide in the treatment of patients with systemic sclerosis with interstitial lung disease.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Placebo Comparator: Placebo
|
Drug: Placebo
Matching placebo capsules taken orally once a day
|
| Experimental: Pomalidomide Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
Drug: Pomalidomide (CC-4047)
1 mg orally every day for 52 weeks
|
Outcome Measures
Primary Outcome Measures
- Number of Participants With Treatment-Emergent Adverse Events (TEAEs) [From the start of study drug to 28 days after last dose; Treatment Phase median duration of treatment was 358 and 320 days for Placebo and Pomalidomide; Extension phase median duration of treatment was 161 days and 194 days for Placebo and pomalidomide.]
An adverse event (AE) is any noxious, unintended, or untoward medical occurrence that may appear or worsen during the course of a study. A TEAE is any AE that began or worsened on or after the start of study drug through 28 days after the last dose. A treatment-related TEAE is a TEAE which was considered by the investigator to be related to study drug. The severity/intensity of AEs was assessed by the investigator as Mild (asymptomatic or mild symptoms; intervention not indicated), Moderate (symptoms cause moderate discomfort, intervention may be required), or Severe (symptoms cause severe discomfort/pain, requiring medical intervention, inability to perform daily activities). A serious AE is any AE that: - Resulted in death; - Was life-threatening; - Required inpatient hospitalization or prolongation of existing hospitalization - Resulted in persistent or significant disability/incapacity; - Was a congenital anomaly/birth defect; - Constituted an important medical event.
- Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52 [Baseline (defined as the average of all values between Screening and Baseline) and Weeks 48 and 52]
Forced vital capacity (FVC) is a pulmonary function test and is the volume of air in the lungs that can forcibly be blown out after a full inhalation. Percent predicted values are based comparison between the participant's measured value with expected FVC for someone of the same sex, age and height (reference value). For the analysis of FVC, the baseline value was defined as the average of all values between Screening and Baseline (inclusive), and the average of Weeks 48 and 52 was treated as the Week 52 value, to reduce the total data variability at the key time points.
- Change From Baseline in the Modified Rodnan Skin Score (mRSS) at Week 52/Early Termination [Baseline and Week 52 (or the Treatment Phase Early Termination visit)]
Improvement in skin thickening is associated with improved survival and may be useful as a surrogate measurement in clinical studies. The mRSS is an assessment tool which is used to evaluate the extent and severity of the skin thickening associated with systemic sclerosis (SSc). Seventeen body areas were evaluated on a 4-point scale (0 [normal], 1 [mild], 2 [moderate]), or 3 [severe]). The total score, which is the sum of the 17 individual body assessments, can range from 0 to 51.
- Change From Baseline in University of California, Los Angeles, Scleroderma Clinical Trial Consortium Gastrointestinal Tract (UCLA SCTC GIT 2.0) Total Score at Week 52/Early Termination [Baseline and Week 52 (or Treatment Phase Early Termination visit)]
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets gastrointestinal (GI) activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health (except for Questions 15 and 31 which are scored as 0 (better health) or 1 (worse health). The total score is calculated as the average of the first 6 scale scores (excluding constipation) which captures overall burden (severity) of SSc-associated GIT. The overall score ranges from 0 to 3, where higher scores indicate more severe symptoms.
Secondary Outcome Measures
- Change From Baseline in Percent Predicted Forced Vital Capacity Over Time [Baseline (defined as the average of all values between Screening and Baseline) and Weeks 12, 24, 36, 64, 76, and 156]
Forced vital capacity (FVC) is a pulmonary function test and is the volume of air in the lungs that can forcibly be blown out after a full inhalation. Percent predicted values are based comparison between the participant's measured value with expected FVC for someone of the same sex, age and height (reference value).
- Change From Baseline in Modified Rodnan Skin Score Over Time [Baseline and Weeks 12, 24, 64, 76, and 156 (or the Extension Phase Early Termination visit).]
Improvement in skin thickening is associated with improved survival and may be useful as a surrogate measurement in clinical studies. The mRSS is an assessment tool which is used to evaluate the extent and severity of the skin thickening associated with systemic sclerosis (SSc). Seventeen body areas were evaluated on a 4-point scale (0 [normal], 1 [mild], 2 [moderate], or 3 [severe]). The total score, which is the sum of the 17 individual body assessments, can range from 0 to 51.
- Change From Baseline in UCLA SCTC GIT 2.0 Total Score Over Time [Baseline and Weeks 12, 24, 64, 76, and 156 (or the Extension Phase Early Termination visit).]
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health (except for Questions 15 and 31 which are scored as 0 (better health) or 1 (worse health). The total score is calculated as the average of the first 6 scale scores (excluding constipation) which captures overall burden (severity) of SSc-associated GIT. The overall score ranges from 0 to 3, where higher scores indicate more severe symptoms.
- Change From Baseline in UCLA SCTC GIT 2.0 Reflux Subscale Score Over Time [Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).]
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The reflux subscale score is calculated as the average of eight reflux-related questions; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.
- Change From Baseline in UCLA SCTC GIT 2.0 Distension/Bloating Subscale Score Over Time [Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).]
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The distension/bloating subscale score is calculated as the average of four distension/bloating-related questions; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.
- Change From Baseline in UCLA SCTC GIT 2.0 Fecal Soilage Subscale Score Over Time [Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).]
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The fecal soilage subscale score is calculated from one soilage question; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.
- Change From Baseline in UCLA SCTC GIT 2.0 Diarrhea Subscale Score Over Time [Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).]
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The diarrhea subscale score is calculated as the average of one diarrhea question about the frequency of loose stools (on a scale from 0 [none] to 3 [5-7 days/week] and one question about the presence of watery stools (scored as 0 [No] or 1 [Yes]); the score ranges from 0 to 2, where a higher score indicates more frequent symptoms.
- Change From Baseline in UCLA SCTC GIT 2.0 Social Functioning Subscale Score Over Time [Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).]
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The social functioning subscale score is calculated as the average of six questions about how often symptoms interfered with social activities; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.
- Change From Baseline in UCLA SCTC GIT 2.0 Emotional Well Being Subscale Score Over Time [Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).]
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The emotional well-being subscale score is calculated as the average of nine questions regarding the impact of bowel problems on emotional status; the score ranges from 0 to 3, where higher scores indicate more frequent problems.
- Change From Baseline in UCLA SCTC GIT 2.0 Constipation Subscale Score Over Time [Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).]
The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The constipation subscale score is calculated as the average of three questions regarding the frequency of constipation (scored from 0 [no days] to 3 [5-7 days/week] and one question about the presence of stools becoming harder (scored as 0 [No] or 1 [Yes]); the score ranges from 0 to 2.5, where higher scores indicate more frequent symptoms.
- Change From Baseline in Dyspnea Functional Impairment at Week 12 [Week 12]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
- Change From Baseline in Dyspnea Functional Impairment at Week 24 [Week 24]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
- Change From Baseline in Dyspnea Functional Impairment at Week 52/Early Termination [Week 52 or at the Treatment Phase Early Termination visit]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
- Change From Baseline in Dyspnea Functional Impairment at Week 64 [Week 64]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
- Change From Baseline in Dyspnea Functional Impairment at Week 76 [Week 76]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
- Change From Baseline in Dyspnea Functional Impairment at Week 156/Early Termination [Week 156 or the Extension Phase Early Termination visit]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath.
- Change From Baseline in Dyspnea Magnitude of Task at Week 12 [Week 12]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
- Change From Baseline in Dyspnea Magnitude of Task at Week 24 [Week 24]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
- Change From Baseline in Dyspnea Magnitude of Task at Week 52/Early Termination [Week 52 or at the Treatment Phase Early Termination visit]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
- Change From Baseline in Dyspnea Magnitude of Task at Week 64 [Week 64]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
- Change From Baseline in Dyspnea Magnitude of Task at Week 76 [Week 76]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
- Change From Baseline in Dyspnea Magnitude of Task at Week 156/Early Termination [Week 156 or the Extension Phase Early Termination visit]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carry very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain.
- Change From Baseline in Dyspnea Magnitude of Effort at Week 12 [Week 12]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
- Change From Baseline in Dyspnea Magnitude of Effort at Week 24 [Week 24]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
- Change From Baseline in Dyspnea Magnitude of Effort at Week 52/Early Termination [Week 52 or at the Treatment Phase Early Termination visit]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
- Change From Baseline in Dyspnea Magnitude of Effort at Week 64 [Week 64]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
- Change From Baseline in Dyspnea Magnitude of Effort at Week 76 [Week 76]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
- Change From Baseline in Dyspnea Magnitude of Effort at Week 156/Early Termination [Week 156 or at the Extension Phase Early Termination visit]
The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath.
- Oxygen Saturation Over Time [Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).]
Oxygen saturation was measured by pulse oximetry.
- Pharmacokinetic Parameters of Pomalidomide in Plasma [Day 1 and week 4 pre-dose and up to 24 hours post-dose.]
Pharmacokinetic (PK) analyses were not conducted as there were too few participants with available data.
Eligibility Criteria
Criteria
Key Inclusion Criteria:
-
Male or females between 18 and 80 years of age (inclusive) at the time of consent
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Diagnosis of systemic sclerosis (SSC) as defined by American College of Rheumatology (ACR) criteria
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Onset of the first non-Raynaud's manifestation of SSC within 7 years of Screening
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Subjects are required to meet at least one of the following 2 pulmonary-related criteria to be eligible for the study:
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Forced vital capacity (FVC) ≥ 45% and <70% at Screening and Baseline (Visit 2) [with or without a documented pre-specified FVC decline or fibrosis score] OR
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FVC readings ≥ 70% and ≤ 80% at Screening and Baseline (Visit 2) with a documented history of either or both of:
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A ≥ 5% decrease (expressed as percent predicted or in liters) in FVC in the 24-month period prior to Baseline (Visit 2) based on 3 or more assessments. Two assessments may be done during the Screening phase provided the assessments are completed at least 2 weeks apart.
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A high resolution computed tomography (HRCT) fibrosis score > 20%
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FVC at Baseline (Visit 2) within 5% of the FVC measured at Screening
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Carbon monoxide diffusing capacity (DLco) ≥ 35% and ≤ 80% of predicted value at Screening
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Abnormalities on High-Resolution CT consistent with parenchymal changes encountered in SSc: honeycombing or reticular changes with or without ground glass.
Key Exclusion Criteria:
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Oxygen saturation (SpO2) < 92% (room air [sea level] at rest) at Screening or Baseline
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Known diagnosis of obstructive lung disease as defined by forced expiratory volume (FEV1)/FVC ratio < 0.7
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Diagnosis of pulmonary arterial hypertension (PAH) requiring treatment
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Known diagnosis of other significant respiratory disorders (e.g., asthma, tuberculosis, sarcoidosis, aspergillosis, chronic bronchitis, neoplastic disease, cystic fibrosis, etc.)
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Current clinical diagnosis of another inflammatory connective tissue disease (eg, systemic lupus erythematosus, rheumatoid arthritis, primary Sjogren's syndrome, etc.). Subjects having Sjogren's syndrome secondary to SSc are eligible
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Pregnant or lactating females
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History of a thromboembolic event (eg, deep vein thrombosis, thrombotic cerebrovascular or cardiovascular events)
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History or current diagnosis of peripheral neuropathy
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Use of concomitant medication(s) which could increase the risk for developing deep vein thrombosis, including sex steroid-based contraceptives (oral, injectable or implanted) and hormone replacement therapies, if use of a low-dose aspirin regimen is contraindicated.
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Additional concomitant medications which prolong the QT/QTc interval (measure of heart's electrical cycle) during the course of the study
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Use of any anti-coagulant or anti-thrombotic medications (other than low dose-aspirin [≤ 100 mg/day])
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Use of any cytotoxic/immunosuppressive agent (other than prednisone ≤ 10 mg/day [mean dose] or equivalent), including but not limited to azathioprine, cyclophosphamide, methotrexate, mycophenolate and cyclosporine within 28 days (4 weeks) of Screening
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Use of any biologic agent within 84 days (12 weeks) or 5 half-lives of Screening. In the case of rituximab, use within 168 days (24 weeks) of Screening or no recovery of CD20-positive B lymphocytes if the last dose of rituximab has been more than 24 weeks prior to Screening
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Use of bosentan, ambrisentan, sildenafil, tadalafil and macitentan for PAH within 28 days (4 weeks) of Screening
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Use of medications (e.g., D-penicillamine, Potaba) with putative scleroderma disease-modifying properties within 4 weeks of Screening
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Use of melphalan within 52 weeks of Screening
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Use of any investigational drug within 4 weeks of Screening or 5 pharmacodynamic/pharmacokinetic half-lives if known (whichever is longer)
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Smoking of cigars, pipes or cigarettes within 24 weeks of Screening
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | UCLA Division of Rheumatology | Los Angeles | California | United States | 90095 |
| 2 | Advances in Medicine | Rancho Mirage | California | United States | 92270 |
| 3 | Delaware Medical Care Associates, LLC | Newark | Delaware | United States | 19713 |
| 4 | Georgetown University School of Medicine | Washington | District of Columbia | United States | 20057 |
| 5 | USF Health Faculty Office Building-FOB | Tampa | Florida | United States | 33612 |
| 6 | Arthritis Research and Treatment Center | Stockbridge | Georgia | United States | 30281 |
| 7 | University of Illinois at Chicago | Chicago | Illinois | United States | 60612 |
| 8 | LaPorte County Institute for Clinical Research, Inc | Michigan City | Indiana | United States | 46360 |
| 9 | University of Kentucky | Lexington | Kentucky | United States | 40536-0284 |
| 10 | Louisiana State University | Shreveport | Louisiana | United States | 71103 |
| 11 | Boston University of Medicine BUMC | Boston | Massachusetts | United States | 02118 |
| 12 | University of Michigan | Ann Arbor | Michigan | United States | 48109 |
| 13 | UMDNJ-Robert Wood Johnson Medical School Clinical Research Center | New Brunswick | New Jersey | United States | 08903 |
| 14 | North Shore-LIJ Health System-Division of Rheumatology | Great Neck | New York | United States | 11021 |
| 15 | Cleveland Clinic | Cleveland | Ohio | United States | 44195 |
| 16 | University of Toledo College of Medicine | Toledo | Ohio | United States | 43614 |
| 17 | University of Pennsylvania | Philadelphia | Pennsylvania | United States | 19104 |
| 18 | University of Pittsburgh | Pittsburgh | Pennsylvania | United States | 15261 |
| 19 | University of Texas Health Science Center at Houston | Houston | Texas | United States | 77030 |
| 20 | Medical College of Wisconsin | Milwaukee | Wisconsin | United States | 53226 |
| 21 | Monash Medical Centre | Clayton | Victoria | Australia | 3168 |
| 22 | The Prince Charles Hospital | Chermside | Australia | 4032 | |
| 23 | The Queen Elizabeth Hospital | Woodville South | Australia | 5011 | |
| 24 | CHRU de Lille FR | Lille | France | 59037 | |
| 25 | Hopital Saint Louis | Paris | France | 75010 | |
| 26 | Groupe Hospitalier Saint Vincent de Paul | Paris | France | 75014 | |
| 27 | Kerckhoff-Klinik gGmbH | Bad Nauheim | Germany | 61231 | |
| 28 | Charite - Universitätsmedizin Berlin | Berlin | Germany | 10117 | |
| 29 | University of Erlangen-Nuremberg | Erlangen | Germany | 91054 | |
| 30 | Klinikum der J.W. Gothe-Universitat Frankfurt | Frankfurt | Germany | 60590 | |
| 31 | Rheumazentrum Ruhrgebiet | Herne | Germany | 44652 | |
| 32 | University Hospital of Ulm | Ulm | Germany | 89081 | |
| 33 | Azienda Ospedaliera Universitaria S. Martino di Genova | Genova | Italy | 16132 | |
| 34 | Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico | Milano | Italy | 20122 | |
| 35 | Ospedale Luigi Sacco | Milano | Italy | 20157 | |
| 36 | IRCCS Policlinico S. Matteo di Pavia | Pavia | Italy | 27100 | |
| 37 | Azienda Ospedaliera Universitaria Pisana | Pisa | Italy | 56126 | |
| 38 | Policlinico Umberto I | Roma | Italy | 00161 | |
| 39 | Policlinico Universitario Agostino Gemelli | Roma | Italy | 00168 | |
| 40 | Centrum Miriada Prywatny Gabinet Specjalistyczny Profesora Dra Stanislawa Sierakowskiego | Bialystok | Poland | 15-297 | |
| 41 | Szpital Uniwersytecki nr 2 im. Dr Jana Biziela w Bydgoszczy | Bydgoszcz | Poland | 85-168 | |
| 42 | SPSK Nr 7 Slaskiego Uniwersytetu Medycznego, Oddzial Chorob Wewnetrznych i Reumatologii | Katowice | Poland | 40-634 | |
| 43 | Samodzielny Publiczny Szpital Kliniczny nr 1 im. prof.Tadeusza Sokolowskiego Pomorskiego UM w Szczec | Szczecin | Poland | 71-252 | |
| 44 | Instytut Reumatologii, Klinika i Poliklinika Ukladowych Chorób Tkanki Lacznej | Warszawa | Poland | 02-637 | |
| 45 | Akademicki Szpital Kliniczny Klinika Reumatologii i Chorob Wewnetrznych | Wroclaw | Poland | 50-556 | |
| 46 | Institution of the Russian Academy of Medical Sciences Research Institute of Rheumatology of the Ru | Moscow | Russian Federation | 115522 | |
| 47 | Penza Regional Clinical Hospital n.a. N.N. Burdenko | Penza | Russian Federation | 440026 | |
| 48 | St. Petersburg State Healthcare Institution "Clinical Rheumatology Hospital # 25" | St. Petersburg | Russian Federation | 190068 | |
| 49 | Hospital Universitario Gregorio Maranon | Madrid | Spain | 28007 | |
| 50 | Hospital Universitario 12 de Octubre | Madrid | Spain | 28041 | |
| 51 | Complejo Hospitalario de Santiago | Santiago de Compostela | Spain | 15706 | |
| 52 | Hospital Universitario Dr. Peset | Valencia | Spain | 46017 | |
| 53 | University Hospital Basel | Basel | Switzerland | 4031 | |
| 54 | Chapel Allerton Hospital | Leeds | United Kingdom | LS7 4SA | |
| 55 | Royal Free Hospital | London | United Kingdom | NW3 2PF | |
| 56 | Royal Brompton Hospital - Interstitial Lung Disease Unit | London | United Kingdom | SW3 6NP |
Sponsors and Collaborators
- Celgene
Investigators
- Study Director: Shimon Korish, MD, Celgene Corporation
Study Documents (Full-Text)
None provided.More Information
Publications
- Hsu V, et al. A Phase 2 Study of Pomalidomide (CC-4047) to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Effectiveness in Subjects with Systemic Sclerosis with Interstitial Lung Disease. Presented at the 2016 ACR/ARHP Annual Meeting, November 11-16, 2016, Washington, DC. Abstract No. 823.
- Hsu VM, Denton CP, Domsic RT, Furst DE, Rischmueller M, Stanislav M, Steen VD, Distler JHW, Korish S, Cooper A, Choi S, Schafer PH, Horan G, Hough DR. Pomalidomide in Patients with Interstitial Lung Disease due to Systemic Sclerosis: A Phase II, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study. J Rheumatol. 2018 Mar;45(3):405-410. doi: 10.3899/jrheum.161040. Epub 2017 Nov 1.
- CC-4047-SSC-001
- 2010-023047-15
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and continued to receive the same treatment for up to 2 years during the open-label extension phase. |
| Period Title: Treatment Phase | ||
| STARTED | 12 | 11 |
| Received Treatment | 12 | 10 |
| COMPLETED | 7 | 4 |
| NOT COMPLETED | 5 | 7 |
| Period Title: Treatment Phase | ||
| STARTED | 6 | 2 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 6 | 2 |
Baseline Characteristics
| Arm/Group Title | Placebo | Pomalidomide | Total |
|---|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and continued to receive the same treatment for up to 2 years during the open-label extension phase. | Total of all reporting groups |
| Overall Participants | 12 | 11 | 23 |
| Age (years) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [years] |
44.8
(13.77)
|
48.9
(9.84)
|
46.7
(11.97)
|
| Age, Customized (Count of Participants) | |||
| < 65 years |
10
83.3%
|
10
90.9%
|
20
87%
|
| ≥ 65 years |
2
16.7%
|
1
9.1%
|
3
13%
|
| Sex: Female, Male (Count of Participants) | |||
| Female |
10
83.3%
|
10
90.9%
|
20
87%
|
| Male |
2
16.7%
|
1
9.1%
|
3
13%
|
| Race/Ethnicity, Customized (Count of Participants) | |||
| Asian |
0
0%
|
2
18.2%
|
2
8.7%
|
| Black or African American |
0
0%
|
1
9.1%
|
1
4.3%
|
| Native Hawaiian or Other Pacific Islanders |
1
8.3%
|
0
0%
|
1
4.3%
|
| White |
10
83.3%
|
8
72.7%
|
18
78.3%
|
| Other |
1
8.3%
|
0
0%
|
1
4.3%
|
| Body Mass Index (BMI) (kg/m^2) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [kg/m^2] |
30.64
(5.392)
|
27.61
(9.4)
|
29.19
(7.556)
|
Outcome Measures
| Title | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) |
|---|---|
| Description | An adverse event (AE) is any noxious, unintended, or untoward medical occurrence that may appear or worsen during the course of a study. A TEAE is any AE that began or worsened on or after the start of study drug through 28 days after the last dose. A treatment-related TEAE is a TEAE which was considered by the investigator to be related to study drug. The severity/intensity of AEs was assessed by the investigator as Mild (asymptomatic or mild symptoms; intervention not indicated), Moderate (symptoms cause moderate discomfort, intervention may be required), or Severe (symptoms cause severe discomfort/pain, requiring medical intervention, inability to perform daily activities). A serious AE is any AE that: - Resulted in death; - Was life-threatening; - Required inpatient hospitalization or prolongation of existing hospitalization - Resulted in persistent or significant disability/incapacity; - Was a congenital anomaly/birth defect; - Constituted an important medical event. |
| Time Frame | From the start of study drug to 28 days after last dose; Treatment Phase median duration of treatment was 358 and 320 days for Placebo and Pomalidomide; Extension phase median duration of treatment was 161 days and 194 days for Placebo and pomalidomide. |
Outcome Measure Data
| Analysis Population Description |
|---|
| All randomized participants who received at least one dose of the study treatment (either pomalidomide or placebo). |
| Arm/Group Title | Treatment Phase: Placebo | Treatment Phase: Pomalidomide | Extension Phase: Placebo/Pomalidomide | Extension Phase: Pomalidomide/Pomalidomide |
|---|---|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase. | In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase. |
| Measure Participants | 12 | 10 | 6 | 2 |
| Any TEAE |
12
100%
|
9
81.8%
|
5
21.7%
|
2
NaN
|
| Drug-related TEAE |
8
66.7%
|
6
54.5%
|
3
13%
|
1
NaN
|
| Severe TEAE |
1
8.3%
|
4
36.4%
|
0
0%
|
0
NaN
|
| Serious TEAE |
1
8.3%
|
4
36.4%
|
0
0%
|
1
NaN
|
| Serious Drug-related TEAE |
0
0%
|
1
9.1%
|
0
0%
|
0
NaN
|
| TEAE Leading to Drug Interruption |
2
16.7%
|
1
9.1%
|
0
0%
|
0
NaN
|
| TEAE Leading to Drug Withdrawal |
0
0%
|
4
36.4%
|
0
0%
|
0
NaN
|
| TEAE Leading to Death |
0
0%
|
0
0%
|
0
0%
|
0
NaN
|
| Title | Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52 |
|---|---|
| Description | Forced vital capacity (FVC) is a pulmonary function test and is the volume of air in the lungs that can forcibly be blown out after a full inhalation. Percent predicted values are based comparison between the participant's measured value with expected FVC for someone of the same sex, age and height (reference value). For the analysis of FVC, the baseline value was defined as the average of all values between Screening and Baseline (inclusive), and the average of Weeks 48 and 52 was treated as the Week 52 value, to reduce the total data variability at the key time points. |
| Time Frame | Baseline (defined as the average of all values between Screening and Baseline) and Weeks 48 and 52 |
Outcome Measure Data
| Analysis Population Description |
|---|
| The Full Analysis Set (FAS) consisted of all randomized participants who received at least one dose of study drug. Participants with a Baseline value and a Week 52 were included in the analysis. |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 11 | 8 |
| Mean (Standard Deviation) [percent predicted] |
-2.8
(4.02)
|
-5.2
(5.29)
|
| Title | Change From Baseline in the Modified Rodnan Skin Score (mRSS) at Week 52/Early Termination |
|---|---|
| Description | Improvement in skin thickening is associated with improved survival and may be useful as a surrogate measurement in clinical studies. The mRSS is an assessment tool which is used to evaluate the extent and severity of the skin thickening associated with systemic sclerosis (SSc). Seventeen body areas were evaluated on a 4-point scale (0 [normal], 1 [mild], 2 [moderate]), or 3 [severe]). The total score, which is the sum of the 17 individual body assessments, can range from 0 to 51. |
| Time Frame | Baseline and Week 52 (or the Treatment Phase Early Termination visit) |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with a Baseline value and a post-baseline value at Week 52 or Early Termination |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 11 | 10 |
| Mean (Standard Deviation) [units on a scale] |
-3.7
(6.99)
|
-2.7
(5.66)
|
| Title | Change From Baseline in University of California, Los Angeles, Scleroderma Clinical Trial Consortium Gastrointestinal Tract (UCLA SCTC GIT 2.0) Total Score at Week 52/Early Termination |
|---|---|
| Description | The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets gastrointestinal (GI) activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health (except for Questions 15 and 31 which are scored as 0 (better health) or 1 (worse health). The total score is calculated as the average of the first 6 scale scores (excluding constipation) which captures overall burden (severity) of SSc-associated GIT. The overall score ranges from 0 to 3, where higher scores indicate more severe symptoms. |
| Time Frame | Baseline and Week 52 (or Treatment Phase Early Termination visit) |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with a Baseline value and a Week 52 value |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 12 | 10 |
| Mean (Standard Deviation) [units on a scale] |
0.00
(0.18)
|
0.1
(0.29)
|
| Title | Change From Baseline in Percent Predicted Forced Vital Capacity Over Time |
|---|---|
| Description | Forced vital capacity (FVC) is a pulmonary function test and is the volume of air in the lungs that can forcibly be blown out after a full inhalation. Percent predicted values are based comparison between the participant's measured value with expected FVC for someone of the same sex, age and height (reference value). |
| Time Frame | Baseline (defined as the average of all values between Screening and Baseline) and Weeks 12, 24, 36, 64, 76, and 156 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with a Baseline value and post-baseline value at the time point. |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 12 | 10 |
| Week 12 |
-2.4
(3.07)
|
-1.8
(3.39)
|
| Week 24 |
-1.3
(3.33)
|
2.3
(12.58)
|
| Week 36 |
-2.6
(2.48)
|
-4.4
(3.97)
|
| Week 64 |
-7.0
(4.29)
|
-6.4
(4.49)
|
| Week 76 |
-8.7
(10.34)
|
-5.2
(NA)
|
| Week 156 |
-6.7
(6.19)
|
-5.9
(2.31)
|
| Title | Change From Baseline in Modified Rodnan Skin Score Over Time |
|---|---|
| Description | Improvement in skin thickening is associated with improved survival and may be useful as a surrogate measurement in clinical studies. The mRSS is an assessment tool which is used to evaluate the extent and severity of the skin thickening associated with systemic sclerosis (SSc). Seventeen body areas were evaluated on a 4-point scale (0 [normal], 1 [mild], 2 [moderate], or 3 [severe]). The total score, which is the sum of the 17 individual body assessments, can range from 0 to 51. |
| Time Frame | Baseline and Weeks 12, 24, 64, 76, and 156 (or the Extension Phase Early Termination visit). |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with a Baseline value and a post-baseline value at each time point. |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 11 | 10 |
| Week 12 |
-1.8
(3.94)
|
-0.3
(1.83)
|
| Week 24 |
-3.6
(5.68)
|
-2.0
(3.38)
|
| Week 64 |
-4.3
(8.36)
|
-4.0
(2.83)
|
| Week 76 |
-8.5
(7.78)
|
-7.0
(NA)
|
| Week 156/Early Termination |
-3.7
(8.52)
|
-4.5
(3.54)
|
| Title | Change From Baseline in UCLA SCTC GIT 2.0 Total Score Over Time |
|---|---|
| Description | The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health (except for Questions 15 and 31 which are scored as 0 (better health) or 1 (worse health). The total score is calculated as the average of the first 6 scale scores (excluding constipation) which captures overall burden (severity) of SSc-associated GIT. The overall score ranges from 0 to 3, where higher scores indicate more severe symptoms. |
| Time Frame | Baseline and Weeks 12, 24, 64, 76, and 156 (or the Extension Phase Early Termination visit). |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with a Baseline value and post-baseline value at each time point. |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 12 | 10 |
| Week 12 |
0.2
(0.36)
|
-0.1
(0.30)
|
| Week 24 |
0.1
(0.23)
|
-0.1
(0.20)
|
| Week 64 |
0.0
(0.20)
|
0.4
(0.01)
|
| Week 76 |
-0.1
(0.01)
|
0.5
(NA)
|
| Week 156/Early Termination |
0.0
(0.14)
|
0.0
(0.14)
|
| Title | Change From Baseline in UCLA SCTC GIT 2.0 Reflux Subscale Score Over Time |
|---|---|
| Description | The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The reflux subscale score is calculated as the average of eight reflux-related questions; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms. |
| Time Frame | Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit). |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with a Baseline value and post-baseline value at each time point. |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 12 | 10 |
| Week 12 |
0.1
(0.36)
|
-0.1
(0.51)
|
| Week 24 |
-0.1
(0.27)
|
0.0
(0.47)
|
| Week 52/Early Termination |
-0.1
(0.54)
|
0.1
(0.38)
|
| Week 64 |
-0.2
(0.14)
|
0.2
(0.46)
|
| Week 76 |
-0.5
(0.18)
|
-0.2
(NA)
|
| Week 156/Early Termination |
0.0
(0.44)
|
0.1
(0.28)
|
| Title | Change From Baseline in UCLA SCTC GIT 2.0 Distension/Bloating Subscale Score Over Time |
|---|---|
| Description | The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The distension/bloating subscale score is calculated as the average of four distension/bloating-related questions; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms. |
| Time Frame | Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit). |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with a Baseline value and post-baseline value at each time point. |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 12 | 10 |
| Week 12 |
0.4
(0.56)
|
0.2
(1.21)
|
| Week 24 |
0.0
(0.53)
|
0.3
(0.80)
|
| Week 52/Early Termination |
0.0
(0.55)
|
0.2
(1.07)
|
| Week 64 |
-0.2
(0.70)
|
1.0
(1.41)
|
| Week 76 |
0.0
(0.00)
|
3.0
(NA)
|
| Week 156/Early Termination |
-0.3
(0.30)
|
0.9
(1.24)
|
| Title | Change From Baseline in UCLA SCTC GIT 2.0 Fecal Soilage Subscale Score Over Time |
|---|---|
| Description | The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The fecal soilage subscale score is calculated from one soilage question; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms. |
| Time Frame | Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit). |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with a Baseline value and post-baseline value at each time point. |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 12 | 10 |
| Week 12 |
0.0
(0.00)
|
0.0
(0.00)
|
| Week 24 |
0.2
(0.42)
|
0.0
(0.00)
|
| Week 52/Early Termination |
0.0
(0.00)
|
0.1
(0.33)
|
| Week 64 |
0.2
(0.41)
|
0.0
(0.00)
|
| Week 76 |
0.0
(0.00)
|
0.0
(NA)
|
| Week 156/Early Termination |
0.2
(0.41)
|
0.0
(0.00)
|
| Title | Change From Baseline in UCLA SCTC GIT 2.0 Diarrhea Subscale Score Over Time |
|---|---|
| Description | The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The diarrhea subscale score is calculated as the average of one diarrhea question about the frequency of loose stools (on a scale from 0 [none] to 3 [5-7 days/week] and one question about the presence of watery stools (scored as 0 [No] or 1 [Yes]); the score ranges from 0 to 2, where a higher score indicates more frequent symptoms. |
| Time Frame | Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit). |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with a Baseline value and post-baseline value at each time point. |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 12 | 10 |
| Week 12 |
0.4
(0.57)
|
-0.2
(0.65)
|
| Week 24 |
0.1
(0.21)
|
-0.3
(0.46)
|
| Week 52/Early Termination |
0.3
(0.34)
|
0.0
(0.75)
|
| Week 64 |
0.1
(0.38)
|
0.5
(0.71)
|
| Week 76 |
0.0
(0.00)
|
-0.5
(NA)
|
| Week 156/Early Termination |
0.3
(0.42)
|
-0.8
(0.35)
|
| Title | Change From Baseline in UCLA SCTC GIT 2.0 Social Functioning Subscale Score Over Time |
|---|---|
| Description | The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The social functioning subscale score is calculated as the average of six questions about how often symptoms interfered with social activities; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms. |
| Time Frame | Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit). |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with a Baseline value and post-baseline value at each time point. |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 11 | 10 |
| Week 12 |
0.3
(0.49)
|
0.0
(0.28)
|
| Week 24 |
0.1
(0.30)
|
-0.1
(0.20)
|
| Week 52/Early termination |
0.0
(0.25)
|
0.2
(0.45)
|
| Week 64 |
0.0
(0.18)
|
0.3
(0.35)
|
| Week 76 |
0.2
(NA)
|
0.0
(NA)
|
| Week 156/Early Termination |
0.0
(0.25)
|
-0.2
(0.23)
|
| Title | Change From Baseline in UCLA SCTC GIT 2.0 Emotional Well Being Subscale Score Over Time |
|---|---|
| Description | The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The emotional well-being subscale score is calculated as the average of nine questions regarding the impact of bowel problems on emotional status; the score ranges from 0 to 3, where higher scores indicate more frequent problems. |
| Time Frame | Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit). |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with a Baseline value and post-baseline value at each time point. |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 12 | 10 |
| Week 12 |
0.2
(0.40)
|
-0.4
(0.80)
|
| Week 24 |
0.1
(0.16)
|
-0.3
(0.56)
|
| Week 52/Early Termination |
0.1
(0.25)
|
-0.1
(0.42)
|
| Week 64 |
0.0
(0.36)
|
0.2
(0.00)
|
| Week 76 |
0.0
(0.00)
|
0.3
(NA)
|
| Week 156/Early Termination |
-0.1
(0.15)
|
0.0
(0.00)
|
| Title | Change From Baseline in UCLA SCTC GIT 2.0 Constipation Subscale Score Over Time |
|---|---|
| Description | The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The constipation subscale score is calculated as the average of three questions regarding the frequency of constipation (scored from 0 [no days] to 3 [5-7 days/week] and one question about the presence of stools becoming harder (scored as 0 [No] or 1 [Yes]); the score ranges from 0 to 2.5, where higher scores indicate more frequent symptoms. |
| Time Frame | Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit). |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with a Baseline value and post-baseline value at each time point. |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 12 | 10 |
| Week 12 |
-0.1
(0.36)
|
0.2
(0.98)
|
| Week 24 |
-0.2
(0.31)
|
0.3
(0.81)
|
| Week 52/Early Termination |
0.0
(0.25)
|
0.3
(0.51)
|
| Week 64 |
0.0
(0.60)
|
0.1
(0.18)
|
| Week 76 |
0.0
(0.00)
|
0.0
(NA)
|
| Week 156/Early Termination |
-0.2
(0.20)
|
0.1
(0.18)
|
| Title | Change From Baseline in Dyspnea Functional Impairment at Week 12 |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath. |
| Time Frame | Week 12 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 10 | 7 |
| Major deterioration |
0
0%
|
0
0%
|
| Moderate deterioration |
1
8.3%
|
1
9.1%
|
| Minor deterioration |
0
0%
|
1
9.1%
|
| No change |
7
58.3%
|
3
27.3%
|
| Minor improvement |
2
16.7%
|
1
9.1%
|
| Moderate improvement |
0
0%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
1
9.1%
|
| Title | Change From Baseline in Dyspnea Functional Impairment at Week 24 |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath. |
| Time Frame | Week 24 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 10 | 7 |
| Major deterioration |
1
8.3%
|
0
0%
|
| Moderate deterioration |
0
0%
|
2
18.2%
|
| Minor deterioration |
1
8.3%
|
1
9.1%
|
| No change |
4
33.3%
|
2
18.2%
|
| Minor improvement |
3
25%
|
1
9.1%
|
| Moderate improvement |
1
8.3%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
1
9.1%
|
| Title | Change From Baseline in Dyspnea Functional Impairment at Week 52/Early Termination |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath. |
| Time Frame | Week 52 or at the Treatment Phase Early Termination visit |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 12 | 10 |
| Major deterioration |
0
0%
|
0
0%
|
| Moderate deterioration |
0
0%
|
4
36.4%
|
| Minor deterioration |
1
8.3%
|
1
9.1%
|
| No change |
8
66.7%
|
4
36.4%
|
| Minor improvement |
3
25%
|
1
9.1%
|
| Moderate improvement |
0
0%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
0
0%
|
| Title | Change From Baseline in Dyspnea Functional Impairment at Week 64 |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath. |
| Time Frame | Week 64 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 6 | 2 |
| Major deterioration |
0
0%
|
1
9.1%
|
| Moderate deterioration |
0
0%
|
1
9.1%
|
| Minor deterioration |
1
8.3%
|
0
0%
|
| No change |
4
33.3%
|
0
0%
|
| Minor improvement |
1
8.3%
|
0
0%
|
| Moderate improvement |
0
0%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
0
0%
|
| Title | Change From Baseline in Dyspnea Functional Impairment at Week 76 |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath. |
| Time Frame | Week 76 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 2 | 1 |
| Major deterioration |
0
0%
|
0
0%
|
| Moderate deterioration |
0
0%
|
1
9.1%
|
| Minor deterioration |
1
8.3%
|
0
0%
|
| No change |
0
0%
|
0
0%
|
| Minor improvement |
1
8.3%
|
0
0%
|
| Moderate improvement |
0
0%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
0
0%
|
| Title | Change From Baseline in Dyspnea Functional Impairment at Week 156/Early Termination |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. Changes in dyspnea functional impairment were assessed on a scale from Major Deterioration (formerly working but had to stop working and abandoned usual activities due to shortness of breath) to Major Improvement (able to return to work at former pace and return to full activities with only mild restriction due to improvement of shortness of breath). Further impairment for other reasons includes participants who gave up or reduced work or other activities for reasons other than shortness of breath. |
| Time Frame | Week 156 or the Extension Phase Early Termination visit |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 6 | 2 |
| Major deterioration |
0
0%
|
0
0%
|
| Moderate deterioration |
0
0%
|
1
9.1%
|
| Minor deterioration |
2
16.7%
|
1
9.1%
|
| No change |
2
16.7%
|
0
0%
|
| Minor improvement |
1
8.3%
|
0
0%
|
| Moderate improvement |
1
8.3%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
0
0%
|
| Title | Change From Baseline in Dyspnea Magnitude of Task at Week 12 |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain. |
| Time Frame | Week 12 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 10 | 7 |
| Major deterioration |
0
0%
|
0
0%
|
| Moderate deterioration |
0
0%
|
0
0%
|
| Minor deterioration |
2
16.7%
|
1
9.1%
|
| No change |
5
41.7%
|
5
45.5%
|
| Minor improvement |
3
25%
|
1
9.1%
|
| Moderate improvement |
0
0%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
0
0%
|
| Title | Change From Baseline in Dyspnea Magnitude of Task at Week 24 |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain. |
| Time Frame | Week 24 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 10 | 7 |
| Major deterioration |
0
0%
|
0
0%
|
| Moderate deterioration |
1
8.3%
|
1
9.1%
|
| Minor deterioration |
2
16.7%
|
1
9.1%
|
| No change |
5
41.7%
|
2
18.2%
|
| Minor improvement |
2
16.7%
|
3
27.3%
|
| Moderate improvement |
0
0%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
0
0%
|
| Title | Change From Baseline in Dyspnea Magnitude of Task at Week 52/Early Termination |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain. |
| Time Frame | Week 52 or at the Treatment Phase Early Termination visit |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 12 | 10 |
| Major deterioration |
0
0%
|
0
0%
|
| Moderate deterioration |
0
0%
|
3
27.3%
|
| Minor deterioration |
1
8.3%
|
1
9.1%
|
| No change |
8
66.7%
|
6
54.5%
|
| Minor improvement |
2
16.7%
|
0
0%
|
| Moderate improvement |
0
0%
|
0
0%
|
| Major improvement |
1
8.3%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
0
0%
|
| Title | Change From Baseline in Dyspnea Magnitude of Task at Week 64 |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain. |
| Time Frame | Week 64 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 6 | 2 |
| Major deterioration |
0
0%
|
1
9.1%
|
| Moderate deterioration |
0
0%
|
0
0%
|
| Minor deterioration |
2
16.7%
|
0
0%
|
| No change |
3
25%
|
0
0%
|
| Minor improvement |
1
8.3%
|
1
9.1%
|
| Moderate improvement |
0
0%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
0
0%
|
| Title | Change From Baseline in Dyspnea Magnitude of Task at Week 76 |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carrying very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain. |
| Time Frame | Week 76 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 2 | 1 |
| Major deterioration |
0
0%
|
0
0%
|
| Moderate deterioration |
0
0%
|
0
0%
|
| Minor deterioration |
1
8.3%
|
0
0%
|
| No change |
1
8.3%
|
0
0%
|
| Minor improvement |
0
0%
|
1
9.1%
|
| Moderate improvement |
0
0%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
0
0%
|
| Title | Change From Baseline in Dyspnea Magnitude of Task at Week 156/Early Termination |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with extraordinary activity such as running or carry very heavy loads). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (deteriorated ≥ 2 grades from Baseline) to Major Improvement (Improved ≥ 2 grades from Baseline). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath, for example musculoskeletal problems or chest pain. |
| Time Frame | Week 156 or the Extension Phase Early Termination visit |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 6 | 2 |
| Major deterioration |
0
0%
|
0
0%
|
| Moderate deterioration |
1
8.3%
|
0
0%
|
| Minor deterioration |
1
8.3%
|
1
9.1%
|
| No change |
2
16.7%
|
0
0%
|
| Minor improvement |
2
16.7%
|
1
9.1%
|
| Moderate improvement |
0
0%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
0
0%
|
| Title | Change From Baseline in Dyspnea Magnitude of Effort at Week 12 |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath. |
| Time Frame | Week 12 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 10 | 7 |
| Major deterioration |
0
0%
|
0
0%
|
| Moderate deterioration |
1
8.3%
|
0
0%
|
| Minor deterioration |
2
16.7%
|
2
18.2%
|
| No change |
4
33.3%
|
4
36.4%
|
| Minor improvement |
2
16.7%
|
1
9.1%
|
| Moderate improvement |
1
8.3%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
0
0%
|
| Title | Change From Baseline in Dyspnea Magnitude of Effort at Week 24 |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath. |
| Time Frame | Week 24 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 10 | 7 |
| Major deterioration |
1
8.3%
|
1
9.1%
|
| Moderate deterioration |
0
0%
|
1
9.1%
|
| Minor deterioration |
1
8.3%
|
0
0%
|
| No change |
4
33.3%
|
3
27.3%
|
| Minor improvement |
3
25%
|
2
18.2%
|
| Moderate improvement |
1
8.3%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
0
0%
|
| Title | Change From Baseline in Dyspnea Magnitude of Effort at Week 52/Early Termination |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath. |
| Time Frame | Week 52 or at the Treatment Phase Early Termination visit |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 12 | 10 |
| Major deterioration |
0
0%
|
0
0%
|
| Moderate deterioration |
0
0%
|
3
27.3%
|
| Minor deterioration |
1
8.3%
|
1
9.1%
|
| No change |
7
58.3%
|
6
54.5%
|
| Minor improvement |
3
25%
|
0
0%
|
| Moderate improvement |
1
8.3%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
0
0%
|
| Title | Change From Baseline in Dyspnea Magnitude of Effort at Week 64 |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath. |
| Time Frame | Week 64 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 6 | 2 |
| Major deterioration |
0
0%
|
1
9.1%
|
| Moderate deterioration |
0
0%
|
0
0%
|
| Minor deterioration |
3
25%
|
0
0%
|
| No change |
2
16.7%
|
1
9.1%
|
| Minor improvement |
1
8.3%
|
0
0%
|
| Moderate improvement |
0
0%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
0
0%
|
| Title | Change From Baseline in Dyspnea Magnitude of Effort at Week 76 |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath. |
| Time Frame | Week 76 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 2 | 1 |
| Major deterioration |
0
0%
|
0
0%
|
| Moderate deterioration |
0
0%
|
0
0%
|
| Minor deterioration |
1
8.3%
|
0
0%
|
| No change |
0
0%
|
1
9.1%
|
| Minor improvement |
1
8.3%
|
0
0%
|
| Moderate improvement |
0
0%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
0
0%
|
| Title | Change From Baseline in Dyspnea Magnitude of Effort at Week 156/Early Termination |
|---|---|
| Description | The Transition Dyspnea Index (TDI) provides interview-based measurements of breathlessness related to activities of daily living. The TDI is an evaluative instrument that includes specific criteria for each of three components (functional impairment, magnitude of task and magnitude of effort) to measure changes from a baseline state. At Baseline magnitude of task was assessed on a scale from Grade 0 (becomes short of breath at rest, while sitting or lying) to Grade 4 (becomes short of breath only with greatest imaginable effort). Changes in dyspnea magnitude of task were assessed on a scale from Major Deterioration (severe decrease in effort from Baseline to avoid shortness of breath, activities take 50-100% longer to complete) to Major Improvement (able to do things with much greater effort than previously with few, if any, pauses). Further impairment for other reasons includes participants with reduced exertion capacity for reasons other than shortness of breath. |
| Time Frame | Week 156 or at the Extension Phase Early Termination visit |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with available data |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 6 | 2 |
| Major deterioration |
0
0%
|
0
0%
|
| Moderate deterioration |
1
8.3%
|
0
0%
|
| Minor deterioration |
2
16.7%
|
1
9.1%
|
| No change |
2
16.7%
|
1
9.1%
|
| Minor improvement |
1
8.3%
|
0
0%
|
| Moderate improvement |
0
0%
|
0
0%
|
| Major improvement |
0
0%
|
0
0%
|
| Further impairment for other reasons |
0
0%
|
0
0%
|
| Title | Oxygen Saturation Over Time |
|---|---|
| Description | Oxygen saturation was measured by pulse oximetry. |
| Time Frame | Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit). |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS participants with a value at each time point. |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 12 | 9 |
| Baseline |
97.5
(2.07)
|
96.8
(1.92)
|
| Week 12 |
97.1
(2.18)
|
97.4
(1.51)
|
| Week 24 |
97.6
(1.65)
|
96.5
(4.31)
|
| Week 52/Early Termination |
96.8
(2.18)
|
96.8
(1.86)
|
| Week 64 |
96.3
(2.94)
|
94.0
(2.83)
|
| Week 76 |
98.5
(0.71)
|
97.0
(NA)
|
| Week 156/Early Termination |
95.8
(5.12)
|
97.5
(0.71)
|
| Title | Pharmacokinetic Parameters of Pomalidomide in Plasma |
|---|---|
| Description | Pharmacokinetic (PK) analyses were not conducted as there were too few participants with available data. |
| Time Frame | Day 1 and week 4 pre-dose and up to 24 hours post-dose. |
Outcome Measure Data
| Analysis Population Description |
|---|
| PK analyses were not conducted as there were too few participants with available data. No data was analyzed. |
| Arm/Group Title | Placebo | Pomalidomide |
|---|---|---|
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and for up to 2 years during the open-label extension phase. |
| Measure Participants | 0 | 0 |
Adverse Events
| Time Frame | From the start of study drug to 28 days after last dose; Treatment Phase median duration of treatment was 358 and 320 days for Placebo and Pomalidomide; Extension phase median duration of treatment was 161 days and 194 days for Placebo and Pomalidomide. | |||||||
|---|---|---|---|---|---|---|---|---|
| Adverse Event Reporting Description | ||||||||
| Arm/Group Title | Treatment Phase: Placebo | Treatment Phase: Pomalidomide | Extension Phase: Placebo/Pomalidomide | Extension Phase: Pomalidomide/Pomalidomide | ||||
| Arm/Group Description | Participants received placebo orally once a day for 52 weeks during the treatment phase. | Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase. | In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years. | Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase. | ||||
All Cause Mortality |
||||||||
| Treatment Phase: Placebo | Treatment Phase: Pomalidomide | Extension Phase: Placebo/Pomalidomide | Extension Phase: Pomalidomide/Pomalidomide | |||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | / (NaN) | / (NaN) | / (NaN) | / (NaN) | ||||
Serious Adverse Events |
||||||||
| Treatment Phase: Placebo | Treatment Phase: Pomalidomide | Extension Phase: Placebo/Pomalidomide | Extension Phase: Pomalidomide/Pomalidomide | |||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 1/12 (8.3%) | 4/10 (40%) | 0/6 (0%) | 1/2 (50%) | ||||
| Infections and infestations | ||||||||
| Pneumonia | 1/12 (8.3%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Sepsis | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Upper respiratory tract infection | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Renal and urinary disorders | ||||||||
| Renal failure | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Respiratory, thoracic and mediastinal disorders | ||||||||
| Acute respiratory failure | 0/12 (0%) | 0/10 (0%) | 0/6 (0%) | 1/2 (50%) | ||||
| Chronic respiratory failure | 0/12 (0%) | 0/10 (0%) | 0/6 (0%) | 1/2 (50%) | ||||
| Pulmonary embolism | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Pulmonary hypertension | 0/12 (0%) | 0/10 (0%) | 0/6 (0%) | 1/2 (50%) | ||||
Other (Not Including Serious) Adverse Events |
||||||||
| Treatment Phase: Placebo | Treatment Phase: Pomalidomide | Extension Phase: Placebo/Pomalidomide | Extension Phase: Pomalidomide/Pomalidomide | |||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 12/12 (100%) | 9/10 (90%) | 5/6 (83.3%) | 2/2 (100%) | ||||
| Blood and lymphatic system disorders | ||||||||
| Anaemia | 1/12 (8.3%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Splenomegaly | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Cardiac disorders | ||||||||
| Palpitations | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Ear and labyrinth disorders | ||||||||
| Tinnitus | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Eye disorders | ||||||||
| Conjunctivitis | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Gastrointestinal disorders | ||||||||
| Abdominal pain upper | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Barrett's oesophagus | 0/12 (0%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Colonic polyp | 0/12 (0%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Constipation | 1/12 (8.3%) | 3/10 (30%) | 0/6 (0%) | 0/2 (0%) | ||||
| Diarrhoea | 3/12 (25%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Dry mouth | 1/12 (8.3%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Dyspepsia | 0/12 (0%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Gastric polyps | 0/12 (0%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Gastrooesophageal reflux disease | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 1/2 (50%) | ||||
| Hiatus hernia | 0/12 (0%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Mouth ulceration | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Mucous stools | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Nausea | 2/12 (16.7%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| General disorders | ||||||||
| Chills | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Device breakage | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Influenza like illness | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Non-cardiac chest pain | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Oedema | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 1/2 (50%) | ||||
| Oedema peripheral | 1/12 (8.3%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Pain | 1/12 (8.3%) | 1/10 (10%) | 0/6 (0%) | 1/2 (50%) | ||||
| Pyrexia | 0/12 (0%) | 1/10 (10%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Hepatobiliary disorders | ||||||||
| Biliary colic | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Cholecystitis | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Cholelithiasis | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Hepatic steatosis | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Immune system disorders | ||||||||
| Contrast media allergy | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Seasonal allergy | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Infections and infestations | ||||||||
| Bronchitis | 0/12 (0%) | 2/10 (20%) | 0/6 (0%) | 0/2 (0%) | ||||
| Diverticulitis | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Folliculitis | 0/12 (0%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Gastroenteritis | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Infected skin ulcer | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Influenza | 2/12 (16.7%) | 0/10 (0%) | 2/6 (33.3%) | 0/2 (0%) | ||||
| Laryngitis viral | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Localised infection | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Lower respiratory tract infection | 0/12 (0%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Tooth abscess | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Upper respiratory tract infection | 3/12 (25%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Urinary tract infection | 2/12 (16.7%) | 0/10 (0%) | 2/6 (33.3%) | 0/2 (0%) | ||||
| Viral upper respiratory tract infection | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Injury, poisoning and procedural complications | ||||||||
| Contusion | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Fall | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Ligament sprain | 0/12 (0%) | 2/10 (20%) | 0/6 (0%) | 0/2 (0%) | ||||
| Procedural pain | 0/12 (0%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Investigations | ||||||||
| Alanine aminotransferase increased | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Aspartate aminotransferase increased | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Blood creatinine increased | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Blood iron decreased | 0/12 (0%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Blood lactate dehydrogenase increased | 0/12 (0%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| C-reactive protein increased | 1/12 (8.3%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Heart rate increased | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Hepatic enzyme increased | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| White blood cell count increased | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Metabolism and nutrition disorders | ||||||||
| Hyperglycaemia | 0/12 (0%) | 0/10 (0%) | 0/6 (0%) | 1/2 (50%) | ||||
| Hypomagnesaemia | 1/12 (8.3%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Hypophosphataemia | 0/12 (0%) | 0/10 (0%) | 0/6 (0%) | 1/2 (50%) | ||||
| Iron deficiency | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Vitamin D deficiency | 0/12 (0%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Musculoskeletal and connective tissue disorders | ||||||||
| Arthralgia | 4/12 (33.3%) | 4/10 (40%) | 0/6 (0%) | 0/2 (0%) | ||||
| Joint swelling | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Muscle spasms | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Musculoskeletal chest pain | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Myalgia | 1/12 (8.3%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Pain in jaw | 0/12 (0%) | 0/10 (0%) | 0/6 (0%) | 1/2 (50%) | ||||
| Synovitis | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Tendon disorder | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Tendon pain | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ||||||||
| Seborrhoeic keratosis | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Nervous system disorders | ||||||||
| Ageusia | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Dizziness | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 1/2 (50%) | ||||
| Headache | 3/12 (25%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Hypoaesthesia | 1/12 (8.3%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Migraine | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Psychiatric disorders | ||||||||
| Anxiety | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Depressed mood | 0/12 (0%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Insomnia | 1/12 (8.3%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Renal and urinary disorders | ||||||||
| Dysuria | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Glycosuria | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Renal failure | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Reproductive system and breast disorders | ||||||||
| Amenorrhoea | 1/12 (8.3%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Nipple pain | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Ovarian cyst ruptured | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Respiratory, thoracic and mediastinal disorders | ||||||||
| Cough | 1/12 (8.3%) | 1/10 (10%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Dysphonia | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Dyspnoea | 2/12 (16.7%) | 0/10 (0%) | 0/6 (0%) | 1/2 (50%) | ||||
| Oropharyngeal pain | 2/12 (16.7%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Productive cough | 0/12 (0%) | 0/10 (0%) | 2/6 (33.3%) | 0/2 (0%) | ||||
| Pulmonary hypertension | 1/12 (8.3%) | 0/10 (0%) | 1/6 (16.7%) | 1/2 (50%) | ||||
| Rhinorrhoea | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Sinus congestion | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Skin and subcutaneous tissue disorders | ||||||||
| Alopecia | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Butterfly rash | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Dermatitis acneiform | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Digital ulcer | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Dry skin | 1/12 (8.3%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Eczema | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Hyperhidrosis | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Hyperkeratosis | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Macule | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Night sweats | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Papule | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Rash | 1/12 (8.3%) | 2/10 (20%) | 0/6 (0%) | 0/2 (0%) | ||||
| Rash maculo-papular | 0/12 (0%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Rash papular | 0/12 (0%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Rash pruritic | 0/12 (0%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Rosacea | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Seborrhoeic dermatitis | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Skin ulcer | 2/12 (16.7%) | 0/10 (0%) | 2/6 (33.3%) | 0/2 (0%) | ||||
| Toxic skin eruption | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Social circumstances | ||||||||
| Menopause | 1/12 (8.3%) | 0/10 (0%) | 0/6 (0%) | 0/2 (0%) | ||||
| Vascular disorders | ||||||||
| Hot flush | 1/12 (8.3%) | 0/10 (0%) | 1/6 (16.7%) | 0/2 (0%) | ||||
| Hypertension | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
| Raynaud's phenomenon | 0/12 (0%) | 1/10 (10%) | 0/6 (0%) | 0/2 (0%) | ||||
Limitations/Caveats
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
Results from a center cannot be submitted for publication before results of multicenter study are published unless it is more than 1year since study completion. Then, Investigator can publish if manuscript is submitted to Celgene 60 days prior to submission. If Celgene decides publication would hinder drug development, Investigator must delay submission for up to 90 days. Investigator must delete confidential information before submission or defer publication to permit patent applications.
Results Point of Contact
| Name/Title | Clinical Trial Disclosure |
|---|---|
| Organization | Celgene Corporation |
| Phone | 888-260-1599 |
| ClinicalTrialDisclosure@Celgene.com |
- CC-4047-SSC-001
- 2010-023047-15