A Trial of PF-06252616 in Ambulatory Participants With LGMD2I
Sponsor
Kathryn Wagner (Other)
Overall Status
Completed
CT.gov ID
NCT02841267
Collaborator
Pfizer (Industry)
19
1
3
30
0.6
Study Details
Study Description
Brief Summary
The investigational product PF 06252616, a humanized anti myostatin monoclonal antibody that neutralizes myostatin (GDF8) is in development for the treatment of Limb Girdle Muscular Dystrophy 2I (LGMD2I) to preserve and/or improve muscle function.
This study will provide the clinical assessment of the safety, tolerability, Pharmacokinetics and Pharmacodynamics of PF 06252616 following repeat IV doses in ambulatory adults with LGMD2I.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1/Phase 2 |
Detailed Description
This study is a Phase 1b/2, open-label multiple ascending dose escalation study to evaluate the safety, tolerability, efficacy, PK and PD of PF 06252616 in ambulatory adults with LGMD2I. The study design is intended to determine the optimal safe and pharmacologically active dose of PF 06252616 in LGMD2I while providing an opportunity for all subjects to receive active drug for a rare and disabling disorder. The study will be conducted in three periods: Lead-In, Treatment and Follow-up periods. The Lead-In and Follow-up periods will each be 16 weeks to allow an assessment of the change of various outcome measures of this period of time and comparison of change in function before, during and after treatment. The Treatment period will be 32 weeks. Three cohorts of participants will be enrolled and receive escalating doses of PF 06252616. The first cohort will have the option to crossover to the highest dose.
Study Design
Study Type:
Interventional
Actual Enrollment
:
19 participants
Allocation:
Non-Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase 1b/2, Open-Label, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Ambulatory Participants With LGMD2I
Study Start Date
:
Jul 1, 2016
Actual Primary Completion Date
:
Jan 1, 2019
Actual Study Completion Date
:
Jan 1, 2019
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Active Comparator: Low Dose, Cohort 1 4 subjects will be enrolled in cohort 1 and will receive an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, if no stopping rules have been met, subjects will be receive an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. |
Drug: PF 06252616
|
| Active Comparator: Middle dose, Cohort 2 8 subjects will be enrolled in cohort 2 and receive 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. |
Drug: PF 06252616
|
| Active Comparator: High dose, Cohort 3 8 subjects will be enrolled in cohort 3 and receive 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks. |
Drug: PF 06252616
|
Outcome Measures
Primary Outcome Measures
- Incidence of Dose Limiting or Intolerability Treatment Related Adverse Events [Baseline through 64 weeks]
Adverse events include subject-reported symptoms as well as clinically-significant changes in laboratory testing, vital signs, and suicide screening (based on the Columbia Suicide Severity Rating Scale).
Secondary Outcome Measures
- Maximum Observed Serum Concentration at Steady State (Cmax, ss) of GDF-8 [Day 337 and Day 393 for Cohort 1; Day 113 and 169 for Cohorts 2 and 3]
The concentration of myostatin (GDF-8) was measured in serum 2 hours after dose administration at two visits (Day 337 and Day 393 for Cohort 1; Day 113 and 169 for Cohorts 2 and 3)where drug concentration had reached steady state. The highest concentration from these two time points was averaged for each cohort.
- Minimum Observed Serum Trough Concentration at Steady State (Ctrough,ss) of GDF-8 [Day 337 and Day 393 for Cohort 1; Day 113 and 169 for Cohorts 2 and 3]
The concentration of myostatin (GDF-8) was measured in serum prior to dose administration at two time points (Day 337 and Day 393 for Cohort 1; Day 113 and 169 for Cohorts 2 and 3) where drug concentration had reached steady state. The lowest concentration from these two time points was averaged for each cohort.
- Maximum Observed Serum Concentration (Cmax) of PF-06252616 [Day 113 and Day 169]
The peak concentration of study drug (PF-06252616) was measured in serum following dose administration at two time points (Day 113 and Day 169) where drug concentration had reached steady state. The higher concentration from these two time points was averaged for each cohort.
- Minimum Observed Serum Trough Concentration (Ctrough) of PF-06252616 [Day 113 and Day 169]
The peak concentration of study drug (PF-06252616) was measured in serum prior to dose administration at two time points (Day 113 and Day 169) where drug concentration had reached steady state. The lower concentration from these two time points was averaged for each cohort.
- Immunogenicity: Incidence of Anti-drug Antibody [Baseline through 96 weeks]
Blood samples were tested for the presence of anti-drug antibodies prior to the initiation of study drug, at dose escalation (Cohort 1 only), and at 3 separate time points after the last dose was given.
- Mean Change From Baseline in 10 Meter Walk/Run Time in Seconds [Baseline through 32 weeks]
Subjects are asked to run or walk as quickly as possible for 10 meters from a standing position. The total time to traverse 10 meters is recorded in seconds.
- Mean Change From Baseline of Forced Vital Capacity in Liters [Baseline through 32 weeks]
The total forced vital capacity was measured using a bedside spirometer. The best of 3 trials was recorded.
- Mean Change From Baseline in 2MWD in Meters [Baseline through 32 weeks]
Average change in distance (in meters) walked in 2 minutes.
- Mean Change From Baseline in TUG in Seconds [Baseline through 32 weeks]
The timed-up-and-go test (TUG) is the total time it takes the subject to rise from a seated position, walk to a marker 3 meters away, return to the chair, and sit.
- Mean Change From Baseline in Muscle Strength as Measured by Modified MRC Scale [Baseline through 32 weeks]
Twenty-two muscle groups were measured on a modified MRC scale ranging from 1 through 12. The total scores from all 22 muscle groups were added to generate a summary score ranging from 12 to 264 with higher scores signifying greater strength. The change in summary score was calculated over the first 32 weeks of treatment.
Eligibility Criteria
Criteria
Ages Eligible for Study:
18 Years
to 99 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
-
Male and female patients age ≥ 18
-
Diagnosis of LGMD2I as defined by clinical presentation consistent with LGMD2I and FKRP gene testing showing biallelic alterations known or likely to be pathogenic. Diagnosis must be confirmed in subject's medical history and by genetic testing obtained during routine clinical care for diagnostic purposes as reported from an appropriate regulated laboratory using a clinically validated genetic test (genetic testing is not provided by the sponsor).
-
Ability to walk/run 10m
-
Ability to rise from chair
-
Adequate hepatic and renal function on screening laboratory assessments
-
Iron content estimate on the screening liver MRI within the normal range as determined by R2* value (R2* ≤ 139 Hz at 3.0T).
-
Participant must provide written informed consent for participating in study.
-
Participant must possess the ability, per the Principal Investigator (PI), to understand and comply with protocol instruction for the entire duration of the study.
Exclusion Criteria:
-
Known cognitive impairment or behavioral issues that would impede the ability to provide informed consent or to follow study instructions.
-
History of major surgical procedure within 6 weeks of signing the informed consent or planned surgery during the study.
-
Any injury which may impact functional testing. Previous injuries must be fully healed prior to consent. Prior lower limb fractures must be fully healed and at least 3 months from injury dates.
-
Previous treatment with another investigational product within 30 days or 5 half-lives, (whichever is longer) prior to consenting.
-
Corticosteroid treatment within 3 months prior to consenting.
-
Compromised cardiac function (left ventricular ejection fraction <50%).
-
Unwilling or unable (e.g. metal implants, requires sedation) to undergo examination with closed MRI without sedation.
-
History of allergic or anaphylactic reaction to a therapeutic or diagnostic protein.
-
Female subjects who are pregnant or nursing.
-
Subjects who, are biologically capable of having children who are unwilling or unable to use highly effective methods of contraception (as outlined in this protocol) during sexual activity for the duration of the study and through completion of final study visit.
-
Predisposition to iron accumulation. (Serum iron >1.2 X ULN, serum ferritin >1.2 ULNN).
-
Underlying disposition for bleeding disorder on screening laboratory assessment (PT/INR>1.25 X ULN, aPTT > 1.25 ULN, fecal occult blood is positive)
-
Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, neurologic, or allergic disease.
-
Unwillingness or inability to comply with the requirements of this protocol (in the opinion of the PI) including, but not limited to, the presence of any condition (physical, mental, or social) that is likely to affect the participant's ability to return for study visits or adhere to the visit schedule.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | Baltimore | Maryland | United States | 21205 |
Sponsors and Collaborators
- Kathryn Wagner
- Pfizer
Investigators
- Principal Investigator: Kathryn R Wagner, MD/PhD, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Study Documents (Full-Text)
More Information
Publications
None provided.Responsible Party:
Kathryn Wagner,
Director, Center for Genetic Muscle Disorders,
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
ClinicalTrials.gov Identifier:
NCT02841267
Other Study ID Numbers:
- WI203720
First Posted:
Jul 22, 2016
Last Update Posted:
Oct 19, 2020
Last Verified:
Sep 1, 2020
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail | One participant in Cohort 2 was consented, but elected to withdraw from the study prior to assignment to a treatment arm. |
| Arm/Group Title | Low Dose, Cohort 1 | Middle Dose, Cohort 2 | High Dose, Cohort 3 |
|---|---|---|---|
| Arm/Group Description | 4 subjects will be enrolled in cohort 1 and will receive an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, if no stopping rules have been met, subjects will be receive an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. PF 06252616 | 8 subjects will be enrolled in cohort 2 and receive 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. PF 06252616 | 8 subjects will be enrolled in cohort 3 and receive 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks. PF 06252616 |
| Period Title: Lead-in (Untreated) | |||
| STARTED | 4 | 7 | 8 |
| COMPLETED | 4 | 7 | 8 |
| NOT COMPLETED | 0 | 0 | 0 |
| Period Title: Lead-in (Untreated) | |||
| STARTED | 4 | 7 | 8 |
| COMPLETED | 4 | 7 | 8 |
| NOT COMPLETED | 0 | 0 | 0 |
| Period Title: Lead-in (Untreated) | |||
| STARTED | 4 | 6 | 6 |
| COMPLETED | 4 | 6 | 6 |
| NOT COMPLETED | 0 | 0 | 0 |
Baseline Characteristics
| Arm/Group Title | Cohort 1 - Low Dose | Cohort 2 - Middle Dose | Cohort 3 - High Dose | Total |
|---|---|---|---|---|
| Arm/Group Description | 4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. | 7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. | 8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks. | Total of all reporting groups |
| Overall Participants | 4 | 7 | 8 | 19 |
| Age (years) [Mean (Standard Deviation) ] | ||||
| Mean (Standard Deviation) [years] |
45
(8)
|
41
(18)
|
34
(9.9)
|
39
(13)
|
| Sex: Female, Male (Count of Participants) | ||||
| Female |
3
75%
|
4
57.1%
|
5
62.5%
|
12
63.2%
|
| Male |
1
25%
|
3
42.9%
|
3
37.5%
|
7
36.8%
|
| Race (NIH/OMB) (Count of Participants) | ||||
| American Indian or Alaska Native |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Asian |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Native Hawaiian or Other Pacific Islander |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Black or African American |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| White |
4
100%
|
7
100%
|
8
100%
|
19
100%
|
| More than one race |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Unknown or Not Reported |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
Outcome Measures
| Title | Incidence of Dose Limiting or Intolerability Treatment Related Adverse Events |
|---|---|
| Description | Adverse events include subject-reported symptoms as well as clinically-significant changes in laboratory testing, vital signs, and suicide screening (based on the Columbia Suicide Severity Rating Scale). |
| Time Frame | Baseline through 64 weeks |
Outcome Measure Data
| Analysis Population Description |
|---|
| All enrolled subjects are included in the analysis. |
| Arm/Group Title | Cohort 1 | Cohort 2, Middle Dose | Cohort 3, High Dose |
|---|---|---|---|
| Arm/Group Description | 4 subjects received 32 weeks of treatment with 5mg/kg PF 06252616 IV every 4 weeks followed by an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. | 7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. | 8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks. |
| Measure Participants | 4 | 7 | 8 |
| Any adverse event |
45
|
21
|
40
|
| Serious adverse event |
0
|
0
|
0
|
| Title | Maximum Observed Serum Concentration at Steady State (Cmax, ss) of GDF-8 |
|---|---|
| Description | The concentration of myostatin (GDF-8) was measured in serum 2 hours after dose administration at two visits (Day 337 and Day 393 for Cohort 1; Day 113 and 169 for Cohorts 2 and 3)where drug concentration had reached steady state. The highest concentration from these two time points was averaged for each cohort. |
| Time Frame | Day 337 and Day 393 for Cohort 1; Day 113 and 169 for Cohorts 2 and 3 |
Outcome Measure Data
| Analysis Population Description |
|---|
| All enrolled participants were analyzed. For Cohort 1, measurements are provided for the 40mg/kg dosing period. |
| Arm/Group Title | Cohort 1 - Low Dose | Cohort 2 - Middle Dose | Cohort 3 - High Dose |
|---|---|---|---|
| Arm/Group Description | 4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. | 7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. | 8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks. |
| Measure Participants | 4 | 7 | 8 |
| Mean (Standard Deviation) [ng/ml] |
9.02
(3.20)
|
8.96
(3.02)
|
9.67
(6.45)
|
| Title | Minimum Observed Serum Trough Concentration at Steady State (Ctrough,ss) of GDF-8 |
|---|---|
| Description | The concentration of myostatin (GDF-8) was measured in serum prior to dose administration at two time points (Day 337 and Day 393 for Cohort 1; Day 113 and 169 for Cohorts 2 and 3) where drug concentration had reached steady state. The lowest concentration from these two time points was averaged for each cohort. |
| Time Frame | Day 337 and Day 393 for Cohort 1; Day 113 and 169 for Cohorts 2 and 3 |
Outcome Measure Data
| Analysis Population Description |
|---|
| All enrolled participants were analyzed. For Cohort 1, measurements are provided for the 40mg/kg dosing period. |
| Arm/Group Title | Cohort 1 - Low Dose | Cohort 2 - Middle Dose | Cohort 3 - High Dose |
|---|---|---|---|
| Arm/Group Description | 4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. | 7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. | 8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks. |
| Measure Participants | 4 | 7 | 8 |
| Mean (Standard Deviation) [ng/ml] |
8.80
(2.96)
|
9.68
(2.21)
|
9.78
(7.61)
|
| Title | Maximum Observed Serum Concentration (Cmax) of PF-06252616 |
|---|---|
| Description | The peak concentration of study drug (PF-06252616) was measured in serum following dose administration at two time points (Day 113 and Day 169) where drug concentration had reached steady state. The higher concentration from these two time points was averaged for each cohort. |
| Time Frame | Day 113 and Day 169 |
Outcome Measure Data
| Analysis Population Description |
|---|
| All enrolled participants were analyzed. For Cohort 1, measurements are provided for 5mg/kg dosing period. |
| Arm/Group Title | Cohort 1 - Low Dose | Cohort 2 - Middle Dose | Cohort 3 - High Dose |
|---|---|---|---|
| Arm/Group Description | 4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. | 7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. | 8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks. |
| Measure Participants | 4 | 7 | 8 |
| Mean (Standard Deviation) [ng/ml] |
215306.5
(29490.4)
|
779015.7
(115450.0)
|
1625203
(271159.6)
|
| Title | Minimum Observed Serum Trough Concentration (Ctrough) of PF-06252616 |
|---|---|
| Description | The peak concentration of study drug (PF-06252616) was measured in serum prior to dose administration at two time points (Day 113 and Day 169) where drug concentration had reached steady state. The lower concentration from these two time points was averaged for each cohort. |
| Time Frame | Day 113 and Day 169 |
Outcome Measure Data
| Analysis Population Description |
|---|
| All enrolled participants were analyzed. For Cohort 1, measurements are provided for the 5mg/kg dosing period. |
| Arm/Group Title | Cohort 1 - Low Dose | Cohort 2 - Middle Dose | Cohort 3 - High Dose |
|---|---|---|---|
| Arm/Group Description | 4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. | 7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. | 8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks. |
| Measure Participants | 4 | 7 | 7 |
| Mean (Standard Deviation) [ng/ml] |
67016.8
(25887.3)
|
224024.1
(58734.5)
|
408814.1
(96607.6)
|
| Title | Immunogenicity: Incidence of Anti-drug Antibody |
|---|---|
| Description | Blood samples were tested for the presence of anti-drug antibodies prior to the initiation of study drug, at dose escalation (Cohort 1 only), and at 3 separate time points after the last dose was given. |
| Time Frame | Baseline through 96 weeks |
Outcome Measure Data
| Analysis Population Description |
|---|
| All enrolled subjects were included in the analysis. |
| Arm/Group Title | Cohort 1 - Low Dose | Cohort 2 - Middle Dose | Cohort 3 - High Dose |
|---|---|---|---|
| Arm/Group Description | 4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. | 7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. | 8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks. |
| Measure Participants | 4 | 7 | 8 |
| Count of Participants [Participants] |
0
0%
|
0
0%
|
0
0%
|
| Title | Mean Change From Baseline in 10 Meter Walk/Run Time in Seconds |
|---|---|
| Description | Subjects are asked to run or walk as quickly as possible for 10 meters from a standing position. The total time to traverse 10 meters is recorded in seconds. |
| Time Frame | Baseline through 32 weeks |
Outcome Measure Data
| Analysis Population Description |
|---|
| All enrolled subject were included in the analysis. For Cohort 1, data is provided for the 5mg/kg dosing period. |
| Arm/Group Title | Cohort 1 - Low Dose | Cohort 2 - Middle Dose | Cohort 3 - High Dose |
|---|---|---|---|
| Arm/Group Description | 4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. | 7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. | 8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks. |
| Measure Participants | 4 | 7 | 8 |
| Mean (Standard Deviation) [seconds] |
1.60
(2.95)
|
-0.09
(0.11)
|
0.18
(0.31)
|
| Title | Mean Change From Baseline of Forced Vital Capacity in Liters |
|---|---|
| Description | The total forced vital capacity was measured using a bedside spirometer. The best of 3 trials was recorded. |
| Time Frame | Baseline through 32 weeks |
Outcome Measure Data
| Analysis Population Description |
|---|
| All enrolled subjects were included in the analysis. For Cohort 1, data is provided for the 5mg/kg dosing period. |
| Arm/Group Title | Cohort 1 - Low Dose | Cohort 2 - Middle Dose | Cohort 3 - High Dose |
|---|---|---|---|
| Arm/Group Description | 4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. | 7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. | 8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks. |
| Measure Participants | 4 | 7 | 8 |
| Mean (Standard Deviation) [liters] |
0.03
(0.14)
|
0.02
(0.15)
|
-0.05
(0.14)
|
| Title | Mean Change From Baseline in 2MWD in Meters |
|---|---|
| Description | Average change in distance (in meters) walked in 2 minutes. |
| Time Frame | Baseline through 32 weeks |
Outcome Measure Data
| Analysis Population Description |
|---|
| All enrolled participants were analyzed. For Cohort 1, measurements are provided for the 5mg/kg dosing period. |
| Arm/Group Title | Cohort 1 - Low Dose | Cohort 2 - Middle Dose | Cohort 3 - High Dose |
|---|---|---|---|
| Arm/Group Description | 4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. | 7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. | 8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks. |
| Measure Participants | 4 | 7 | 8 |
| Mean (Standard Deviation) [meters] |
-4.25
(9.91)
|
2.00
(7.77)
|
2.13
(5.17)
|
| Title | Mean Change From Baseline in TUG in Seconds |
|---|---|
| Description | The timed-up-and-go test (TUG) is the total time it takes the subject to rise from a seated position, walk to a marker 3 meters away, return to the chair, and sit. |
| Time Frame | Baseline through 32 weeks |
Outcome Measure Data
| Analysis Population Description |
|---|
| All enrolled subjects are included in the analysis. For Cohort 1, data is provided for the 5mg/kg dosing period. |
| Arm/Group Title | Cohort 1 - Low Dose | Cohort 2 - Middle Dose | Cohort 3 - High Dose |
|---|---|---|---|
| Arm/Group Description | 4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. | 7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. | 8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks. |
| Measure Participants | 4 | 7 | 8 |
| Mean (Standard Deviation) [seconds] |
0.67
(1.15)
|
0.29
(1.11)
|
-0.50
(1.51)
|
| Title | Mean Change From Baseline in Muscle Strength as Measured by Modified MRC Scale |
|---|---|
| Description | Twenty-two muscle groups were measured on a modified MRC scale ranging from 1 through 12. The total scores from all 22 muscle groups were added to generate a summary score ranging from 12 to 264 with higher scores signifying greater strength. The change in summary score was calculated over the first 32 weeks of treatment. |
| Time Frame | Baseline through 32 weeks |
Outcome Measure Data
| Analysis Population Description |
|---|
| All enrolled participants were analyzed after 32 weeks of treatment. |
| Arm/Group Title | Cohort 1 - Low Dose | Cohort 2 - Middle Dose | Cohort 3 - High Dose |
|---|---|---|---|
| Arm/Group Description | 4 subjects were enrolled in cohort 1 and received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks. Following 32 weeks of treatment and a safety review, subjects received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. | 7 subjects were enrolled in cohort 2 and received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. | 8 subjects were enrolled in cohort 3 and received 40 mg/kg of PF06252616 IV every 4 weeks for 32 weeks. |
| Measure Participants | 4 | 7 | 8 |
| Mean (Standard Deviation) [score on a scale] |
9.75
(6.18)
|
7.57
(8.62)
|
2.43
(4.54)
|
Adverse Events
| Time Frame | Baseline through 124 weeks | |||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Adverse Event Reporting Description | Subject reported adverse events, as well as clinically relevant changes in laboratory screening, physical examination, suicide screening, cardiac screening are reported. | |||||||||||||||
| Arm/Group Title | Lead-in | Cohort 1 - Low Dose | Cohort 1 - High Dose | Cohort 1 Extension | Cohort 2 | Cohort 2 Extension | Cohort 3 | Cohort 3 Extension | ||||||||
| Arm/Group Description | All subjects were observed in a 16-week untreated lead-in-phase prior to allocation to dosing cohorts. | Subjects received an initial dose of 5mg/kg PF 06252616 IV every 4 weeks for 32 weeks. | Following the Low Dose period, subjects in Cohort 1 received an additional 32 weeks of treatment with 40 mg/kg PF 06252616 IV every 4 weeks. | Subjects enrolled in Cohort 1 were given the option to receive PF 06252616 at 40 mg/kg every 4 weeks for an additional 28 weeks. | Subjects received 20 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. | Subjects enrolled in Cohort 2 were given the option to receive PF 06252616 at 40 mg/kg every 4 weeks for an additional 40 weeks. | Subjects received 40 mg/kg of PF 06252616 IV every 4 weeks for 32 weeks. | Subjects enrolled in Cohort 3 were given the option to receive PF 06252616 at 40 mg/kg every 4 weeks for an additional 24 weeks. | ||||||||
All Cause Mortality |
||||||||||||||||
| Lead-in | Cohort 1 - Low Dose | Cohort 1 - High Dose | Cohort 1 Extension | Cohort 2 | Cohort 2 Extension | Cohort 3 | Cohort 3 Extension | |||||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/19 (0%) | 0/4 (0%) | 0/4 (0%) | 0/4 (0%) | 0/7 (0%) | 0/6 (0%) | 0/8 (0%) | 0/6 (0%) | ||||||||
Serious Adverse Events |
||||||||||||||||
| Lead-in | Cohort 1 - Low Dose | Cohort 1 - High Dose | Cohort 1 Extension | Cohort 2 | Cohort 2 Extension | Cohort 3 | Cohort 3 Extension | |||||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 1/19 (5.3%) | 0/4 (0%) | 0/4 (0%) | 0/4 (0%) | 0/7 (0%) | 1/6 (16.7%) | 0/8 (0%) | 0/6 (0%) | ||||||||
| Gastrointestinal disorders | ||||||||||||||||
| Intestinal obstruction | 1/19 (5.3%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Musculoskeletal and connective tissue disorders | ||||||||||||||||
| Stress fracture | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 1/6 (16.7%) | 4 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
Other (Not Including Serious) Adverse Events |
||||||||||||||||
| Lead-in | Cohort 1 - Low Dose | Cohort 1 - High Dose | Cohort 1 Extension | Cohort 2 | Cohort 2 Extension | Cohort 3 | Cohort 3 Extension | |||||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 9/19 (47.4%) | 4/4 (100%) | 4/4 (100%) | 4/4 (100%) | 6/7 (85.7%) | 4/6 (66.7%) | 7/8 (87.5%) | 6/6 (100%) | ||||||||
| Blood and lymphatic system disorders | ||||||||||||||||
| Anemia | 1/19 (5.3%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Leukopenia | 0/19 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Cardiac disorders | ||||||||||||||||
| Worsening cardiomyopathy | 1/19 (5.3%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 3/7 (42.9%) | 3 | 0/6 (0%) | 0 | 2/8 (25%) | 2 | 1/6 (16.7%) | 1 |
| Ear and labyrinth disorders | ||||||||||||||||
| Ear infection | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| Ear pain | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Hearing loss | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Eye disorders | ||||||||||||||||
| Blepharitis | 1/19 (5.3%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Vision abnormality - not otherwise specified | 0/19 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Eye twitching | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/7 (14.3%) | 1 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Gastrointestinal disorders | ||||||||||||||||
| Abdominal pain | 1/19 (5.3%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| Diarrhea | 1/19 (5.3%) | 1 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/7 (14.3%) | 1 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| Gastroesophageal reflux | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/7 (14.3%) | 2 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Nausea | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 2/8 (25%) | 2 | 0/6 (0%) | 0 |
| Hemoccult positive | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| New diagnosis - umbilical hernia | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| General disorders | ||||||||||||||||
| Decreased appetite | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| Fatigue | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 2/8 (25%) | 2 | 0/6 (0%) | 0 |
| Syncope | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 1/6 (16.7%) | 1 |
| Hypoglycemia | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| Hepatobiliary disorders | ||||||||||||||||
| New diagnosis - hepatic steatosis | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| Immune system disorders | ||||||||||||||||
| Environmental allergies | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/7 (14.3%) | 1 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Infections and infestations | ||||||||||||||||
| Upper respiratory symptoms | 1/19 (5.3%) | 1 | 2/4 (50%) | 2 | 1/4 (25%) | 2 | 3/4 (75%) | 3 | 3/7 (42.9%) | 3 | 0/6 (0%) | 0 | 2/8 (25%) | 5 | 1/6 (16.7%) | 1 |
| Flu-like illness | 1/19 (5.3%) | 1 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 1/6 (16.7%) | 1 | 0/8 (0%) | 0 | 1/6 (16.7%) | 1 |
| Infectious illness - not otherwise specified | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 1/6 (16.7%) | 1 |
| Cold sore | 0/19 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| Wound infection | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 1/6 (16.7%) | 1 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Dental infection | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/7 (14.3%) | 1 | 1/6 (16.7%) | 1 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Injury, poisoning and procedural complications | ||||||||||||||||
| Pain associated with muscle biopsy | 1/19 (5.3%) | 1 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 1/7 (14.3%) | 1 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| Numbness following biopsy | 1/19 (5.3%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Lower extremity edema following biopsy | 0/19 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Skin avulsion following biopsy | 0/19 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Intravenous catheter infiltration | 0/19 (0%) | 0 | 1/4 (25%) | 2 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Ecchymosis at IV site | 0/19 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Fall - not otherwise specified | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 1/4 (25%) | 2 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 1/6 (16.7%) | 1 | 0/8 (0%) | 0 | 1/6 (16.7%) | 1 |
| Musculoskeletal pain post fall | 1/19 (5.3%) | 1 | 0/4 (0%) | 0 | 2/4 (50%) | 2 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 2/6 (33.3%) | 3 | 1/8 (12.5%) | 1 | 3/6 (50%) | 3 |
| Fracture - pelvic | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 1/4 (25%) | 2 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Knee injury post fall | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 2/4 (50%) | 2 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Shoulder injury post fall | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 2/4 (50%) | 2 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Facial injury post fall | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 1/6 (16.7%) | 1 |
| Ankle injury post fall | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/7 (14.3%) | 1 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| Dental injury | 1/19 (5.3%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Investigations | ||||||||||||||||
| Ketones detected in urine | 0/19 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Red blood cells in urine | 0/19 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Elevated serum iron levels | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 2/4 (50%) | 2 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 2/6 (33.3%) | 2 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Low serum iron levels | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 2/7 (28.6%) | 2 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 1/6 (16.7%) | 1 |
| Elevated amylase | 0/19 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Elevated serum ferritin | 0/19 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Low serum ferritin | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/7 (14.3%) | 1 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Metabolism and nutrition disorders | ||||||||||||||||
| Weight gain | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| Weight loss | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 1/6 (16.7%) | 1 |
| Musculoskeletal and connective tissue disorders | ||||||||||||||||
| Shoulder pain - not otherwise specified | 1/19 (5.3%) | 1 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 1/4 (25%) | 1 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Knee pain - not otherwise specified | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Chest pain | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/7 (14.3%) | 1 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Neck pain - not otherwise specified | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 1/6 (16.7%) | 1 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Hip pain - not otherwise specified | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/7 (14.3%) | 1 | 1/6 (16.7%) | 1 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Nervous system disorders | ||||||||||||||||
| Headache | 3/19 (15.8%) | 3 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/7 (14.3%) | 1 | 0/6 (0%) | 0 | 2/8 (25%) | 2 | 0/6 (0%) | 0 |
| New diagnosis - carpal tunnel syndrome | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Psychiatric disorders | ||||||||||||||||
| Anxiety | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| New diagnosis - bipolar disorder | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| New diagnosis - personality disorder | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| Renal and urinary disorders | ||||||||||||||||
| Urinary tract infection | 2/19 (10.5%) | 2 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 2 | 0/6 (0%) | 0 |
| Urinary tract pain | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Nephrolithiasis | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Prostatic hypertrophy | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 1/6 (16.7%) | 1 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Hematuria | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 1/6 (16.7%) | 1 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| Reproductive system and breast disorders | ||||||||||||||||
| Vaginal yeast infection | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 2 | 0/6 (0%) | 0 |
| Skin and subcutaneous tissue disorders | ||||||||||||||||
| Dermatitis | 1/19 (5.3%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 2 | 0/6 (0%) | 0 |
| Rash - not otherwise specified | 0/19 (0%) | 0 | 1/4 (25%) | 1 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Acne | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Cyst - not otherwise specified | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/7 (14.3%) | 1 | 0/6 (0%) | 0 | 0/8 (0%) | 0 | 0/6 (0%) | 0 |
| Alopecia | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
| Pruritis | 0/19 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/7 (0%) | 0 | 0/6 (0%) | 0 | 1/8 (12.5%) | 1 | 0/6 (0%) | 0 |
Limitations/Caveats
[Not Specified]
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
| Name/Title | Dr. Kathryn R. Wagner |
|---|---|
| Organization | Hugo W. Moser Research Institute at Kennedy Krieger Inc. |
| Phone | 443-923-9525 |
| wagnerk@kennedykrieger.org |
Responsible Party:
Kathryn Wagner,
Director, Center for Genetic Muscle Disorders,
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
ClinicalTrials.gov Identifier:
NCT02841267
Other Study ID Numbers:
- WI203720
First Posted:
Jul 22, 2016
Last Update Posted:
Oct 19, 2020
Last Verified:
Sep 1, 2020