Open Label Extension Study of Romiplostim (AMG 531) in Thrombocytopenic Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Sponsor
Amgen (Industry)
Overall Status
Completed
CT.gov ID
NCT00116688
Collaborator
(none)
313
1
65
Study Details
Study Description
Brief Summary
The purpose of this study is to determine the safety of romiplostim as a long-term treatment in thrombocytopenic subjects with ITP, to evaluate the long-term platelet response to romiplostim, and to evaluate changes in patient reported outcomes due to the use of romiplostim. Participants must have previously completed a romiplostim ITP study.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
Study Design
Study Type:
Interventional
Actual Enrollment
:
313 participants
Allocation:
Randomized
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
An Open Label Study Evaluating the Safety and Efficacy of Long-Term Dosing of AMG 531 in Thrombocytopenic Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Study Start Date
:
Aug 1, 2004
Actual Primary Completion Date
:
Jan 1, 2010
Actual Study Completion Date
:
Jan 1, 2010
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Romiplostim Romiplostim weekly subcutaneous dosing based on screening weight and platelet count. Starting dose of 1 µg/kg up to a maximum dose of 10 µg/kg. |
Biological: Romiplostim
Romiplostim is supplied in a 5 mL single use glass vial as a sterile, white, preservative-free, lyophilized powder.
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Number of Participants With Adverse Events [Duration of treatment plus 8 weeks (up to 285 weeks)]
Participants with one or more occurrences of one or more adverse events up to 8 weeks after the end of treatment. Participants with more than one event were only counted once.
Secondary Outcome Measures
- Number of Participants With a Platelet Response [Duration of treatment (up to 277 weeks)]
Platelet response was defined as having a platelet count of ≥ 50 x 10^9/L at any time on study, excluding platelet counts within 8 weeks after receiving any rescue medications.
- Number of Participants With a Reduction or Discontinuation of Concurrent ITP Therapies [Duration of treatment (up to 277 weeks)]
The number of participants with a reduction or discontinuation of concurrent immune (idiopathic) thrombocytopenic purpura (ITP) therapies (corticosteroids, danazol, azathioprine) during the study.
- Change From Baseline in ITP Patient Assessment Questionnaire [Baseline to Week 48]
The ITP Patient Assessment Questionnaire (ITP-PAQ) assesses ITP-specific health-related quality of life (HRQOL). This questionnaire assesses ITP specific health-related quality of life (HRQOL). The questionnaire consists of 44 items and has six domains: These domains assess the impact of ITP on Physical Health, Mental Health, Work, Social Activity, Women's Health and Overall QOL. The impact of ITP on Physical Health consists of four sub-scales, which evaluate ITP related Symptoms, Fatigue, Bother and Activity. The impact of ITP on Mental Health consists of two sub-scales, which evaluate Psychological distress and Fear in a population with ITP. Items are scored from 0-100 with higher scores indicating better HRQOL.
- Change From Baseline in Short Form 36 (SF-36) [Baseline to Week 48]
The SF-36 is a widely used generic health-related quality of life measure. It has 36 questions with 8 domains: Physical Functioning, Role Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role Emotional and Mental Health. Items are scored from 0 to 100 with higher scores indicating better health status.
- Change From Baseline in Euroqol-5D (EQ-5D) Index Score [Baseline to Week 48]
The EQ-5D is a patient-completed, multidimensional measure of health related quality of life. The instrument is applicable to a wide range of health conditions and treatments and results in a single index score and a visual analog scale (VAS) score. The EQ-5D descriptive health profile comprises five dimensions of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Each dimension comprises three levels (no problems, some/moderate problems, extreme problems). A unique EQ-5D health state is defined by combining one level from each of the five dimensions. EQ-5D index values range from -0.59 to 1.00. Higher EQ-5D Index scores represent better health status.
- Change From Baseline in Euroqol-5D (EQ-5D) Visual Analogue Scale (VAS) [Baseline to Week 48]
The EQ-5D is a patient-completed, multidimensional measure of health related quality of life. The EQ-5D VAS records the respondent's self-rated health status on a vertical graduated (0-100) visual analogue scale. Higher EQ-5D VAS scores represent better health status.
- Patient Global Assessment [Week 1 and Week 48]
The Patient Global Assessment is two questions which assess the overall health-related quality of life (HRQOL) and symptoms of the patient. Each item is answered on a 15-point Likert scale ranging from 'A very great deal worse' (1) to 'A very great deal better' (15). A higher score indicates that quality of life or symptoms have improved.
Eligibility Criteria
Criteria
Ages Eligible for Study:
1 Year
and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
-
Must have previously completed a romiplostim ITP study
-
Platelet count ≤ 50 x 10 ^9/L
-
Written informed consent
Exclusion Criteria:
-
Bone marrow stem cell disorder or new active malignancies diagnosed since enrollment in the previous romiplostim ITP study
-
Received any alkylating agents within 4 weeks before screening visit or anticipated use during the time of the proposed study
-
Currently enrolled in or has not yet completed at least 4 weeks since ending device or drug trial(s) (other than the previous romiplostim ITP study), or subject is receiving other investigational agent(s) other than romiplostim
-
Not using adequate contraceptive precautions
-
Not available for follow-up assessments
-
Has any kind of disorder that compromises the ability of the participant to give informed consent and does not have a legally-acceptable representative and/or is unable to comply with study procedures.
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Amgen
Investigators
- Study Director: MD, Amgen
Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
- Bussel JB, Kuter DJ, Pullarkat V, Lyons RM, Guo M, Nichol JL. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood. 2009 Mar 5;113(10):2161-71. doi: 10.1182/blood-2008-04-150078. Epub 2008 Nov 3. Erratum in: Blood. 2009 May 7;113(19):4822.
- Kuter DJ, Bussel JB, Newland A, Baker RI, Lyons RM, Wasser J, Viallard JF, Macik G, Rummel M, Nie K, Jun S. Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: safety and efficacy. Br J Haematol. 2013 May;161(3):411-23. doi: 10.1111/bjh.12260. Epub 2013 Feb 22.
- Terrell D, George J, Bussel J, Lyons R, Pullarkat V, Redner R, Nie, Selleslag D, Nie K, Woodard P.Home administration of romiplostim by patients with chronic immune thrombocytopenia (ITP).Journal-001500;
- Terrell D, George J, Bussel J, Lyons R, Pullarkat V, Redner R, Nie, Selleslag D, Nie K, Woodard P.Home administration of romiplostim by patients with chronic immune thrombocytopenia (ITP).Journal-001752;
Responsible Party:
Amgen
ClinicalTrials.gov Identifier:
NCT00116688
Other Study ID Numbers:
- 20030213
First Posted:
Jul 1, 2005
Last Update Posted:
Dec 18, 2013
Last Verified:
Nov 1, 2013
Keywords provided by Amgen
Additional relevant MeSH terms:
Study Results
Participant Flow
| Recruitment Details | Participants were enrolled from 2 August 2004 through 15 April 2009 |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | Romiplostim in Adults | Romiplostim in Pediatric Population |
|---|---|---|
| Arm/Group Description | Romiplostim administered to adult participants subcutaneously weekly at doses up to 30 µg/kg based on platelet counts. After Amendment 1 the maximum weekly dose was reduced to 15 µg/kg, and after Amendment 2 the maximum weekly dose was reduced to 10 µg/kg. However, participants enrolled prior to Amendment 2 who were receiving >10 µg/kg were permitted to remain on that higher dose, but could not increase their dose. In addition, if the participant's dose was decreased, it could not be increased to >10 µg/kg. | Romiplostim administered to pediatric participants subcutaneously weekly at doses up to 10 µg/kg based on platelet counts. |
| Period Title: Overall Study | ||
| STARTED | 292 | 21 |
| Received Study Medication | 291 | 20 |
| COMPLETED | 200 | 17 |
| NOT COMPLETED | 92 | 4 |
Baseline Characteristics
| Arm/Group Title | Romiplostim in Adults | Romiplostim in Pediatric Population | Total |
|---|---|---|---|
| Arm/Group Description | Romiplostim administered to adult participants subcutaneously weekly at doses up to 30 µg/kg based on platelet counts. After Amendment 1 the maximum weekly dose was reduced to 15 µg/kg, and after Amendment 2 the maximum weekly dose was reduced to 10 µg/kg. However, participants enrolled prior to Amendment 2 who were receiving >10 µg/kg were permitted to remain on that higher dose, but could not increase their dose. In addition, if the participant's dose was decreased, it could not be increased to >10 µg/kg. | Romiplostim administered to pediatric participants subcutaneously weekly at doses up to 10 µg/kg based on platelet counts. | Total of all reporting groups |
| Overall Participants | 292 | 21 | 313 |
| Age (Years) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [Years] |
54.2
(16.9)
|
10.2
(5.1)
|
51.3
(19.7)
|
| Sex: Female, Male (Count of Participants) | |||
| Female |
184
63%
|
6
28.6%
|
190
60.7%
|
| Male |
108
37%
|
15
71.4%
|
123
39.3%
|
| Race/Ethnicity, Customized (Number) [Number] | |||
| Black or African American |
13
4.5%
|
3
14.3%
|
16
5.1%
|
| White or Caucasian |
246
84.2%
|
13
61.9%
|
259
82.7%
|
| Hispanic or Latino |
21
7.2%
|
4
19%
|
25
8%
|
| Asian |
9
3.1%
|
0
0%
|
9
2.9%
|
| Japanese |
1
0.3%
|
0
0%
|
1
0.3%
|
| American Indian or Alaska Native |
1
0.3%
|
0
0%
|
1
0.3%
|
| Native Hawaiian or Other Pacific Islander |
1
0.3%
|
0
0%
|
1
0.3%
|
| Other |
0
0%
|
1
4.8%
|
1
0.3%
|
Outcome Measures
| Title | Number of Participants With Adverse Events |
|---|---|
| Description | Participants with one or more occurrences of one or more adverse events up to 8 weeks after the end of treatment. Participants with more than one event were only counted once. |
| Time Frame | Duration of treatment plus 8 weeks (up to 285 weeks) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety Analysis Set, composed of all participants who received at least one dose of romiplostim |
| Arm/Group Title | Romiplostim in Adults | Romiplostim in Pediatric Population |
|---|---|---|
| Arm/Group Description | Romiplostim administered to adult participants subcutaneously weekly at doses up to 30 µg/kg based on platelet counts. After Amendment 1 the maximum weekly dose was reduced to 15 µg/kg, and after Amendment 2 the maximum weekly dose was reduced to 10 µg/kg. However, participants enrolled prior to Amendment 2 who were receiving >10 µg/kg were permitted to remain on that higher dose, but could not increase their dose. In addition, if the participant's dose was decreased, it could not be increased to >10 µg/kg. | Romiplostim administered to pediatric participants subcutaneously weekly at doses up to 10 µg/kg based on platelet counts. |
| Measure Participants | 291 | 20 |
| Number [Participants] |
284
97.3%
|
19
90.5%
|
| Title | Number of Participants With a Platelet Response |
|---|---|
| Description | Platelet response was defined as having a platelet count of ≥ 50 x 10^9/L at any time on study, excluding platelet counts within 8 weeks after receiving any rescue medications. |
| Time Frame | Duration of treatment (up to 277 weeks) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Efficacy Analysis Set, composed of all enrolled participants who received at least one dose of romiplostim |
| Arm/Group Title | Romiplostim in Adults | Romiplostim in Pediatric Population |
|---|---|---|
| Arm/Group Description | Romiplostim administered to adult participants subcutaneously weekly at doses up to 30 µg/kg based on platelet counts. After Amendment 1 the maximum weekly dose was reduced to 15 µg/kg, and after Amendment 2 the maximum weekly dose was reduced to 10 µg/kg. However, participants enrolled prior to Amendment 2 who were receiving >10 µg/kg were permitted to remain on that higher dose, but could not increase their dose. In addition, if the participant's dose was decreased, it could not be increased to >10 µg/kg. | Romiplostim administered to pediatric participants subcutaneously weekly at doses up to 10 µg/kg based on platelet counts. |
| Measure Participants | 291 | 20 |
| Number [Participants] |
275
94.2%
|
20
95.2%
|
| Title | Number of Participants With a Reduction or Discontinuation of Concurrent ITP Therapies |
|---|---|
| Description | The number of participants with a reduction or discontinuation of concurrent immune (idiopathic) thrombocytopenic purpura (ITP) therapies (corticosteroids, danazol, azathioprine) during the study. |
| Time Frame | Duration of treatment (up to 277 weeks) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Subset of Efficacy Analysis Set, composed of all enrolled participants who received at least one dose of romiplostim and with baseline concurrent ITP therapy. |
| Arm/Group Title | Romiplostim in Adults | Romiplostim in Pediatric Population |
|---|---|---|
| Arm/Group Description | Romiplostim administered to adult participants subcutaneously weekly at doses up to 30 µg/kg based on platelet counts. After Amendment 1 the maximum weekly dose was reduced to 15 µg/kg, and after Amendment 2 the maximum weekly dose was reduced to 10 µg/kg. However, participants enrolled prior to Amendment 2 who were receiving >10 µg/kg were permitted to remain on that higher dose, but could not increase their dose. In addition, if the participant's dose was decreased, it could not be increased to >10 µg/kg. | Romiplostim administered to pediatric participants subcutaneously weekly at doses up to 10 µg/kg based on platelet counts. |
| Measure Participants | 37 | 2 |
| Number [Participants] |
30
10.3%
|
1
4.8%
|
| Title | Change From Baseline in ITP Patient Assessment Questionnaire |
|---|---|
| Description | The ITP Patient Assessment Questionnaire (ITP-PAQ) assesses ITP-specific health-related quality of life (HRQOL). This questionnaire assesses ITP specific health-related quality of life (HRQOL). The questionnaire consists of 44 items and has six domains: These domains assess the impact of ITP on Physical Health, Mental Health, Work, Social Activity, Women's Health and Overall QOL. The impact of ITP on Physical Health consists of four sub-scales, which evaluate ITP related Symptoms, Fatigue, Bother and Activity. The impact of ITP on Mental Health consists of two sub-scales, which evaluate Psychological distress and Fear in a population with ITP. Items are scored from 0-100 with higher scores indicating better HRQOL. |
| Time Frame | Baseline to Week 48 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Full analysis set with available data. Patient reported outcomes were only analyzed in adult participants. |
| Arm/Group Title | Romiplostim in Adults |
|---|---|
| Arm/Group Description | Romiplostim administered to adult participants subcutaneously weekly at doses up to 30 µg/kg based on platelet counts. After Amendment 1 the maximum weekly dose was reduced to 15 µg/kg, and after Amendment 2 the maximum weekly dose was reduced to 10 µg/kg. However, participants enrolled prior to Amendment 2 who were receiving >10 µg/kg were permitted to remain on that higher dose, but could not increase their dose. In addition, if the participant's dose was decreased, it could not be increased to >10 µg/kg. |
| Measure Participants | 292 |
| Physical Health Symptoms (N=209) |
4.20
(13.15)
|
| Physical Health Fatigue (N=208) |
3.99
(16.00)
|
| Physical Health Bother (N=204) |
6.43
(17.39)
|
| Physical Health Activity (N=208) |
5.23
(21.08)
|
| Emotional Health Psychological (N=208) |
4.01
(15.81)
|
| Emotional Health Fear (N=209) |
3.48
(12.59)
|
| Overall Quality of Life (N=210) |
8.32
(19.65)
|
| Social Quality of Life (N=209) |
3.89
(13.41)
|
| Women's Reproductive Health (N=71) |
4.51
(16.35)
|
| Work Quality of Life (N=75) |
2.78
(14.54)
|
| Title | Change From Baseline in Short Form 36 (SF-36) |
|---|---|
| Description | The SF-36 is a widely used generic health-related quality of life measure. It has 36 questions with 8 domains: Physical Functioning, Role Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role Emotional and Mental Health. Items are scored from 0 to 100 with higher scores indicating better health status. |
| Time Frame | Baseline to Week 48 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Full analysis set with available data. Patient reported outcomes were only analyzed in adult participants. |
| Arm/Group Title | Romiplostim in Adults |
|---|---|
| Arm/Group Description | Romiplostim administered to adult participants subcutaneously weekly at doses up to 30 µg/kg based on platelet counts. After Amendment 1 the maximum weekly dose was reduced to 15 µg/kg, and after Amendment 2 the maximum weekly dose was reduced to 10 µg/kg. However, participants enrolled prior to Amendment 2 who were receiving >10 µg/kg were permitted to remain on that higher dose, but could not increase their dose. In addition, if the participant's dose was decreased, it could not be increased to >10 µg/kg. |
| Measure Participants | 292 |
| Physical Functioning (N=206) |
1.38
(6.34)
|
| Role-Physical (N=208) |
2.03
(8.89)
|
| Bodily Pain (N=206) |
0.94
(7.99)
|
| General Health Perception (N=208) |
1.31
(7.03)
|
| Vitality (N=208) |
1.68
(7.47)
|
| Social Functioning (N=208) |
0.52
(8.21)
|
| Role-Emotional (N=205) |
1.75
(12.22)
|
| Mental Health Index (N=208) |
0.91
(7.45)
|
| Physical Component Summary (N=200) |
1.49
(6.51)
|
| Mental Component Summary (N=200) |
1.06
(8.80)
|
| Title | Change From Baseline in Euroqol-5D (EQ-5D) Index Score |
|---|---|
| Description | The EQ-5D is a patient-completed, multidimensional measure of health related quality of life. The instrument is applicable to a wide range of health conditions and treatments and results in a single index score and a visual analog scale (VAS) score. The EQ-5D descriptive health profile comprises five dimensions of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Each dimension comprises three levels (no problems, some/moderate problems, extreme problems). A unique EQ-5D health state is defined by combining one level from each of the five dimensions. EQ-5D index values range from -0.59 to 1.00. Higher EQ-5D Index scores represent better health status. |
| Time Frame | Baseline to Week 48 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Full analysis set with available data. Patient reported outcomes were only analyzed in adult participants. |
| Arm/Group Title | Romiplostim in Adults |
|---|---|
| Arm/Group Description | Romiplostim administered to adult participants subcutaneously weekly at doses up to 30 µg/kg based on platelet counts. After Amendment 1 the maximum weekly dose was reduced to 15 µg/kg, and after Amendment 2 the maximum weekly dose was reduced to 10 µg/kg. However, participants enrolled prior to Amendment 2 who were receiving >10 µg/kg were permitted to remain on that higher dose, but could not increase their dose. In addition, if the participant's dose was decreased, it could not be increased to >10 µg/kg. |
| Measure Participants | 154 |
| Mean (Standard Deviation) [scores on a scale] |
0.03
(0.16)
|
| Title | Change From Baseline in Euroqol-5D (EQ-5D) Visual Analogue Scale (VAS) |
|---|---|
| Description | The EQ-5D is a patient-completed, multidimensional measure of health related quality of life. The EQ-5D VAS records the respondent's self-rated health status on a vertical graduated (0-100) visual analogue scale. Higher EQ-5D VAS scores represent better health status. |
| Time Frame | Baseline to Week 48 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Full analysis set with available data. Patient reported outcomes were only analyzed in adult participants. |
| Arm/Group Title | Romiplostim in Adults |
|---|---|
| Arm/Group Description | Romiplostim administered to adult participants subcutaneously weekly at doses up to 30 µg/kg based on platelet counts. After Amendment 1 the maximum weekly dose was reduced to 15 µg/kg, and after Amendment 2 the maximum weekly dose was reduced to 10 µg/kg. However, participants enrolled prior to Amendment 2 who were receiving >10 µg/kg were permitted to remain on that higher dose, but could not increase their dose. In addition, if the participant's dose was decreased, it could not be increased to >10 µg/kg. |
| Measure Participants | 150 |
| Mean (Standard Deviation) [scores on a scale] |
6.05
(14.85)
|
| Title | Patient Global Assessment |
|---|---|
| Description | The Patient Global Assessment is two questions which assess the overall health-related quality of life (HRQOL) and symptoms of the patient. Each item is answered on a 15-point Likert scale ranging from 'A very great deal worse' (1) to 'A very great deal better' (15). A higher score indicates that quality of life or symptoms have improved. |
| Time Frame | Week 1 and Week 48 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Full analysis set with available data. Patient reported outcomes were only analyzed in adult participants. |
| Arm/Group Title | Romiplostim in Adults |
|---|---|
| Arm/Group Description | Romiplostim administered to adult participants subcutaneously weekly at doses up to 30 µg/kg based on platelet counts. After Amendment 1 the maximum weekly dose was reduced to 15 µg/kg, and after Amendment 2 the maximum weekly dose was reduced to 10 µg/kg. However, participants enrolled prior to Amendment 2 who were receiving >10 µg/kg were permitted to remain on that higher dose, but could not increase their dose. In addition, if the participant's dose was decreased, it could not be increased to >10 µg/kg. |
| Measure Participants | 292 |
| Week 1 (N=271) |
7.61
(2.02)
|
| Week 48 (N=216) |
8.18
(1.78)
|
Adverse Events
| Time Frame | For adult participants the average duration was 110 weeks; for pediatric participants the average duration is 82 weeks | |||
|---|---|---|---|---|
| Adverse Event Reporting Description | The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events. One adult patient and one pediatric patient did not receive the investigational product and were not included in the adverse event table. | |||
| Arm/Group Title | Romiplostim in Adults | Romiplostim in Pediatric Population | ||
| Arm/Group Description | Romiplostim administered to adult participants subcutaneously weekly at doses up to 30 µg/kg based on platelet counts. After Amendment 1 the maximum weekly dose was reduced to 15 µg/kg, and after Amendment 2 the maximum weekly dose was reduced to 10 µg/kg. However, participants enrolled prior to Amendment 2 who were receiving >10 µg/kg were permitted to remain on that higher dose, but could not increase their dose. In addition, if the participant's dose was decreased, it could not be increased to >10 µg/kg. | Romiplostim administered to pediatric participants subcutaneously weekly at doses up to 10 µg/kg based on platelet counts. | ||
All Cause Mortality |
||||
| Romiplostim in Adults | Romiplostim in Pediatric Population | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | / (NaN) | / (NaN) | ||
Serious Adverse Events |
||||
| Romiplostim in Adults | Romiplostim in Pediatric Population | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 117/291 (40.2%) | 2/20 (10%) | ||
| Blood and lymphatic system disorders | ||||
| Anaemia | 3/291 (1%) | 0/20 (0%) | ||
| Bicytopenia | 1/291 (0.3%) | 0/20 (0%) | ||
| Bone marrow disorder | 4/291 (1.4%) | 0/20 (0%) | ||
| Bone marrow reticulin fibrosis | 1/291 (0.3%) | 0/20 (0%) | ||
| Evans syndrome | 1/291 (0.3%) | 0/20 (0%) | ||
| Haemolytic anaemia | 1/291 (0.3%) | 0/20 (0%) | ||
| Idiopathic thrombocytopenic purpura | 7/291 (2.4%) | 1/20 (5%) | ||
| Leukocytosis | 1/291 (0.3%) | 0/20 (0%) | ||
| Neutropenia | 1/291 (0.3%) | 0/20 (0%) | ||
| Thrombocytopenia | 23/291 (7.9%) | 0/20 (0%) | ||
| Cardiac disorders | ||||
| Acute myocardial infarction | 3/291 (1%) | 0/20 (0%) | ||
| Angina unstable | 2/291 (0.7%) | 0/20 (0%) | ||
| Atrial fibrillation | 3/291 (1%) | 0/20 (0%) | ||
| Cardiac arrest | 1/291 (0.3%) | 0/20 (0%) | ||
| Cardiac failure | 4/291 (1.4%) | 0/20 (0%) | ||
| Cardiac failure congestive | 5/291 (1.7%) | 0/20 (0%) | ||
| Cardiac tamponade | 1/291 (0.3%) | 0/20 (0%) | ||
| Coronary artery disease | 2/291 (0.7%) | 0/20 (0%) | ||
| Myocardial infarction | 5/291 (1.7%) | 0/20 (0%) | ||
| Pericardial haemorrhage | 1/291 (0.3%) | 0/20 (0%) | ||
| Trifascicular block | 1/291 (0.3%) | 0/20 (0%) | ||
| Congenital, familial and genetic disorders | ||||
| Atrial septal defect | 1/291 (0.3%) | 0/20 (0%) | ||
| Ear and labyrinth disorders | ||||
| Vertigo | 2/291 (0.7%) | 0/20 (0%) | ||
| Vestibular disorder | 1/291 (0.3%) | 0/20 (0%) | ||
| Eye disorders | ||||
| Blindness | 1/291 (0.3%) | 0/20 (0%) | ||
| Conjunctival haemorrhage | 1/291 (0.3%) | 0/20 (0%) | ||
| Papilloedema | 1/291 (0.3%) | 0/20 (0%) | ||
| Gastrointestinal disorders | ||||
| Abdominal distension | 1/291 (0.3%) | 0/20 (0%) | ||
| Abdominal pain | 2/291 (0.7%) | 0/20 (0%) | ||
| Abdominal pain lower | 1/291 (0.3%) | 0/20 (0%) | ||
| Abdominal pain upper | 1/291 (0.3%) | 0/20 (0%) | ||
| Anal fistula | 1/291 (0.3%) | 0/20 (0%) | ||
| Ascites | 1/291 (0.3%) | 0/20 (0%) | ||
| Colitis | 2/291 (0.7%) | 0/20 (0%) | ||
| Colitis ischaemic | 1/291 (0.3%) | 0/20 (0%) | ||
| Diarrhoea | 1/291 (0.3%) | 0/20 (0%) | ||
| Dyspepsia | 1/291 (0.3%) | 0/20 (0%) | ||
| Femoral hernia | 1/291 (0.3%) | 0/20 (0%) | ||
| Gastrointestinal haemorrhage | 4/291 (1.4%) | 0/20 (0%) | ||
| Gingival bleeding | 2/291 (0.7%) | 0/20 (0%) | ||
| Haematemesis | 1/291 (0.3%) | 0/20 (0%) | ||
| Irritable bowel syndrome | 1/291 (0.3%) | 0/20 (0%) | ||
| Mouth cyst | 1/291 (0.3%) | 0/20 (0%) | ||
| Mouth haemorrhage | 1/291 (0.3%) | 0/20 (0%) | ||
| Mouth ulceration | 1/291 (0.3%) | 0/20 (0%) | ||
| Nausea | 1/291 (0.3%) | 0/20 (0%) | ||
| Periodontitis | 1/291 (0.3%) | 0/20 (0%) | ||
| Rectal haemorrhage | 2/291 (0.7%) | 0/20 (0%) | ||
| Small intestinal obstruction | 1/291 (0.3%) | 0/20 (0%) | ||
| Tooth impacted | 1/291 (0.3%) | 0/20 (0%) | ||
| Tooth loss | 1/291 (0.3%) | 0/20 (0%) | ||
| Upper gastrointestinal haemorrhage | 1/291 (0.3%) | 0/20 (0%) | ||
| Vomiting | 1/291 (0.3%) | 0/20 (0%) | ||
| General disorders | ||||
| Adverse drug reaction | 1/291 (0.3%) | 0/20 (0%) | ||
| Asthenia | 1/291 (0.3%) | 0/20 (0%) | ||
| Chest pain | 3/291 (1%) | 0/20 (0%) | ||
| Death | 1/291 (0.3%) | 0/20 (0%) | ||
| Fatigue | 1/291 (0.3%) | 0/20 (0%) | ||
| Generalised oedema | 1/291 (0.3%) | 0/20 (0%) | ||
| Hernia | 1/291 (0.3%) | 0/20 (0%) | ||
| Hernia obstructive | 2/291 (0.7%) | 0/20 (0%) | ||
| Hernia pain | 1/291 (0.3%) | 0/20 (0%) | ||
| Hyperpyrexia | 1/291 (0.3%) | 0/20 (0%) | ||
| Mechanical complication of implant | 1/291 (0.3%) | 0/20 (0%) | ||
| Oedema peripheral | 1/291 (0.3%) | 0/20 (0%) | ||
| Pyrexia | 4/291 (1.4%) | 0/20 (0%) | ||
| Hepatobiliary disorders | ||||
| Biliary colic | 1/291 (0.3%) | 0/20 (0%) | ||
| Cholecystitis | 2/291 (0.7%) | 0/20 (0%) | ||
| Cholecystitis acute | 1/291 (0.3%) | 0/20 (0%) | ||
| Cholelithiasis | 3/291 (1%) | 0/20 (0%) | ||
| Hepatic failure | 2/291 (0.7%) | 0/20 (0%) | ||
| Hepatic steatosis | 1/291 (0.3%) | 0/20 (0%) | ||
| Hepatitis | 1/291 (0.3%) | 0/20 (0%) | ||
| Portal vein thrombosis | 1/291 (0.3%) | 0/20 (0%) | ||
| Infections and infestations | ||||
| Anal abscess | 1/291 (0.3%) | 0/20 (0%) | ||
| Appendicitis | 2/291 (0.7%) | 0/20 (0%) | ||
| Bacteraemia | 1/291 (0.3%) | 0/20 (0%) | ||
| Bronchitis | 3/291 (1%) | 0/20 (0%) | ||
| Candidiasis | 1/291 (0.3%) | 0/20 (0%) | ||
| Catheter bacteraemia | 1/291 (0.3%) | 0/20 (0%) | ||
| Catheter related infection | 2/291 (0.7%) | 0/20 (0%) | ||
| Cellulitis | 3/291 (1%) | 0/20 (0%) | ||
| Device related infection | 1/291 (0.3%) | 0/20 (0%) | ||
| Epiglottitis | 1/291 (0.3%) | 0/20 (0%) | ||
| Gastroenteritis | 1/291 (0.3%) | 0/20 (0%) | ||
| Haematoma infection | 1/291 (0.3%) | 0/20 (0%) | ||
| Influenza | 0/291 (0%) | 1/20 (5%) | ||
| Klebsiella sepsis | 1/291 (0.3%) | 0/20 (0%) | ||
| Localised infection | 1/291 (0.3%) | 0/20 (0%) | ||
| Meningitis listeria | 1/291 (0.3%) | 0/20 (0%) | ||
| Nasopharyngitis | 1/291 (0.3%) | 0/20 (0%) | ||
| Parotitis | 1/291 (0.3%) | 0/20 (0%) | ||
| Pharyngitis streptococcal | 0/291 (0%) | 1/20 (5%) | ||
| Pneumococcal sepsis | 1/291 (0.3%) | 0/20 (0%) | ||
| Pneumonia | 8/291 (2.7%) | 0/20 (0%) | ||
| Pneumonia streptococcal | 1/291 (0.3%) | 0/20 (0%) | ||
| Post procedural cellulitis | 1/291 (0.3%) | 0/20 (0%) | ||
| Progressive multifocal leukoencephalopathy | 1/291 (0.3%) | 0/20 (0%) | ||
| Sepsis | 1/291 (0.3%) | 0/20 (0%) | ||
| Thrombophlebitis septic | 1/291 (0.3%) | 0/20 (0%) | ||
| Tooth abscess | 1/291 (0.3%) | 0/20 (0%) | ||
| Urosepsis | 2/291 (0.7%) | 0/20 (0%) | ||
| Injury, poisoning and procedural complications | ||||
| Arteriovenous fistula site complication | 1/291 (0.3%) | 0/20 (0%) | ||
| Contusion | 1/291 (0.3%) | 0/20 (0%) | ||
| Fractured sacrum | 1/291 (0.3%) | 0/20 (0%) | ||
| Head injury | 1/291 (0.3%) | 0/20 (0%) | ||
| Hip fracture | 2/291 (0.7%) | 0/20 (0%) | ||
| Humerus fracture | 1/291 (0.3%) | 0/20 (0%) | ||
| Incisional hernia | 1/291 (0.3%) | 0/20 (0%) | ||
| Medical device complication | 1/291 (0.3%) | 0/20 (0%) | ||
| Meniscus lesion | 1/291 (0.3%) | 0/20 (0%) | ||
| Pelvic fracture | 1/291 (0.3%) | 0/20 (0%) | ||
| Subdural haemorrhage | 1/291 (0.3%) | 0/20 (0%) | ||
| Upper limb fracture | 1/291 (0.3%) | 0/20 (0%) | ||
| Wound | 1/291 (0.3%) | 0/20 (0%) | ||
| Investigations | ||||
| Megakaryocytes increased | 1/291 (0.3%) | 0/20 (0%) | ||
| Platelet count decreased | 3/291 (1%) | 0/20 (0%) | ||
| Platelet count increased | 2/291 (0.7%) | 0/20 (0%) | ||
| Metabolism and nutrition disorders | ||||
| Decreased appetite | 1/291 (0.3%) | 0/20 (0%) | ||
| Dehydration | 4/291 (1.4%) | 0/20 (0%) | ||
| Hyperkalaemia | 2/291 (0.7%) | 0/20 (0%) | ||
| Hypokalaemia | 1/291 (0.3%) | 0/20 (0%) | ||
| Musculoskeletal and connective tissue disorders | ||||
| Arthritis | 1/291 (0.3%) | 0/20 (0%) | ||
| Intervertebral disc protrusion | 1/291 (0.3%) | 0/20 (0%) | ||
| Osteoarthritis | 3/291 (1%) | 0/20 (0%) | ||
| Osteonecrosis | 1/291 (0.3%) | 0/20 (0%) | ||
| Pain in extremity | 1/291 (0.3%) | 0/20 (0%) | ||
| Rhabdomyolysis | 1/291 (0.3%) | 0/20 (0%) | ||
| Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ||||
| Breast cancer | 1/291 (0.3%) | 0/20 (0%) | ||
| Chronic lymphocytic leukaemia | 1/291 (0.3%) | 0/20 (0%) | ||
| Colon cancer recurrent | 1/291 (0.3%) | 0/20 (0%) | ||
| Hepatic neoplasm malignant | 2/291 (0.7%) | 0/20 (0%) | ||
| Lung neoplasm malignant | 1/291 (0.3%) | 0/20 (0%) | ||
| Lymphoma | 1/291 (0.3%) | 0/20 (0%) | ||
| Metastases to central nervous system | 1/291 (0.3%) | 0/20 (0%) | ||
| Multiple myeloma | 1/291 (0.3%) | 0/20 (0%) | ||
| Myelofibrosis | 1/291 (0.3%) | 0/20 (0%) | ||
| Neoplasm of orbit | 1/291 (0.3%) | 0/20 (0%) | ||
| Renal cell carcinoma | 1/291 (0.3%) | 0/20 (0%) | ||
| Transitional cell carcinoma | 1/291 (0.3%) | 0/20 (0%) | ||
| Nervous system disorders | ||||
| Cerebrovascular accident | 2/291 (0.7%) | 0/20 (0%) | ||
| Complex regional pain syndrome | 1/291 (0.3%) | 0/20 (0%) | ||
| Convulsion | 2/291 (0.7%) | 0/20 (0%) | ||
| Headache | 1/291 (0.3%) | 0/20 (0%) | ||
| Intracranial aneurysm | 1/291 (0.3%) | 0/20 (0%) | ||
| Loss of consciousness | 1/291 (0.3%) | 0/20 (0%) | ||
| Migraine | 1/291 (0.3%) | 0/20 (0%) | ||
| Multiple sclerosis relapse | 1/291 (0.3%) | 0/20 (0%) | ||
| Presyncope | 1/291 (0.3%) | 0/20 (0%) | ||
| Syncope | 2/291 (0.7%) | 0/20 (0%) | ||
| Transient ischaemic attack | 2/291 (0.7%) | 0/20 (0%) | ||
| Transverse sinus thrombosis | 1/291 (0.3%) | 0/20 (0%) | ||
| Pregnancy, puerperium and perinatal conditions | ||||
| Abortion spontaneous | 1/291 (0.3%) | 0/20 (0%) | ||
| Psychiatric disorders | ||||
| Agitation | 1/291 (0.3%) | 0/20 (0%) | ||
| Anxiety | 2/291 (0.7%) | 0/20 (0%) | ||
| Confusional state | 1/291 (0.3%) | 0/20 (0%) | ||
| Mental status changes | 2/291 (0.7%) | 0/20 (0%) | ||
| Suicidal ideation | 1/291 (0.3%) | 0/20 (0%) | ||
| Renal and urinary disorders | ||||
| Renal failure | 3/291 (1%) | 0/20 (0%) | ||
| Renal failure acute | 2/291 (0.7%) | 0/20 (0%) | ||
| Renal failure chronic | 1/291 (0.3%) | 0/20 (0%) | ||
| Urinary bladder polyp | 1/291 (0.3%) | 0/20 (0%) | ||
| Urinary retention | 1/291 (0.3%) | 0/20 (0%) | ||
| Reproductive system and breast disorders | ||||
| Menorrhagia | 1/291 (0.3%) | 0/20 (0%) | ||
| Metrorrhagia | 1/291 (0.3%) | 0/20 (0%) | ||
| Ovarian cyst | 1/291 (0.3%) | 0/20 (0%) | ||
| Vaginal haemorrhage | 2/291 (0.7%) | 0/20 (0%) | ||
| Respiratory, thoracic and mediastinal disorders | ||||
| Acute pulmonary oedema | 1/291 (0.3%) | 0/20 (0%) | ||
| Asthma | 1/291 (0.3%) | 0/20 (0%) | ||
| Cough | 1/291 (0.3%) | 0/20 (0%) | ||
| Dyspnoea | 4/291 (1.4%) | 0/20 (0%) | ||
| Epistaxis | 3/291 (1%) | 1/20 (5%) | ||
| Haemoptysis | 1/291 (0.3%) | 0/20 (0%) | ||
| Pleuritic pain | 1/291 (0.3%) | 0/20 (0%) | ||
| Pulmonary embolism | 1/291 (0.3%) | 0/20 (0%) | ||
| Pulmonary haemorrhage | 1/291 (0.3%) | 0/20 (0%) | ||
| Respiratory arrest | 1/291 (0.3%) | 0/20 (0%) | ||
| Respiratory failure | 2/291 (0.7%) | 0/20 (0%) | ||
| Skin and subcutaneous tissue disorders | ||||
| Blister | 1/291 (0.3%) | 0/20 (0%) | ||
| Ecchymosis | 1/291 (0.3%) | 0/20 (0%) | ||
| Petechiae | 2/291 (0.7%) | 0/20 (0%) | ||
| Psoriasis | 1/291 (0.3%) | 0/20 (0%) | ||
| Purpura | 1/291 (0.3%) | 0/20 (0%) | ||
| Rash | 2/291 (0.7%) | 0/20 (0%) | ||
| Systemic lupus erythematosus rash | 1/291 (0.3%) | 0/20 (0%) | ||
| Urticaria | 1/291 (0.3%) | 0/20 (0%) | ||
| Surgical and medical procedures | ||||
| Cholecystectomy | 1/291 (0.3%) | 0/20 (0%) | ||
| Elective surgery | 1/291 (0.3%) | 0/20 (0%) | ||
| Knee arthroplasty | 2/291 (0.7%) | 0/20 (0%) | ||
| Plastic surgery | 1/291 (0.3%) | 0/20 (0%) | ||
| Skin cosmetic procedure | 1/291 (0.3%) | 0/20 (0%) | ||
| Stent placement | 1/291 (0.3%) | 0/20 (0%) | ||
| Vascular disorders | ||||
| Aortic aneurysm | 1/291 (0.3%) | 0/20 (0%) | ||
| Deep vein thrombosis | 2/291 (0.7%) | 0/20 (0%) | ||
| Femoral arterial stenosis | 1/291 (0.3%) | 0/20 (0%) | ||
| Haematoma | 1/291 (0.3%) | 0/20 (0%) | ||
| Haemorrhage | 1/291 (0.3%) | 0/20 (0%) | ||
| Phlebitis | 1/291 (0.3%) | 0/20 (0%) | ||
| Subgaleal haematoma | 1/291 (0.3%) | 0/20 (0%) | ||
| Thrombosis | 1/291 (0.3%) | 0/20 (0%) | ||
Other (Not Including Serious) Adverse Events |
||||
| Romiplostim in Adults | Romiplostim in Pediatric Population | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 270/291 (92.8%) | 18/20 (90%) | ||
| Blood and lymphatic system disorders | ||||
| Anaemia | 17/291 (5.8%) | 1/20 (5%) | ||
| Idiopathic thrombocytopenic purpura | 29/291 (10%) | 2/20 (10%) | ||
| Gastrointestinal disorders | ||||
| Abdominal discomfort | 17/291 (5.8%) | 1/20 (5%) | ||
| Abdominal pain | 33/291 (11.3%) | 3/20 (15%) | ||
| Abdominal pain upper | 21/291 (7.2%) | 4/20 (20%) | ||
| Constipation | 26/291 (8.9%) | 1/20 (5%) | ||
| Diarrhoea | 72/291 (24.7%) | 2/20 (10%) | ||
| Dyspepsia | 19/291 (6.5%) | 0/20 (0%) | ||
| Gingival bleeding | 43/291 (14.8%) | 5/20 (25%) | ||
| Mouth haemorrhage | 21/291 (7.2%) | 4/20 (20%) | ||
| Mouth ulceration | 4/291 (1.4%) | 2/20 (10%) | ||
| Nausea | 69/291 (23.7%) | 4/20 (20%) | ||
| Toothache | 17/291 (5.8%) | 1/20 (5%) | ||
| Vomiting | 46/291 (15.8%) | 7/20 (35%) | ||
| General disorders | ||||
| Asthenia | 22/291 (7.6%) | 0/20 (0%) | ||
| Chest pain | 16/291 (5.5%) | 1/20 (5%) | ||
| Chills | 12/291 (4.1%) | 2/20 (10%) | ||
| Fatigue | 93/291 (32%) | 7/20 (35%) | ||
| Injection site haematoma | 15/291 (5.2%) | 0/20 (0%) | ||
| Oedema peripheral | 41/291 (14.1%) | 0/20 (0%) | ||
| Pain | 32/291 (11%) | 4/20 (20%) | ||
| Pyrexia | 36/291 (12.4%) | 9/20 (45%) | ||
| Immune system disorders | ||||
| Seasonal allergy | 16/291 (5.5%) | 1/20 (5%) | ||
| Infections and infestations | ||||
| Bronchitis | 22/291 (7.6%) | 1/20 (5%) | ||
| Ear infection | 11/291 (3.8%) | 2/20 (10%) | ||
| Gastroenteritis | 13/291 (4.5%) | 2/20 (10%) | ||
| Influenza | 23/291 (7.9%) | 0/20 (0%) | ||
| Nasopharyngitis | 100/291 (34.4%) | 5/20 (25%) | ||
| Sinusitis | 38/291 (13.1%) | 1/20 (5%) | ||
| Upper respiratory tract infection | 76/291 (26.1%) | 10/20 (50%) | ||
| Urinary tract infection | 36/291 (12.4%) | 0/20 (0%) | ||
| Viral infection | 5/291 (1.7%) | 2/20 (10%) | ||
| Viral upper respiratory tract infection | 7/291 (2.4%) | 3/20 (15%) | ||
| Injury, poisoning and procedural complications | ||||
| Animal bite | 3/291 (1%) | 2/20 (10%) | ||
| Arthropod bite | 6/291 (2.1%) | 2/20 (10%) | ||
| Contusion | 88/291 (30.2%) | 8/20 (40%) | ||
| Excoriation | 10/291 (3.4%) | 3/20 (15%) | ||
| Fall | 19/291 (6.5%) | 0/20 (0%) | ||
| Joint sprain | 11/291 (3.8%) | 2/20 (10%) | ||
| Procedural pain | 17/291 (5.8%) | 2/20 (10%) | ||
| Skin laceration | 18/291 (6.2%) | 0/20 (0%) | ||
| Musculoskeletal and connective tissue disorders | ||||
| Arthralgia | 69/291 (23.7%) | 5/20 (25%) | ||
| Back pain | 54/291 (18.6%) | 1/20 (5%) | ||
| Joint swelling | 16/291 (5.5%) | 0/20 (0%) | ||
| Muscle spasms | 28/291 (9.6%) | 1/20 (5%) | ||
| Musculoskeletal pain | 29/291 (10%) | 1/20 (5%) | ||
| Myalgia | 35/291 (12%) | 3/20 (15%) | ||
| Pain in extremity | 55/291 (18.9%) | 2/20 (10%) | ||
| Nervous system disorders | ||||
| Dizziness | 51/291 (17.5%) | 2/20 (10%) | ||
| Headache | 109/291 (37.5%) | 9/20 (45%) | ||
| Migraine | 15/291 (5.2%) | 1/20 (5%) | ||
| Paraesthesia | 28/291 (9.6%) | 0/20 (0%) | ||
| Psychiatric disorders | ||||
| Anxiety | 21/291 (7.2%) | 0/20 (0%) | ||
| Depression | 19/291 (6.5%) | 0/20 (0%) | ||
| Insomnia | 41/291 (14.1%) | 0/20 (0%) | ||
| Renal and urinary disorders | ||||
| Dysuria | 15/291 (5.2%) | 0/20 (0%) | ||
| Respiratory, thoracic and mediastinal disorders | ||||
| Cough | 70/291 (24.1%) | 9/20 (45%) | ||
| Dyspnoea | 22/291 (7.6%) | 2/20 (10%) | ||
| Epistaxis | 73/291 (25.1%) | 5/20 (25%) | ||
| Nasal congestion | 30/291 (10.3%) | 6/20 (30%) | ||
| Oropharyngeal blistering | 20/291 (6.9%) | 0/20 (0%) | ||
| Oropharyngeal pain | 50/291 (17.2%) | 6/20 (30%) | ||
| Rhinorrhoea | 26/291 (8.9%) | 5/20 (25%) | ||
| Skin and subcutaneous tissue disorders | ||||
| Blood blister | 19/291 (6.5%) | 0/20 (0%) | ||
| Ecchymosis | 25/291 (8.6%) | 1/20 (5%) | ||
| Petechiae | 53/291 (18.2%) | 9/20 (45%) | ||
| Pruritus | 23/291 (7.9%) | 0/20 (0%) | ||
| Rash | 44/291 (15.1%) | 5/20 (25%) | ||
| Scab | 0/291 (0%) | 2/20 (10%) | ||
| Skin lesion | 18/291 (6.2%) | 1/20 (5%) | ||
| Vascular disorders | ||||
| Haematoma | 37/291 (12.7%) | 1/20 (5%) | ||
| Hypertension | 17/291 (5.8%) | 0/20 (0%) | ||
Limitations/Caveats
[Not Specified]
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
The Clinical Trial Agreement generally does not restrict an investigator's discussion of trial results aftercompletion. The Agreement permits Amgen a limited period of time to review material discussing trial results (typically up to 45 days and possible extension). Amgen may remove confidential information, but authors have final control and approval of publication content. For multicenter studies, the investigator agrees not to publish any results before the first multi-center publication.
Results Point of Contact
| Name/Title | Study Director |
|---|---|
| Organization | Amgen Inc. |
| Phone | 866-572-6436 |
Responsible Party:
Amgen
ClinicalTrials.gov Identifier:
NCT00116688
Other Study ID Numbers:
- 20030213
First Posted:
Jul 1, 2005
Last Update Posted:
Dec 18, 2013
Last Verified:
Nov 1, 2013