Back to resultsOpen Label Extension Study of Romiplostim (AMG 531) in Thrombocytopenic Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Primary Purpose
Thrombocytopenia, Idiopathic Thrombocytopenic Purpura
Status
Completed
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
Romiplostim
Sponsored by
About this trial
This is an interventional treatment trial for Thrombocytopenia focused on measuring Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenic
Eligibility Criteria
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.
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Romiplostim
Arm Description
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.
Outcomes
Primary Outcome Measures
Number of Participants With Adverse Events
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
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
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
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)
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
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)
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
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00116688
Brief Title
Open Label Extension Study of Romiplostim (AMG 531) in Thrombocytopenic Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
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 Type
Interventional
2. Study Status
Record Verification Date
November 2013
Overall Recruitment Status
Completed
Study Start Date
August 2004 (undefined)
Primary Completion Date
January 2010 (Actual)
Study Completion Date
January 2010 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Amgen
4. Oversight
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Thrombocytopenia, Idiopathic Thrombocytopenic Purpura
Keywords
Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenic
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
313 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Romiplostim
Arm Type
Experimental
Arm Description
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.
Intervention Type
Biological
Intervention Name(s)
Romiplostim
Other Intervention Name(s)
AMG 531, Nplate
Intervention Description
Romiplostim is supplied in a 5 mL single use glass vial as a sterile, white, preservative-free, lyophilized powder.
Primary Outcome Measure Information:
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)
Secondary Outcome Measure Information:
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)
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)
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
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
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
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
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
MD
Organizational Affiliation
Amgen
Official's Role
Study Director
12. IPD Sharing Statement
Citations:
PubMed Identifier
18981291
Citation
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.
Results Reference
background
PubMed Identifier
23432528
Citation
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.
Results Reference
background
Citation
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;
Results Reference
background
Citation
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;
Results Reference
background
PubMed Identifier
19671919
Citation
Kuter DJ, Mufti GJ, Bain BJ, Hasserjian RP, Davis W, Rutstein M. Evaluation of bone marrow reticulin formation in chronic immune thrombocytopenia patients treated with romiplostim. Blood. 2009 Oct 29;114(18):3748-56. doi: 10.1182/blood-2009-05-224766. Epub 2009 Aug 11.
Results Reference
derived
Links:
URL
http://www.amgentrials.com
Description
AmgenTrials clinical trials website
Learn more about this trial
Open Label Extension Study of Romiplostim (AMG 531) in Thrombocytopenic Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
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