Open Label Extension Study of Romiplostim (AMG 531) in Thrombocytopenic Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
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.
Idiopathic Thrombocytopenic Purpura
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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)|
- Number of Participants With Adverse Events [ Time Frame: Duration of treatment plus 8 weeks (up to 285 weeks) ] [ Designated as safety issue: Yes ]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.
- Number of Participants With a Platelet Response [ Time Frame: Duration of treatment (up to 277 weeks) ] [ Designated as safety issue: No ]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.
- Numberof Participants With a Reduction or Discontinuation of Concurrent ITP Therapies [ Time Frame: Duration of treatment (up to 277 weeks) ] [ Designated as safety issue: No ]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 [ Time Frame: Baseline to Week 48 ] [ Designated as safety issue: No ]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) [ Time Frame: Baseline to Week 48 ] [ Designated as safety issue: No ]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 [ Time Frame: Baseline to Week 48 ] [ Designated as safety issue: No ]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) [ Time Frame: Baseline to Week 48 ] [ Designated as safety issue: No ]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 [ Time Frame: Week 1 and Week 48 ] [ Designated as safety issue: No ]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.
|Study Start Date:||August 2004|
|Study Completion Date:||January 2010|
|Primary Completion Date:||January 2010 (Final data collection date for primary outcome measure)|
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.
Romiplostim is supplied in a 5 mL single use glass vial as a sterile, white, preservative-free, lyophilized powder.