RAISE: Randomized Placebo-Controlled Idiopathic Thrombocytopenic Purpura (ITP) Study With Eltrombopag
- Conditions
- Purpura, Thrombocytopaenic, Idiopathic
- Interventions
- Drug: Placebo
- Registration Number
- NCT00370331
- Lead Sponsor
- GlaxoSmithKline
- Brief Summary
The rationale for this Phase III study is to evaluate the 6 month safety and efficacy of eltrombopag in the treatment of previously treated subjects with chronic ITP. The starting dose of eltrombopag, 50 mg, once daily was selected based upon the observed efficacy, safety and pharmacokinetics in a dose-finding Study (TRA100773). This Phase III study is a randomized, double-blind, placebo-controlled, Phase III study, to evaluate efficacy, safety and tolerability of eltrombopag, initially administered as 50 mg oral tablets once daily for six months in adult subjects with previously treated chronic ITP. Subjects will be randomized 2:1, eltrombopag to placebo, and will be stratified based upon splenectomy status, use of ITP medication at baseline and baseline platelet count less than or equal to 15,000/µL. Subjects will receive study medication for 6 months, during which the dose of study medication may be adjusted based upon individual platelet counts. In addition, subjects may taper off concomitant ITP medications and may receive any rescue treatments as dictated by local standard of care. After discontinuation of study medication, subjects will complete follow-up visits at weeks 1, 2, 4 and months 3 and 6.
- Detailed Description
A randomized, double-blind, placebo-controlled phase III study, to evaluate the efficacy, safety and tolerability of eltrombopag olamine (SB-497115-GR), a thrombopoietin receptor agonist, administered for 6 months as oral tablets once daily in adult subjects with previously treated chronic idiopathic thrombocytopenic purpura (ITP).
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 197
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description placebo plus standard of care Placebo Subjects will initiate treatment with 50 mg eltrombopag or matching placebo once daily. Based upon the subjects platelet count at each visit, the dose of eltrombopag may be adjusted either up or down. Treatment arm plus standard of care eltrombopag Subjects will initiate treatment with 50 mg eltrombopag or matching placebo once daily. Based upon the subjects platelet count at each visit, the dose of eltrombopag may be adjusted either up or down.
- Primary Outcome Measures
Name Time Method Percentage of Responders Baseline; each on-therapy treatment day; Weeks 10, 14, 18, 22, and 26; and Weeks 1, 2, and 4 post-treatment The percentage of evaluable participants who achieved a platelet response (defined as a platelet count between 50,000 and 400,000 microliter) at each nominal on-therapy day and 4 weeks post-treatment
- Secondary Outcome Measures
Name Time Method Summary of Median Platelet Counts Baseline; Day 8 through Week 26 on-treatment; and 1, 2, 4 week follow-up visits Platelet counts were measured by blood draw.
Percentage of Participants Initiating Rescue Treatment On-therapy Anytime from Day 1 to Week 26 Percentage of participants initiating new ITP medication, an increased dose of concomitant ITP medication from baseline, platelet transfusion, or splenectomy.
Maximum and Total Weeks of Platelet Response Day 1 through Week 26 on-treatment Response is defined as a platelet count between 50,000 and 400,000 platelets per microliter.
Percentage of Participants With a Reduction in Use of Baseline ITP Medication From Day 1 through Week 26 on-treatment Percentage of participants who experienced a reduction in their baseline concomitant ITP medication use
WHO Bleeding Scale Baseline, all nominal visits on-therapy defined as Day 8, Day 15, Day 22, Day 29, Day 36, Day 43, Week 10, Week 14, Week 18, Week 22, Week 26, and 1, 2 and 4 week follow-up visits Summary of World Health Organization (WHO) bleeding scores at each nominal visit. WHO Grades 1-4 = any bleeding; WHO Grades 2-4 = clinically significant bleeding
HR-QoL Instrument and Domain Scores From the SF-36v2 Questionnaire at Baseline, Week 6, Week 14, and Week 26 or Early Discontinuation From Study Treatment Baseline, Week 6, Week 14, and Week 26/Early Withdrawal Health-related quality of life (HR-QoL) patient reported outcomes from the short form-36v2 (SF-36v2) questionnaire. Scores could range from 0 (worst possible) to 100 (best possible).
HR-QoL Instrument and Domain Scores From the FACIT-F Questionnaire at Baseline, Week 6, Week 14, and Week 26 or Early Discontinuation From Study Treatment Baseline, Week 6, Week 14, and Week 26/Early Withdrawal Health-related quality of life (HR-QoL) patient reported outcomes from the functional assessment of chronic illness therapy fatigue (FACIT-F) questionnaire. Scores could range from 0 (worst possible) to 52 (best possible).
HR-QoL Instrument and Domain Scores From the MEI-SF Questionnaire at Baseline, Week 6, Week 14, and Week 26 or Early Discontinuation From Study Treatment Baseline, Week 6, Week 14, and Week 26/Early Withdrawal Health-related quality of life (HR-QoL) patient reported outcomes from the motivation and energy inventory-short form (MEI-SF) questionnaire. Scores could range from 0 (worst possible) to 72 (best possible).
HR-QoL Instrument and Domain Scores for the FACT-Th Questionnaire at Baseline, Week 6, Week 14, and Week 26 or Early Discontinuation From Study Treatment Baseline, Week 6, Week 14, and Week 26/Early Withdrawal Health-related quality of life (HR-QoL) patient reported outcomes from the functional assessment of cancer therapy thrombocytopenia (FACT-Th) questionnaire (six selected items). Scores could range from 0 (worst possible) to 24 (best possible).
Trial Locations
- Locations (1)
GSK Investigational Site
🇻🇳Ho Chi Minh, Vietnam