A Clinical Study in Patients With Chronic Idiopathic Thrombocytopenic Purpura in R788
- Registration Number
- NCT04132050
- Lead Sponsor
- Kissei Pharmaceutical Co., Ltd.
- Brief Summary
The purpose of this study is to investigate the efficacy, safety and pharmacokinetics of R788 compared with placebo, and to investigate the safety and efficacy of long term dosing of R788 in patients with chronic idiopathic thrombocytopenic purpura.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 34
- Japanese patients
- Patients diagnosed with idiopathic thrombocytopenic purpura at least 6 months before acquisition of consent
- Patients with a platelet count averages <30000/μL during screening period. Each platelet count should not exceed 35000/μL.
- Patients who have used and failed or who were intolerant at least 1 typical regimen for the treatment of ITP before informed consent (with or without splenectomy)
- Patients with thrombocytopenia associated with other disease
- Patients with autoimmune hemolytic anemia
- Patients with poorly controlled hypertension
- Patients with a history or active coagulopathy
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo Patients are administered Placebo for 24 weeks (double-blind period), followed by R788 for up to 28 weeks (open-label period). Patients who have completed open-label period and meet the criteria are eligible to continue R788 treatment over a 3 year period (extension - period). R788 R788 Patients are administered R788 for 24 weeks (double-blind period), followed by R788 for up to 52 weeks (open-label period). Patients who have completed open-label period and meet the criteria are eligible to continue R788 treatment over a 3 year period (extension - period). Placebo R788 Patients are administered Placebo for 24 weeks (double-blind period), followed by R788 for up to 28 weeks (open-label period). Patients who have completed open-label period and meet the criteria are eligible to continue R788 treatment over a 3 year period (extension - period).
- Primary Outcome Measures
Name Time Method Percentage of patients with stable platelet response 24 weeks Percentage of patients with a stable platelet response by Week 24 defined as a platelet count of ≥ 50000/μL on at least 4 of 6 visits between Week 14 to Week 24
- Secondary Outcome Measures
Name Time Method Percentage of patients with overall response 12 weeks Percentage of patients with a platelet count ≥50000/μL on at least 1 of 6 visits from Week 2 to Week 12
Percentage of patients with a platelet count ≥50000/μL Every 2 weeks up to Week 24, every 4 weeks up to Week 52 and every 8 weeks up to 3 years Percentage of patients with a platelet count ≥50000/μL at the specified evaluation time point
Duration of maintained platelet count 52 weeks Duration of maintained platelet count since first achievement of a platelet count ≥50000/μL after administration of the study drug
Percentage of patients with a platelet count increase ≥20000/μL above baseline and ≥30000/μL Every 2 weeks up to Week 24, every 4 weeks up to Week 52 and every 8 weeks up to 3 years Percentage of patients with a platelet count increase ≥20000/μL above baseline and ≥30000/μL at the specified evaluation time point
Trial Locations
- Locations (1)
Research Site
🇯🇵Multiple Locations, Japan