A Clinical Study in Patients With Chronic Idiopathic Thrombocytopenic Purpura in R788

Study Overview
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Brief Summary
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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.

Official Title
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A Phase III Study in Patients With Chronic Idiopathic Thrombocytopenic Purpura in R788

Conditions
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Idiopathic Thrombocytopenic Purpura

Intervention / Treatment
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* Drug: R788
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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) Exclusion Criteria: Patients with thrombocytopenia associated with other disease Patients with autoimmune hemolytic anemia Patients with poorly controlled hypertension Patients with a history or active coagulopathy

Ages Eligible for Study
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Minimum Age: 20 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: R788<br>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). | Drug: R788<br>* Oral administration<br>|
| Placebo Comparator: Placebo<br>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). | Drug: R788<br>* Oral administration<br>Drug: Placebo<br>* Oral administration<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of patients with stable platelet response | 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 | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of patients with overall response | Percentage of patients with a platelet count ≥50000/μL on at least 1 of 6 visits from Week 2 to Week 12 | 12 weeks | 
| Duration of maintained platelet count | Duration of maintained platelet count since first achievement of a platelet count ≥50000/μL after administration of the study drug | 52 weeks | 
| Percentage of patients with a platelet count ≥50000/μL | Percentage of patients with a platelet count ≥50000/μL at the specified evaluation time point | 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 | Percentage of patients with a platelet count increase ≥20000/μL above baseline and ≥30000/μL at the specified evaluation time point | Every 2 weeks up to Week 24, every 4 weeks up to Week 52 and every 8 weeks up to 3 years |