A Clinical Study in Patients With Chronic Idiopathic Thrombocytopenic Purpura in R788
Primary Purpose
Idiopathic Thrombocytopenic Purpura
Status
Active
Phase
Phase 3
Locations
Japan
Study Type
Interventional
Intervention
R788
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Idiopathic Thrombocytopenic Purpura
Eligibility Criteria
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
Sites / Locations
- Research Site
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
R788
Placebo
Arm Description
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).
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).
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
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
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
Percentage of patients with a platelet count ≥50000/μL
Percentage of patients with a platelet count ≥50000/μL at the specified evaluation time point
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
Full Information
NCT ID
NCT04132050
First Posted
October 15, 2019
Last Updated
April 9, 2023
Sponsor
Kissei Pharmaceutical Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT04132050
Brief Title
A Clinical Study in Patients With Chronic Idiopathic Thrombocytopenic Purpura in R788
Official Title
A Phase III Study in Patients With Chronic Idiopathic Thrombocytopenic Purpura in R788
Study Type
Interventional
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
December 24, 2019 (Actual)
Primary Completion Date
December 21, 2021 (Actual)
Study Completion Date
September 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Kissei Pharmaceutical Co., Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Idiopathic Thrombocytopenic Purpura
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
24 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
R788
Arm Type
Experimental
Arm Description
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).
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
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).
Intervention Type
Drug
Intervention Name(s)
R788
Intervention Description
Oral administration
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Oral administration
Primary Outcome Measure Information:
Title
Percentage of patients with stable platelet response
Description
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
Time Frame
24 weeks
Secondary Outcome Measure Information:
Title
Percentage of patients with overall response
Description
Percentage of patients with a platelet count ≥50000/μL on at least 1 of 6 visits from Week 2 to Week 12
Time Frame
12 weeks
Title
Duration of maintained platelet count
Description
Duration of maintained platelet count since first achievement of a platelet count ≥50000/μL after administration of the study drug
Time Frame
52 weeks
Title
Percentage of patients with a platelet count ≥50000/μL
Description
Percentage of patients with a platelet count ≥50000/μL at the specified evaluation time point
Time Frame
Every 2 weeks up to Week 24, every 4 weeks up to Week 52 and every 8 weeks up to 3 years
Title
Percentage of patients with a platelet count increase ≥20000/μL above baseline and ≥30000/μL
Description
Percentage of patients with a platelet count increase ≥20000/μL above baseline and ≥30000/μL at the specified evaluation time point
Time Frame
Every 2 weeks up to Week 24, every 4 weeks up to Week 52 and every 8 weeks up to 3 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
20 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Yoshitaka Shimizu
Organizational Affiliation
Kissei Pharmaceutical Co., Ltd.
Official's Role
Study Director
Facility Information:
Facility Name
Research Site
City
Multiple Locations
Country
Japan
12. IPD Sharing Statement
Learn more about this trial
A Clinical Study in Patients With Chronic Idiopathic Thrombocytopenic Purpura in R788
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