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

NCT ID: NCT04132050

Last Updated: 2025-08-11

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 34 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-12-24
• Study Completion Date: 2023-09-25

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.

Conditions

Idiopathic Thrombocytopenic Purpura

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

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).

Group Type: EXPERIMENTAL

R788

Intervention Type: DRUG

Oral administration

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).

Group Type: PLACEBO_COMPARATOR

R788

Intervention Type: DRUG

Oral administration

Placebo

Intervention Type: DRUG

Oral administration

Interventions

R788

Oral administration

Intervention Type: DRUG

Placebo

Oral administration

Intervention Type: DRUG

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

• Minimum Eligible Age: 20 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Kissei Pharmaceutical Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Yoshitaka Shimizu

Role: STUDY_DIRECTOR

Kissei Pharmaceutical Co., Ltd.

Locations

Research Site

Multiple Locations, , Japan

Countries

Japan

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

R788-1301

Identifier Type: -

Identifier Source: org_study_id