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

Sponsor
Kissei Pharmaceutical Co., Ltd. (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT04132050
Collaborator
(none)
24
1
2
36.3
0.7

Study Details

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.

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
24 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Double (Participant, Investigator)
Primary Purpose:
Treatment
Official Title:
A Phase III Study in Patients With Chronic Idiopathic Thrombocytopenic Purpura in R788
Actual Study Start Date :
Dec 24, 2019
Actual Primary Completion Date :
Dec 21, 2021
Anticipated Study Completion Date :
Jan 1, 2023

Arms and Interventions

Arm Intervention/Treatment
Experimental: 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).

Drug: R788
Oral administration

Placebo Comparator: 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).

Drug: R788
Oral administration

Drug: Placebo
Oral administration

Outcome Measures

Primary Outcome Measures

  1. 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

  1. 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

  2. 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

  3. 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

  4. 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

Eligibility Criteria

Criteria

Ages Eligible for Study:
20 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Research Site Multiple Locations Japan

Sponsors and Collaborators

  • Kissei Pharmaceutical Co., Ltd.

Investigators

  • Study Director: Yoshitaka Shimizu, Kissei Pharmaceutical Co., Ltd.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Kissei Pharmaceutical Co., Ltd.
ClinicalTrials.gov Identifier:
NCT04132050
Other Study ID Numbers:
  • R788-1301
First Posted:
Oct 18, 2019
Last Update Posted:
Mar 23, 2022
Last Verified:
Apr 1, 2021
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:

Study Results

No Results Posted as of Mar 23, 2022