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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04132050
Recruitment Status : Active, not recruiting
First Posted : October 18, 2019
Last Update Posted : April 11, 2023
Sponsor:
Information provided by (Responsible Party):
Kissei Pharmaceutical Co., Ltd.

Study Description
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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
Idiopathic Thrombocytopenic Purpura Drug: R788 Drug: Placebo Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated 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 : December 24, 2019
Actual Primary Completion Date : December 21, 2021
Estimated Study Completion Date : September 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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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
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Primary Outcome Measures :
  1. Percentage of patients with stable platelet response [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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
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Ages Eligible for Study:   20 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04132050


Locations
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Japan
Research Site
Multiple Locations, Japan
Sponsors and Collaborators
Kissei Pharmaceutical Co., Ltd.
Investigators
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Study Director: Yoshitaka Shimizu Kissei Pharmaceutical Co., Ltd.
More Information
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Responsible Party: Kissei Pharmaceutical Co., Ltd.
ClinicalTrials.gov Identifier: NCT04132050    
Other Study ID Numbers: R788-1301
First Posted: October 18, 2019    Key Record Dates
Last Update Posted: April 11, 2023
Last Verified: April 2023

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Purpura
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhage
Pathologic Processes
 Skin Manifestations
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders
Immune System Diseases
Hemorrhagic Disorders
Autoimmune Diseases