A Study of Rilzabrutinib in Adult Patients With Immune Thrombocytopenia (ITP)
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| ClinicalTrials.gov Identifier: NCT03395210 |
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Recruitment Status :
Active, not recruiting
First Posted : January 10, 2018
Last Update Posted : March 17, 2023
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Immune Thrombocytopenia Immune Thrombocytopenic Purpura | Drug: PRN1008 | Phase 1 Phase 2 |
This is a 2 part (Part A and B) adaptive, open-label, dose-finding study of PRN1008 in approximately 60 patients in Part A and approximately 25 patients in Part B.
Part A enrolls patients with ITP who are refractory or relapsed with no available and approved therapeutic options. Eligible patients have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. The active treatment period is 24 weeks and the post-treatment follow-up period is 4 weeks. In the dose-finding part of the study, each patient enrolled in the study is allowed to up-titrate their dose after 28 days of PRN1008 therapy, if they do not experience a platelet response or a dose-limiting toxicity (DLT) at the last dose level. Patients who respond to PRN1008 per protocol may enter a long term-extension.
Part B of the study will include approximately 25 patients with ITP who have relapsed or have an insufficient response to prior therapies. Eligible patients will have a platelet count <30,000/µL on two occasions no less than 7 days apart, within 15 days before treatment begins and a platelet count of ≤35,000/µL on Study Day 1 (SD1). The study consists of a 28-day screening period, 24-week active treatment period, and a long-term extension. After the last dose of PRN1008 there will be a 4-week safety follow-up period.
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 81 participants |
| Allocation: | N/A |
| Intervention Model: | Sequential Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | An Adaptive, Open-Label, Dose-Finding, Phase 1/2 Study Investigating the Safety, Pharmacokinetics, and Clinical Activity of PRN1008, an Oral BTK Inhibitor, in Patients With Relapsed Immune Thrombocytopenia |
| Actual Study Start Date : | March 22, 2018 |
| Actual Primary Completion Date : | January 30, 2023 |
| Estimated Study Completion Date : | October 30, 2025 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: PRN1008 Daily
Part A approximately 60 patients: Up to 24 weeks open-label treatment with PRN1008 400mg BID; safety and dose evaluation. Patients who respond to PRN1008 per protocol may enter a long-term extension. Part B approximately 25 patients: Up to 24 weeks open-label treatment with PRN1008 400mg BID; safety and dose evaluation. Patients who respond to PRN1008 per protocol may enter a long-term extension |
Drug: PRN1008
BTK inhibitor |
- Part A and B: Incidence of Treatment Emergent Adverse Events (Safety Outcome Measure) [ Time Frame: 24 weeks of treatment, long term extension and 4 weeks of follow up post last dose] ]The incidence, severity and relationship of TEAEs during the treatment period and at the 4-week follow-up from last dose received.
- Part A: Consecutive Increased Platelet Counts (Efficacy Outcome Measure) [ Time Frame: 24 weeks ]Proportion of subjects able to achieve two or more consecutive platelet counts of ≥ 50,000/μL AND an increase of platelet count of ≥20,000/μL from baseline, without the use of rescue medication
- Part B: Sustained Increase in Platelet Counts (Efficacy Outcome Measure) [ Time Frame: 24 weeks ]Proportion of patients able to achieve platelet counts ≥50,000/μL on at least 8 out of the last 12 weeks of the 24-week treatment period without the use of rescue medication
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years to 80 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Male or female patients, aged 18 to 80 years old
- Immune-related ITP (both primary and secondary)
Exclusion Criteria:
- Pregnant or lactating women
- Current drug or alcohol abuse
- History of solid organ transplant
- Positive screening for HIV, hepatitis B, or hepatitis C
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): NCT03395210
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| Study Director: | Olga Bandman, MD | Principia Biopharma |
| Responsible Party: | Principia Biopharma, a Sanofi Company |
| ClinicalTrials.gov Identifier: | NCT03395210 |
| Other Study ID Numbers: |
DFI17124 PRN1008-010 ( Other Identifier: Principia Biopharma ) |
| First Posted: | January 10, 2018 Key Record Dates |
| Last Update Posted: | March 17, 2023 |
| Last Verified: | March 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Yes |
| Plan Description: | Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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ITP |
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Thrombocytopenia Purpura Purpura, Thrombocytopenic, Idiopathic Purpura, Thrombocytopenic Blood Platelet Disorders Hematologic Diseases Blood Coagulation Disorders |
Hemorrhage Pathologic Processes Skin Manifestations Thrombotic Microangiopathies Hemorrhagic Disorders Autoimmune Diseases Immune System Diseases |