A Study of Rilzabrutinib in Adult Patients With Immune Thrombocytopenia (ITP)

January 30, 2024 updated by: Principia Biopharma, a Sanofi Company

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

This is a 2 part (Part A and B) adaptive, open-label, dose-finding study of PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options, with a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. The dose-finding portion of the study has been completed. Part B treatment dose is 400 mg twice daily.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

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

Enrollment (Actual)

81

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • Australian Capital Territory
      • Canberra, Australian Capital Territory, Australia, 2605
        • Investigational Site Number : 105
    • New South Wales
      • Sydney, New South Wales, Australia, 2139
        • Investigational Site Number : 104
    • Queensland
      • Woolloongabba, Queensland, Australia, 4102
        • Investigational Site Number : 102
    • Victoria
      • Clayton, Victoria, Australia, 3168
        • Investigational Site Number : 101
      • Parkville, Victoria, Australia, 3050
        • Investigational Site Number : 106
    • Western Australia
      • Perth, Western Australia, Australia, 6005
        • Investigational Site Number : 103
  • Bulgaria
      • Pleven, Bulgaria, 5800
        • Investigational Site Number : 213
      • Sofia, Bulgaria, 1431
        • Investigational Site Number : 214
      • Varna, Bulgaria, 9010
        • Investigational Site Number : 211
  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5B 1W8
        • Investigational Site Number : 1161
    • Quebec
      • Montreal, Quebec, Canada, H3A 1A1
        • Investigational Site Number : 1162
  • Czechia
      • Brno, Czechia, 62500
        • Investigational Site Number : 431
      • Hradec Kralove, Czechia, 50005
        • Investigational Site Number : 433
      • Ostrava - Poruba, Czechia, 70852
        • Investigational Site Number : 434
      • Praha 2, Czechia, 12808
        • Investigational Site Number : 432
  • Netherlands
      • Rotterdam, Netherlands, 3015 GD
        • Investigational Site Number : 727
      • s-Gravenhage, Netherlands, 2545 CH
        • Investigational Site Number : 728
  • Norway
      • Bergen, Norway, N-5021
        • Investigational Site Number : 542
      • Gralum, Norway, 1714
        • Investigational Site Number : 541
  • United Kingdom
    • Leicestershire
      • Leicester, Leicestershire, United Kingdom, LE1 5WW
        • Investigational Site Number : 981
    • London, City Of
      • London, London, City Of, United Kingdom, E1 2ES
        • Investigational Site Number : 983
      • London, London, City Of, United Kingdom, W12 0HS
        • Investigational Site Number : 980
  • United States
    • Illinois
      • Peoria, Illinois, United States, 61614
        • Bleeding and Clotting Disorders Institute Site Number : 1087
    • Maryland
      • Bethesda, Maryland, United States, 20817-1915
        • RCCA MC LLC Site Number : 1091
    • Massachusetts
      • Boston, Massachusetts, United States, 02114-2603
        • Massachusetts General Hospital Cancer Center Site Number : 1092
      • Boston, Massachusetts, United States, 02215
        • Beth Israel Deaconess Medical Center Site Number : 1099
    • Michigan
      • Midland, Michigan, United States, 48670
        • Mid Michigan Medical Center Site Number : 1086
    • New York
      • New York, New York, United States, 10021
        • New York Presbyterian Hospital/Weill Cornell Medical Center Site Number : 1097
    • North Carolina
      • Greenville, North Carolina, United States, 27834
        • Pitt County Memorial Hospital Site Number : 1095
    • Washington
      • Seattle, Washington, United States, 98109-4405
        • Seattle Cancer Care Alliance Site Number : 1098

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Rilzabrutinib (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: Rilzabrutinib
BTK inhibitor
Other Names:
  • PRN1008

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
24 weeks of treatment, long term extension and 4 weeks of follow up post last dose]
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
24 weeks
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
24 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Principia Biopharma, a Sanofi Company

Investigators

  • Study Director: Olga Bandman, MD, Principia Biopharma

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 22, 2018

Primary Completion (Actual)

January 31, 2023

Study Completion (Estimated)

December 31, 2025

Study Registration Dates

First Submitted

December 22, 2017

First Submitted That Met QC Criteria

January 3, 2018

First Posted (Actual)

January 10, 2018

Study Record Updates

Last Update Posted (Actual)

February 1, 2024

Last Update Submitted That Met QC Criteria

January 30, 2024

Last Verified

January 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • DFI17124
  • PRN1008-010 (Other Identifier: Principia Biopharma)
  • U1111-1260-4044 (Registry Identifier: ICTRP)
  • 2023-509397-39 (Registry Identifier: CTIS)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD 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

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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