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

NCT ID: NCT03395210

Last Updated: 2025-11-14

Study Results

Results available

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Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

86 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-03-22

Study Completion Date

2025-12-18

Brief Summary

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This was 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 was completed. Part B treatment dose was 400 mg twice daily.

Detailed Description

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This was 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 enrolled patients with ITP who were refractory or relapsed with no available and approved therapeutic options. Eligible patients had 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 was 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 was allowed to up-titrate their dose after 28 days of PRN1008 therapy, if they did not experience a platelet response or a dose-limiting toxicity (DLT) at the last dose level. Patients who responded to PRN1008 per protocol may enter a long term-extension.

Part B of the study included approximately 25 patients with ITP who had relapsed or had an insufficient response to prior therapies. Eligible patients had a platelet count \<30,000/µL on two occasions no less than 7 days apart, within 15 days before treatment began and a platelet count of ≤35,000/µL on Study Day 1 (SD1). The study consisted of a 28-day screening period, 24-week active treatment period, and a long-term extension. After the last dose of PRN1008 there was a 4-week safety follow-up period.

Conditions

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Immune Thrombocytopenia

Keywords

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ITP

Study Design

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Allocation Method

NA

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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

Group Type EXPERIMENTAL

Rilzabrutinib

Intervention Type DRUG

BTK inhibitor

Interventions

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Rilzabrutinib

BTK inhibitor

Intervention Type DRUG

Other Intervention Names

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PRN1008

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Principia Biopharma, a Sanofi Company

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Olga Bandman, MD

Role: STUDY_DIRECTOR

Principia Biopharma

Locations

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Bleeding and Clotting Disorders Institute- Site Number : 1087

Peoria, Illinois, United States

Site Status

RCCA MC LLC- Site Number : 1091

Bethesda, Maryland, United States

Site Status

Massachusetts General Hospital Cancer Center- Site Number : 1092

Boston, Massachusetts, United States

Site Status

Beth Israel Deaconess Medical Center- Site Number : 1099

Boston, Massachusetts, United States

Site Status

Mid Michigan Medical Center- Site Number : 1086

Midland, Michigan, United States

Site Status

New York Presbyterian Hospital/Weill Cornell Medical Center- Site Number : 1097

New York, New York, United States

Site Status

Pitt County Memorial Hospital- Site Number : 1095

Greenville, North Carolina, United States

Site Status

Seattle Cancer Care Alliance Site Number : 1098

Seattle, Washington, United States

Site Status

Investigational Site Number : 105

Canberra, Australian Capital Territory, Australia

Site Status

Investigational Site Number : 104

Sydney, New South Wales, Australia

Site Status

Investigational Site Number : 102

Woolloongabba, Queensland, Australia

Site Status

Investigational Site Number : 101

Clayton, Victoria, Australia

Site Status

Investigational Site Number : 106

Parkville, Victoria, Australia

Site Status

Investigational Site Number : 103

Perth, Western Australia, Australia

Site Status

Investigational Site Number : 213

Pleven, , Bulgaria

Site Status

Investigational Site Number : 214

Sofia, , Bulgaria

Site Status

Investigational Site Number : 211

Varna, , Bulgaria

Site Status

Investigational Site Number : 1161

Toronto, Ontario, Canada

Site Status

Investigational Site Number : 1162

Montreal, Quebec, Canada

Site Status

Investigational Site Number : 431

Brno, , Czechia

Site Status

Investigational Site Number : 433

Hradec Králové, , Czechia

Site Status

Investigational Site Number : 434

Ostrava - Poruba, , Czechia

Site Status

Investigational Site Number : 432

Prague, , Czechia

Site Status

Investigational Site Number : 727

Rotterdam, , Netherlands

Site Status

Investigational Site Number : 728

The Hague, , Netherlands

Site Status

Investigational Site Number : 542

Bergen, , Norway

Site Status

Investigational Site Number : 541

Grålum, , Norway

Site Status

Investigational Site Number : 981

Leicester, Leicestershire, United Kingdom

Site Status

Investigational Site Number : 983

London, London, City of, United Kingdom

Site Status

Investigational Site Number : 980

London, London, City of, United Kingdom

Site Status

Investigational Site Number : 984

Birmingham, , United Kingdom

Site Status

Countries

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United States Australia Bulgaria Canada Czechia Netherlands Norway United Kingdom

References

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Cooper N, Jansen AJG, Bird R, Mayer J, Sholzberg M, Tarantino MD, Garg M, Ypma PF, McDonald V, Percy C, Kostal M, Goncalves I, Bogdanov LH, Gernsheimer TB, Diab R, Yao M, Daak A, Kuter DJ. Efficacy and Safety Results With Rilzabrutinib, an Oral Bruton Tyrosine Kinase Inhibitor, in Patients With Immune Thrombocytopenia: Phase 2 Part B Study. Am J Hematol. 2025 Mar;100(3):439-449. doi: 10.1002/ajh.27539. Epub 2025 Jan 22.

Reference Type DERIVED
PMID: 39844469 (View on PubMed)

Kuter DJ, Mayer J, Efraim M, Bogdanov LH, Baker R, Kaplan Z, Garg M, Trneny M, Choi PY, Jansen AJG, McDonald V, Bird R, Gumulec J, Kostal M, Gernsheimer T, Ghanima W, Daak A, Cooper N. Long-term treatment with rilzabrutinib in patients with immune thrombocytopenia. Blood Adv. 2024 Apr 9;8(7):1715-1724. doi: 10.1182/bloodadvances.2023012044.

Reference Type DERIVED
PMID: 38386978 (View on PubMed)

Kuter DJ, Efraim M, Mayer J, Trneny M, McDonald V, Bird R, Regenbogen T, Garg M, Kaplan Z, Tzvetkov N, Choi PY, Jansen AJG, Kostal M, Baker R, Gumulec J, Lee EJ, Cunningham I, Goncalves I, Warner M, Boccia R, Gernsheimer T, Ghanima W, Bandman O, Burns R, Neale A, Thomas D, Arora P, Zheng B, Cooper N. Rilzabrutinib, an Oral BTK Inhibitor, in Immune Thrombocytopenia. N Engl J Med. 2022 Apr 14;386(15):1421-1431. doi: 10.1056/NEJMoa2110297.

Reference Type DERIVED
PMID: 35417637 (View on PubMed)

Del Pozo Martin Y. 2021 ASH annual meeting. Lancet Haematol. 2022 Feb;9(2):e92-e93. doi: 10.1016/S2352-3026(21)00384-7. Epub 2021 Dec 16. No abstract available.

Reference Type DERIVED
PMID: 34922648 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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https://www.trialsummaries.com/Study/StudyDetails?id=26252&tenant=MT_SNY_9011

DFI17124 Plain Language Results Summary

Other Identifiers

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PRN1008-010

Identifier Type: OTHER

Identifier Source: secondary_id

U1111-1260-4044

Identifier Type: REGISTRY

Identifier Source: secondary_id

2023-509397-39

Identifier Type: REGISTRY

Identifier Source: secondary_id

DFI17124

Identifier Type: -

Identifier Source: org_study_id