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

NCT03395210 | Completed Phase 2 Results DMC FDA Drug

• 4 other identifiers: DFI17124PRN1008-010U1111-1260-40442023-509397-39
• Study Type: interventional
• Target: 86
• Locations: 8 countries
• Sites: 31

Brief Summary

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.

Conditions

Immune Thrombocytopenia

Keywords

ITP

Outcome Measures

Primary Outcomes (4)

• Part A: Percentage of Participants Who Achieved 2 or More Consecutive Platelet Counts by Starting Dose Level and Overall

  The percentage of participants who achieved 2 or more consecutive platelet counts, separated by at least 5 days, of \>=50,000/ microliter (μL) and an increase of platelet count of \>=20,000/μL from baseline, by starting dose level and overall, without use of rescue medication in the 4 weeks prior to the latest elevated platelet count. 95% confidence interval (CI) was based on the Clopper-Pearson method. The average of the 2 screening results and the Cycle 1 Day 1 result were used as the baseline value.

  Up to 24 Weeks

• Part B: Percentage of Participants Who Achieved Platelet Counts >=50,000/μL

  The percentage of participants who achieved 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 after 10 weeks of active treatment. 95% CI was based on the Clopper-Pearson exact method.

  Up to 24 Weeks

• Part A: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment Related Treatment-Emergent Adverse Events

  Adverse event (AE): any untoward medical occurrence in participant or clinical study participant, temporally associated with the use of rilzabrutinib, whether or not considered related to rilzabrutinib. TEAEs: AEs that developed or worsened or became serious on or after the first dose administration of rilzabrutinib (Day 1). Any TEAEs are considered treatment-related TEAEs as per Investigator's evaluation of participant's circumstances surrounding the event, and an evaluation of any potential alternative causes to determine whether an TEAE can be considered as related to the rilzabrutinib.

  From first dose of rilzabrutinib (Day 1) up to last dose + 1 (up to 294 days)

• Part B: Number of Participants With Treatment-Emergent Adverse Events and Treatment Related Treatment-Emergent Adverse Events

  AE any untoward medical occurrence in participant or clinical study participant, temporally associated with the use of rilzabrutinib, whether or not considered related to rilzabrutinib. TEAEs: AEs that developed or worsened or became serious during the treatment-emergent period, defined as any time after the first dose administration of rilzabrutinib (Day 1). Any TEAEs are considered treatment-related TEAEs as per Investigator's evaluation of participant's circumstances surrounding the event, and an evaluation of any potential alternative causes to determine whether an TEAE can be considered as related to rilzabrutinib.

  From first dose of rilzabrutinib (Day 1) up to last dose + 1 (approximately 170 days)

Secondary Outcomes (22)

• Part A: Percentage of Weeks With Platelet Counts >=50,000/μL by Starting Dose Level and Overall

  Up to 24 Weeks

• Part A: Percentage of Participants With 4 Out of the Final 8 Platelet Counts >=50,000/μL by Starting Dose Level and Overall

  Up to 24 Weeks

• Part A: Change From Baseline to the Average of Post Day 1 Platelet Counts by Dose Level and Overall

  Baseline and up to 24 Weeks

• Part A: Number of Weeks With Platelet Counts >=50,000/μL by Starting Dose Level and Overall

  Up to 24 Weeks

• Part A: Number of Weeks With Platelet Counts >=30,000/μL by Starting Dose Level and Overall

  Up to 24 Weeks

Study Arms (1)

Rilzabrutinib (PRN1008) Daily

EXPERIMENTAL

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

Interventions

Rilzabrutinib DRUG

BTK inhibitor

Also known as: PRN1008

Rilzabrutinib (PRN1008) Daily

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female patients, aged 18 to 80 years old
• Immune-related ITP (both primary and secondary)

You may not qualify if:

• Pregnant or lactating women
• Current drug or alcohol abuse
• History of solid organ transplant
• Positive screening for HIV, hepatitis B, or hepatitis C

Sponsors & Collaborators

• Principia Biopharma, a Sanofi Company: lead

Study Sites (31)

Bleeding and Clotting Disorders Institute- Site Number : 1087

Peoria, Illinois, 61614-2868, United States

Location

RCCA MC LLC- Site Number : 1091

Bethesda, Maryland, 20817-1915, United States

Location

Massachusetts General Hospital Cancer Center- Site Number : 1092

Boston, Massachusetts, 02114-2603, United States

Location

Beth Israel Deaconess Medical Center- Site Number : 1099

Boston, Massachusetts, 02215, United States

Location

Mid Michigan Medical Center- Site Number : 1086

Midland, Michigan, 48670, United States

Location

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

New York, New York, 10021, United States

Location

Pitt County Memorial Hospital- Site Number : 1095

Greenville, North Carolina, 27834, United States

Location

Seattle Cancer Care Alliance Site Number : 1098

Seattle, Washington, 98109-4405, United States

Location

Investigational Site Number : 105

Canberra, Australian Capital Territory, 2605, Australia

Location

Investigational Site Number : 104

Sydney, New South Wales, 2139, Australia

Location

Investigational Site Number : 102

Woolloongabba, Queensland, 4102, Australia

Location

Investigational Site Number : 101

Clayton, Victoria, 3168, Australia

Location

Investigational Site Number : 106

Parkville, Victoria, 3050, Australia

Location

Investigational Site Number : 103

Perth, Western Australia, 6005, Australia

Location

Investigational Site Number : 213

Pleven, 5800, Bulgaria

Location

Investigational Site Number : 214

Sofia, 1431, Bulgaria

Location

Investigational Site Number : 211

Varna, 9010, Bulgaria

Location

Investigational Site Number : 1161

Toronto, Ontario, M5B 1W8, Canada

Location

Investigational Site Number : 1162

Montreal, Quebec, H3A 1A1, Canada

Location

Investigational Site Number : 431

Brno, 62500, Czechia

Location

Investigational Site Number : 433

Hradec Králové, 50005, Czechia

Location

Investigational Site Number : 434

Ostrava - Poruba, 70852, Czechia

Location

Investigational Site Number : 432

Prague, 12808, Czechia

Location

Investigational Site Number : 727

Rotterdam, 3015 GD, Netherlands

Location

Investigational Site Number : 728

The Hague, 2545 CH, Netherlands

Location

Investigational Site Number : 542

Bergen, N-5021, Norway

Location

Investigational Site Number : 541

Grålum, 1714, Norway

Location

Investigational Site Number : 981

Leicester, Leicestershire, LE1 5WW, United Kingdom

Location

Investigational Site Number : 983

London, London, City of, E1 2ES, United Kingdom

Location

Investigational Site Number : 980

London, London, City of, W12 0HS, United Kingdom

Location

Investigational Site Number : 984

Birmingham, B15 2GW, United Kingdom

Location

Related Publications (4)

• 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.

  PMID: 39844469 DERIVED

• 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.

  PMID: 38386978 DERIVED

• 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.

  PMID: 35417637 DERIVED

• 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.

  PMID: 34922648 DERIVED

Related Links

• DFI17124 Plain Language Results Summary

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Results Point of Contact

• Title: Trial Transparency Team
• Organization: Sanofi aventis recherche & développement

Contact-US@sanofi.com

8006331610

Study Officials

• Olga Bandman, MD

  Principia Biopharma

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 22, 2017
• First Posted: January 10, 2018
• Study Start: March 22, 2018
• Primary Completion: January 31, 2023
• Study Completion: December 11, 2025
• Last Updated: February 23, 2026
• Results First Posted: November 14, 2025

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share

Locations

NL (2); CZ (4); AU (6); US (8); CA (2); NO (2); GB (4); BU (3)