- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03395210
A Study of Rilzabrutinib in Adult Patients With Immune Thrombocytopenia (ITP)
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
Study Overview
Status
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
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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Australian Capital Territory
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Canberra, Australian Capital Territory, Australia, 2605
- Investigational Site Number : 105
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New South Wales
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Sydney, New South Wales, Australia, 2139
- Investigational Site Number : 104
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Queensland
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Woolloongabba, Queensland, Australia, 4102
- Investigational Site Number : 102
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Victoria
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Clayton, Victoria, Australia, 3168
- Investigational Site Number : 101
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Parkville, Victoria, Australia, 3050
- Investigational Site Number : 106
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Western Australia
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Perth, Western Australia, Australia, 6005
- Investigational Site Number : 103
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Pleven, Bulgaria, 5800
- Investigational Site Number : 213
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Sofia, Bulgaria, 1431
- Investigational Site Number : 214
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Varna, Bulgaria, 9010
- Investigational Site Number : 211
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Ontario
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Toronto, Ontario, Canada, M5B 1W8
- Investigational Site Number : 1161
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Quebec
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Montreal, Quebec, Canada, H3A 1A1
- Investigational Site Number : 1162
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Brno, Czechia, 62500
- Investigational Site Number : 431
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Hradec Kralove, Czechia, 50005
- Investigational Site Number : 433
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Ostrava - Poruba, Czechia, 70852
- Investigational Site Number : 434
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Praha 2, Czechia, 12808
- Investigational Site Number : 432
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Rotterdam, Netherlands, 3015 GD
- Investigational Site Number : 727
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s-Gravenhage, Netherlands, 2545 CH
- Investigational Site Number : 728
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Bergen, Norway, N-5021
- Investigational Site Number : 542
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Gralum, Norway, 1714
- Investigational Site Number : 541
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Leicestershire
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Leicester, Leicestershire, United Kingdom, LE1 5WW
- Investigational Site Number : 981
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London, City Of
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London, London, City Of, United Kingdom, E1 2ES
- Investigational Site Number : 983
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London, London, City Of, United Kingdom, W12 0HS
- Investigational Site Number : 980
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Illinois
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Peoria, Illinois, United States, 61614
- Bleeding and Clotting Disorders Institute Site Number : 1087
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Maryland
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Bethesda, Maryland, United States, 20817-1915
- RCCA MC LLC Site Number : 1091
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Massachusetts
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Boston, Massachusetts, United States, 02114-2603
- Massachusetts General Hospital Cancer Center Site Number : 1092
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Boston, Massachusetts, United States, 02215
- Beth Israel Deaconess Medical Center Site Number : 1099
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Michigan
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Midland, Michigan, United States, 48670
- Mid Michigan Medical Center Site Number : 1086
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New York
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New York, New York, United States, 10021
- New York Presbyterian Hospital/Weill Cornell Medical Center Site Number : 1097
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North Carolina
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Greenville, North Carolina, United States, 27834
- Pitt County Memorial Hospital Site Number : 1095
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Washington
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Seattle, Washington, United States, 98109-4405
- Seattle Cancer Care Alliance Site Number : 1098
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
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
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 |
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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 |
BTK inhibitor
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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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]
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The incidence, severity and relationship of TEAEs during the treatment period and at the 4-week follow-up from last dose received.
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24 weeks of treatment, long term extension and 4 weeks of follow up post last dose]
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Part A: Consecutive Increased Platelet Counts (Efficacy Outcome Measure)
Time Frame: 24 weeks
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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
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24 weeks
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Part B: Sustained Increase in Platelet Counts (Efficacy Outcome Measure)
Time Frame: 24 weeks
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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
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24 weeks
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Collaborators and Investigators
Investigators
- Study Director: Olga Bandman, MD, Principia Biopharma
Publications and helpful links
General Publications
- 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.
- 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.
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Pathologic Processes
- Immune System Diseases
- Autoimmune Diseases
- Hematologic Diseases
- Hemorrhage
- Hemorrhagic Disorders
- Blood Coagulation Disorders
- Skin Manifestations
- Blood Platelet Disorders
- Thrombotic Microangiopathies
- Purpura
- Purpura, Thrombocytopenic
- Purpura, Thrombocytopenic, Idiopathic
- Thrombocytopenia
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)?
IPD Plan Description
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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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