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

Principia Biopharma

Status and phase: Active, not recruiting; Phase 2

Conditions: Immune Thrombocytopenia; Immune Thrombocytopenic Purpura

Treatments: Drug: Rilzabrutinib

Study type: Interventional

Funder types: Industry

Identifiers

NCT03395210

2023-509397-39 (Registry Identifier)

PRN1008-010 (Other Identifier)

U1111-1260-4044 (Registry Identifier)

DFI17124

Details and patient eligibility

About

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.

Full 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.

Enrollment: 81 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Sequential Assignment

Masking: None (Open label)

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81 participants in 1 patient group

Rilzabrutinib (PRN1008) Daily

Experimental group

Description:

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

Treatment:

Drug: Rilzabrutinib