Rilzabrutinib for Immune Thrombocytopenia

Recruiting in Palo Alto (17 mi)

+60 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Principia Biopharma, a Sanofi Company

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?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.

Eligibility Criteria

Adults aged 18 to 80 with immune-related ITP, a condition where the immune system destroys platelets, are eligible for this trial. Participants must have a low platelet count and not respond well to other treatments. Those with drug or alcohol abuse, organ transplants, HIV, hepatitis B/C, or who are pregnant/breastfeeding cannot join.

Inclusion Criteria

I am between 18 and 80 years old.

I have immune-related ITP.

Exclusion Criteria

You are currently misusing drugs or alcohol.

You have tested positive for HIV, hepatitis B, or hepatitis C during screening.

I have had a solid organ transplant.

+1 more

Participant Groups

The study is testing Rilzabrutinib (PRN1008), specifically at a dose of 400 mg twice daily for adults with refractory or relapsed ITP after completing an initial dose-finding phase. The goal is to see if it can safely increase platelet counts in these patients.

1Treatment groups

Experimental Treatment

Group I: Rilzabrutinib (PRN1008) DailyExperimental Treatment1 Intervention

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