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BTK Inhibitor

Rilzabrutinib for Immune Thrombocytopenia

Phase 2

Waitlist Available

Research Sponsored by Principia Biopharma, a Sanofi Company

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Immune-related ITP (both primary and secondary)

Be older than 18 years old

Must not have

History of solid organ transplant

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 weeks of treatment, long term extension and 4 weeks of follow up post last dose]

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new drug, PRN1008, for people with ITP who have tried other treatments that haven't worked. The dosage is still being determined, but right now it's 400 mg twice daily.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects for Rilzabrutinib in this trial aren't listed here, common ones may include digestive issues, headaches, fatigue and potential risks associated with lowering the body's immune response.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have immune-related ITP.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I have had a solid organ transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 weeks of treatment, long term extension and 4 weeks of follow up post last dose]

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 weeks of treatment, long term extension and 4 weeks of follow up post last dose]

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 weeks of treatment, long term extension and 4 weeks of follow up post last dose] for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Part A and B: Incidence of Treatment Emergent Adverse Events (Safety Outcome Measure)

Part A: Consecutive Increased Platelet Counts (Efficacy Outcome Measure)

Part B: Sustained Increase in Platelet Counts (Efficacy Outcome Measure)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Rilzabrutinib

2021

Completed Phase 2

~380

0 / 2Eligibility criteria met