Open-label Study of Oral Rilzabrutinib for Adults with Immune Thrombocytopenia Who Did Not Respond to First-line Treatment

What this Trial Is About

Researchers are evaluating the effects of oral rilzabrutinib in adults with immune thrombocytopenia ITP who have not responded to first-line treatment. This multinational, open-label, single-arm study focuses on early multi-immune modulation to manage ITP symptoms and improve platelet response. The trial is sponsored by Sanofi and is designed as a Phase 3 treatment study. Participants will receive rilzabrutinib tablets orally at a dose of 400 mg twice daily. The study is divided into several periods a screening phase lasting up to 8 weeks, a primary analysis phase of up to 28 weeks where the main treatment effects are assessed, followed by a long-term extension phase of 28 weeks for selected participants. Additionally, there is a 24-week follow-up period for eligible participants after treatment. During the study, participants will be monitored regularly to evaluate platelet response and bleeding symptoms using the immune thrombocytopenia bleeding scale, as well as their ability to reduce corticosteroid doses. Safety assessments will track adverse events and clinically significant abnormalities throughout the study, including the extension and follow-up phases. The total duration of participation may extend up to approximately 80 weeks, depending on individual eligibility for the long-term and follow-up periods.

Study to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults With Immune Thrombocytopenia (ITP) Who Failed First-line Treatment