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

Phase 3

Not yet recruiting

• Conditions: Immune Thrombocytopenia
• Interventions: Drug: rilzabrutinib

• Registration Number: NCT07007962
• Lead Sponsor: Sanofi

Brief Summary

This is a multinational, open label, single arm study that will evaluate the impact of early multi-immune modulation with rilzabrutinib in adult ITP patients who failed first-line treatment. The study includes a screening period (up to 8 weeks), a primary analysis period (up to 28 weeks), a long-term extension period for selected participants (28 weeks) and a 24-week follow-up period only for eligible participants.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-05
• Study First Submitted: 2025-05-20
• Study First Submitted QC: 2025-05-28
• Last Update Submitted: 2025-05-28
• Study First Posted: 2025-06-06
• Last Update Posted: 2025-06-06
• Study Start: 2025-07-21
• Primary Completion: 2027-11-05
• Study Completion: 2028-12-30

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

• Male or female participants aged 18 years and older with primary ITP
• Participant received at least a first line therapy and had a history of response while on treatment
• Participant has loss of response, relapse or steroid dependency

Key

Exclusion Criteria

• Participants with Secondary ITP
• Participants with Evans syndrome or history of myelodysplastic syndrome
• Participants with history of lymphoma, leukemia, or any malignancy within the past 5 years except for non-melanoma skin malignancy.
• Participants with history of solid organ transplant
• Participants with history of coagulation or bleeding disorders other than ITP
• Participant received advanced therapy for ITP or was splenectomized
• Pregnancy or nursing The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
rilzabrutinib	rilzabrutinib	400 mg BID

Primary Outcome Measures

Name	Time	Method
Durable platelet response	Until Week 28	Defined as the percentage of participants able to achieve platelet counts ≥50 000/μL (or ≥30 000/μL and \<50 000/μL and at least double from baseline) for ≥50% of 6 biweekly scheduled platelet measurements and at least 4 non-missing, biweekly visits during the last 12 weeks of the primary analysis period (PAP) in absence of rescue therapy

Secondary Outcome Measures

Name	Time	Method
Percentage of participants able to discontinue or reduce CS dose by 50% or to <5 mg from baseline at the end of week 28	Until Week 28
Change from baseline in the immune thrombocytopenia bleeding scale (IBLS) score at the end of week 28	Until Week 28
Frequency and severity of treatment emergent adverse events (TEAEs, including serious adverse events [SAEs], bleeding TEAEs, adverse event of special interest [AESI] and adverse events leading to discontinuation)	Until Week 80
Percentage of participants with potential clinical significant abnormal (PCSA)	Until Week 80	PCSA in physical examination, ECG, vital signs, and clinical laboratory test results: serum chemistry and hematology (except for platelet counts included in the primary efficacy endpoint)
Overall platelet response	Until Week 28	Percentage of participants able to achieve 2 platelet counts at least 5 days apart of ≥50 000/μL (or ≥30 000/μL and \<50 000/μL and at least double from baseline) without rescue medication in the 4 weeks prior to the first elevated platelet count
Duration of platelet response	Until Week 28	Cumulative number and proportion of non-missing weeks with platelet counts ≥50 000/μL (or ≥30 000/μL and \<50 000/μL and at least double from baseline) in absence of rescue medication in the 4 weeks prior to the elevated platelet count in participants who achieve a response (single platelet count)