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

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.

Brief Title

Categories

Immune System

Blood Disorders

Central Contacts

Central Contact Role

Contact

Central Contact Phone

800-633-1610

Central Contact Phone Ext

option 6

Central Contact Email

contact-us@sanofi.com

Completion Date

2028-12-30

Completion Date Type

Estimated

Conditions

Immune Thrombocytopenia

Eligibility Criteria

Key Inclusion Criteria:

* Male or female participants aged 18 years and older with a documented diagnosis of primary ITP in the medical history
* Participant received at least one course of 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, including genetic conditions, 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.

Inclusion Criteria

Inclusion Criteria:

* Male or female participants aged 18 years and older with a documented diagnosis of primary ITP in the medical history
* Participant received at least one course of first-line therapy and had a history of response while on treatment
* Participant has loss of response, relapse, or steroid dependency

Gender

All

Gender Based

false

Healthy Volunteers

No

Last Update Post Date

Fri, 02/27/2026 - 12:00

Last Update Post Date Type

Actual

Last Update Submit Date

Wed, 02/25/2026 - 12:00

Minimum Age

18 Years

NCT Id

NCT07007962

Org Class

Industry

Org Full Name

Sanofi

Org Study Id

LPS18573

Overall Status

Recruiting

Phases

Phase 3

Primary Completion Date

Fri, 11/05/2027 - 12:00

Primary Completion Date Type

Estimated

Official Title

A Multi-center, Open-label, Single-arm Study to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults With Immune Thrombocytopenia (ITP) Who Failed First-line Treatment

Primary Outcomes

Outcome Description

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

Outcome Measure

Durable platelet response

Outcome Time Frame

Until Week 28

Secondary Ids

Secondary Id

U1111-1320-4669

Secondary Outcomes

Outcome Description

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 therapy in the 4 weeks prior to the first elevated platelet count

Outcome Time Frame

Until Week 28

Outcome Measure

Overall platelet response

Outcome Description

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 therapy in the 4 weeks prior to the elevated platelet count in participants who achieve a response (single platelet count)

Outcome Time Frame

Until Week 28

Outcome Measure

Duration of platelet response

Outcome Time Frame

Until Week 28

Outcome Measure

Change from baseline in the immune thrombocytopenia bleeding scale (IBLS) score at the end of week 28

Outcome Time Frame

Until Week 28

Outcome Measure

Percentage of participants able to discontinue or reduce CS dose by at least 50% or to <5 mg/day (prednisone equivalent) from baseline at the end of week 28

Outcome Time Frame

Until Week 80

Outcome Measure

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)

Outcome Description

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)

Outcome Time Frame

Until Week 80

Outcome Measure

Percentage of participants with potential clinical significant abnormal (PCSA)