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

Phase 3RecruitingNCT07007962

Sanofi60 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Immune Thrombocytopenia

Interventions

rilzabrutinibDRUG

Pharmaceutical form:Tablet-Route of administration:Oral

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (20)

University of Michigan Health - Michigan Medicine - University Hospital-Investigational Site Number: 8400001

Ann Arbor, Michigan, United States

Outcomes

Primary Outcomes

Durable platelet response

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

Time frame: Until Week 28

Secondary Outcomes

Overall platelet response

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

Time frame: Until Week 28

Duration of platelet response

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)

Time frame: Until Week 28

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

Time frame: Until Week 28

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

Time frame: 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)

Time frame: Until Week 80

Central Contacts

Trial Transparency email recommended (Toll free for US & Canada)

CONTACT

800-633-1610 contact-us@sanofi.com

Data from ClinicalTrials.gov

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