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

Study on Immune Thrombocytopenia Treatment with Investigational Medication

Recruiting
18 years or above
All
Phase 3
60 participants needed
3 Locations

Study 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.

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions: Immune Thrombocytopenia
  • Age: 18 years or above
  • Gender: All

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.

This study investigates the treatment of immune thrombocytopenia (ITP) in adults who did not respond to first-line treatments. The purpose is to evaluate the effects of a new investigational medication. Participants will be in the study for a total of 60 weeks. This includes a screening period of up to 8 weeks, a primary analysis period of up to 28 weeks, a long-term extension period for selected participants of 28 weeks, and a follow-up period of 24 weeks for eligible participants.

Study procedures include taking the investigational medication and undergoing regular health assessments to monitor the effects of the treatment. Participants will have routine blood tests and medical check-ups to ensure safety and effectiveness of the treatment.

  • Who can participate: Adults aged 18 years and older with a documented diagnosis of primary ITP can participate. They must have received first-line therapy and experienced a loss of response or relapse. Individuals with secondary ITP or certain medical histories, such as other blood disorders or recent malignancies, are excluded.
  • Study details: Participants will take the investigational medication and attend regular medical visits for health assessments.
  • Study timelines: The study will last 60 weeks.
Updated on 25 Feb 2026. Study ID: NCT07007962

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