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

Study of an Investigational Study Drug for Adults With Immune Thrombocytopenia

Age
18 years or above
Gender
All
Phase
Phase 3
Participants needed
60
Locations
12
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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.

The purpose of this research study is to learn about the effects and safety of an investigational study drug in adults with primary immune thrombocytopenia (ITP) whose first treatment worked at first but then stopped working well enough. This includes people who have had a relapse (the condition came back) or who depend on steroid treatment to keep their ITP under control.

Participants will receive an investigational study drug taken by mouth. Everyone in the study will receive the investigational study drug.

Who can participate:
Adults with primary ITP may be able to participate if they:

  • Are 18 years old or older
  • Have a documented diagnosis of primary ITP
  • Have received at least one course of first-line treatment for ITP and responded while on that treatment
  • Have lost response, had a relapse, or are dependent on steroids

Study timeline and visits:
Participation can last up to about 88 weeks for eligible participants, including follow-up. Some participants may continue in an additional study period.

Updated on 18 Jun 2026. Study ID: NCT07007962

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