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NCT Number: NCT07007962

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

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.

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Key information

Conditions

Immune Thrombocytopenia Autoimmune Diseases Blood Coagulation Disorders Blood Platelet Disorders Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Hemorrhagic Disorders Immune System Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Purpura Purpura, Thrombocytopenic Purpura, Thrombocytopenic, Idiopathic Signs and Symptoms Skin Manifestations Thrombocytopenia Thrombotic Microangiopathies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Investigational Site Number : 0400001, Vienna, Austria

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Rilzabrutinib

Drug

Pharmaceutical form:Tablet-Route of administration:Oral

Other names: SAR444671

Primary outcomes

  1. Durable platelet response

    Time frame: 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 outcomes

  1. Overall platelet response

    Time frame: 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 therapy in the 4 weeks prior to the first elevated platelet count

  2. Duration of platelet response

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

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

    Time frame: Until Week 28

  4. 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

  5. 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

  6. Percentage of participants with potential clinical significant abnormal (PCSA)

    Time frame: 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)

Study contacts

Contact information is provided by the study sponsor or research team.

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

CONTACT

[email protected]

800-633-1610 ext. option 6

Sponsors and collaborators

Lead sponsor

Sanofi

Industry

Registry information

Official study 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

Acronym: LUNA 4

Important dates

Study start
2025
Primary completion
2027
Study completion
2028
First posted
Jun 6, 2025
Registry last updated
Jun 22, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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