Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP) (LUNA 3)

Study of an Investigational Medication for Persistent or Chronic Immune Thrombocytopenia (ITP)

Not Recruiting
10 years or above
All
Phase 3
232 participants needed

Study Overview

This is a randomized, double-blind study of rilzabrutinib in patients with persistent or chronic ITP, with an average platelet count of <30,000/μL (and no single platelet count >35,000/μL) on two counts at least 5 days apart in the 14 days before treatment begins. Patients will receive rilzabrutinib or placebo 400mg twice daily.

For each patient, the study will last up to 60 weeks from the start of the Screening Period to the End of Study (EOS) visit. This includes Screening (up to 4 weeks) through a 12 to 24-week Blinded Treatment Period followed by a 28-week Open-Label Period. Followed by a 4-week post dose follow-up.

For adult participants, the maximum duration of the long-term extension (LTE) period will be 12 months from the date of the last adult participant to enter the LTE.

For pediatric participants, the maximum duration of the LTE period will be 12 months from the date of the last pediatric participant to enter the LTE.

Eligibility Criteria

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

  • Conditions: Immune Thrombocytopenia
  • Age: 10 years or above
  • Gender: All

Inclusion Criteria:

  1. Patients will be male and female with primary ITP with duration of >6 months in pediatric participants aged 12 to <18 years (pediatric participants aged 10 to <12 years will be enrolled in the EU [EEA countries] only) and duration of >3 months in ages 18 years and above
  2. Patients who had a response (achievement of platelet count ≥50,000/µL) to IVIg/anti-D or CSs that was not sustained and who have documented intolerance, insufficient response or any contra-indication to any appropriate courses of standard of care ITP therapy
  3. An average of 2 platelet counts at least 5 days apart of <30,000/µL during the Screening period and no single platelet count >35,000/µL, within 14 days prior to the first dose of study drug.
    • Pediatric patients must additionally be determined to need treatment for ITP as per clinical assessment by the Investigator.
  4. Adequate hematologic, hepatic, and renal function (absolute neutrophil count ≥1.5 X

    10^9/L, AST/ALT ≤1.5 x upper limit of normal [ULN], albumin ≥3 g/dL, total bilirubin ≤1.5 x ULN [unless the patient has documented Gilbert syndrome], glomerular filtration rate >50 [Cockcroft and Gault method for adult and Bedside Schwartz Equation for Pediatric participants])

  5. Hemoglobin >9 g/dL within 1 week prior to Study Day 1
  6. All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
  7. Patients must be able to provide written informed consent or informed assent with corresponding informed consent obtained from the patient's guardian and agree to the schedule of assessments

Exclusion Criteria:

  1. Patients with secondary ITP
  2. Pregnant or lactating women
  3. History (within 5 years of Study Day 1) or current, active malignancy requiring or likely to require chemotherapeutic or surgical treatment during the study, with the exception of non melanoma skin cancer
  4. Transfusion with blood, blood products, plasmapheresis, or use of any other rescue medications with intent to increase platelet count within 14 days before Study Day 1
  5. Change in CS and/or TPO-RA dose within 14 days prior to Study Day 1 (more than 10% variation from current doses)
  6. Immunosuppressant drugs other than CSs within 5 times the elimination half-life of the drug or 14 days of Study Day 1, whichever is longer
  7. Treatment with rituximab or splenectomy within the 3 months prior to Study Day 1
    • Patients treated with rituximab will have normal B-cell counts prior to enrollment
  8. Has received any investigational drug within the 30 days before receiving the first

    dose of study medication, or at least 5 times elimination half-life of the drug (whichever is longer); patient should not be using an investigational device at the time of dosing

    • Patients who previously received treatment with Bruton's Tyrosine Kinase (BTK) inhibitors (except rilzabrutinib) within 30 days before the first dose of study drug are not eligible
    • Patients who previously received rilzabrutinib at any time are not eligible
  9. History of solid organ transplant
  10. Myelodysplastic syndrome
  11. Live vaccine within 28 days prior to Study Day 1 or plan to receive one during the study
  12. Planned surgery in the time frame of the dosing period

Updated on 04 Apr 2025. Study ID: NCT04562766

This study investigates the effects of an investigational medication on patients with persistent or chronic Immune Thrombocytopenia (ITP). ITP is a condition where the immune system mistakenly destroys platelets, which are cells that help blood clot. Patients with ITP often have low platelet counts, which can lead to easy bruising and bleeding.

In this study, participants will receive either the investigational medication or a placebo, which is an inactive substance that looks like the investigational medicine but does not contain any medicine. Participants will take the study medication twice daily. The study includes different phases such as a Blinded Treatment Period and an Open-Label Period, where all participants receive the investigational medication.

  • Who can participate: Participants can be male or female with primary ITP. Pediatric participants must be aged 12 to under 18 years, or 10 to under 12 years in certain regions. Adults must be 18 years or older. Key eligibility includes a history of ITP for more than 6 months in children and more than 3 months in adults.
  • Study details: Participants will take the investigational medication or a placebo twice daily. A placebo is an inactive substance that looks like the investigational medicine but does not contain any medicine. The study involves a Blinded Treatment Period, where neither participants nor researchers know who receives the investigational medication, and an Open-Label Period, where all receive the investigational medication.
  • Study Timelines: The study will last up to 60 weeks.