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

  • STATUS
    Recruiting
  • End date
    Jun 5, 2025
  • participants needed
    224
  • sponsor
    Principia Biopharma, a Sanofi Company
Updated on 5 June 2022
platelet count
thrombocytopenia
kidney function tests
neutrophil count
igiv

Summary

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. There is a 4-week post dose follow-up.

Patients who respond per specified criteria at the end of the Open-Label Period will be able to enter a 12-month Long-Term Extension (LTE).

Details
Condition Immune Thrombocytopenia
Treatment Placebo, rilzabrutinib
Clinical Study IdentifierNCT04562766
SponsorPrincipia Biopharma, a Sanofi Company
Last Modified on5 June 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Patients will be male and female with primary ITP with duration of >6 months in ages 12 to <18 years and duration of >3 months in ages 18 years and above
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
An average of 2 platelet counts at least 5 days apart of <30,000/µL (and no single platelet count >35,000/µL) -- Patients from 12 to <18 years of age must additionally be determined to need treatment for ITP as per clinical assessment by the Investigator
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])
Hemoglobin >9 g/dL within 1 week prior to Study Day 1
All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
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

History of solid organ transplant
Patients with secondary ITP
Pregnant or lactating women
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
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
Change in CS and/or TPO-RA dose within 14 days prior to Study Day 1 (more than 10% variation from current doses)
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
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
Has received any investigational drug within the 30 days before receiving the first
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
dose of study medication, or at least 5 times elimination half-life of the
Patients who previously received rilzabrutinib at any time are not eligible
drug (whichever is longer); patient should not be using an investigational
device at the time of dosing
Myelodysplastic syndrome
Live vaccine within 28 days prior to Study Day 1 or plan to receive one during the study
Planned surgery in the time frame of the dosing period
Clear my responses

How to participate?

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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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