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

  • STATUS
    Recruiting
  • End date
    Nov 20, 2026
  • participants needed
    194
  • sponsor
    Principia Biopharma, a Sanofi Company
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Updated on 3 December 2024
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platelet count
thrombocytopenia
kidney function tests
neutrophil count
igiv

Summary

This is a randomized, double-blind study of rilzabrutinib in participants 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. Participants will receive rilzabrutinib or placebo 400mg twice daily.

For each participant, 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.

Details
Condition Immune Thrombocytopenia
Age 10years or above
Treatment Placebo, Rilzabrutinib
Clinical Study IdentifierNCT04562766
SponsorPrincipia Biopharma, a Sanofi Company
Last Modified on3 December 2024

Eligibility

 Yes No Not Sure

Inclusion Criteria

Participants 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
Participants 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 contraindication 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 during the Screening period and no single platelet count >35,000/µL, within 14 days prior to the first dose of study drug
Pediatric participants 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 ×
Hemoglobin >9 g/dL within 1 week prior to Study Day 1
^9/L, AST/ALT ≤1.5 × upper limit of normal [ULN], albumin ≥3 g/dL, total
All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
bilirubin ≤1.5 × ULN [unless the partcipant has documented Gilbert syndrome]
glomerular filtration rate >50 [Cockcroft and Gault method])
Participants must be able to provide written informed consent or informed assent with corresponding informed consent obtained from the participants' guardian and agree to the schedule of assessments

Exclusion Criteria

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
Participants with secondary ITP
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
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
Participants treated with rituximab will have normal B-cell counts prior to enrollment
device at the time of dosing
drug (whichever is longer); participant should not be using an investigational
History of solid organ transplant
Myelodysplastic syndrome
Live vaccine within 28 days prior to Study Day 1 or plan to receive one during the study
Participants 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
Planned surgery in the time frame of the dosing period
Participants who previously received rilzabrutinib at any time are not eligible
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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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