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

Running, not enrolling · Phase 3 · Has a placebo group

Conditions studied: Immune Thrombocytopenia

In brief

This was 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 received rilzabrutinib or placebo 400mg twice daily. For each patient, the study lasted up to 60 weeks from the start of the Screening Period to the End of Study (EOS) visit. This included 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 was 12 months from the date of the last adult participant to enter the LTE. For pediatric participants, the maximum duration of the LTE period was 12 months from the date of the last pediatric participant to enter the LTE.

Key facts

Study ID

NCT04562766

Run by

Principia Biopharma, a Sanofi Company

People needed

232

Starts

2020-12-14

Expected to finish

2026-08-26

Last updated by the study team

2026-02-23

Who can join

Age: 10 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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
• 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 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.
• 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])
• 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

You may not qualify if…

• 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
• Had 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
• 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
• Planned surgery in the time frame of the dosing period

Where it is running

• UCSF Benioff Children's Hospital San Francisco_Investigational Site Number 84020 — San Francisco, California, United States
• Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center_Investigational Site Number 84037 — Torrance, California, United States
• The Oncology Institute of Hope and Innovation_Investigational Site Number 84031 — Whittier, California, United States
• Children's Hospital Colorado_Investigational Site Number 84025 — Aurora, Colorado, United States
• IMMUNOe International Research Centers_Investigational Site Number 84028 — Centennial, Colorado, United States
• ASCLEPES Research Centers_Investigational Site Number 84023 — Weeki Wachee, Florida, United States
• Children's Healthcare of Atlanta_Investigational Site Number 84034 — Atlanta, Georgia, United States
• Rush University Medical Center_Investigational Site Number 84029 — Chicago, Illinois, United States
• University of Louisville - James Graham Brown Cancer Center_Investigational Site Number 84033 — Louisville, Kentucky, United States
• Massachusetts General Hospital Site Number : 84038 — Boston, Massachusetts, United States
• Montefiore Medical Center_Investigational Site Number 84032 — The Bronx, New York, United States
• University Hospitals Cleveland Medical Center Site Number : 84036 — Cleveland, Ohio, United States
• Cleveland Clinic_Investigational Site Number 84026 — Cleveland, Ohio, United States
• The Children's Hospital of Philadelphia (CHOP)_Investigational Site Number 84027 — Philadelphia, Pennsylvania, United States
• University of Utah-Huntsman Cancer Institute_Investigational Site Number 84035 — Salt Lake City, Utah, United States
• University of Washington Medical Centre Site Number : 84041 — Seattle, Washington, United States
• Investigational Site Number : 3206 — Capital Federal, Buenos Aires, Argentina
• Investigational Site Number : 3211 — La Plata, Buenos Aires, Argentina
• Investigational Site Number : 3205 — Córdoba, Córdoba Province, Argentina
• Investigational Site Number : 3209 — Corrientes, Argentina
• Investigational Site Number : 3208 — San Juan, Argentina
• Investigational Site Number : 3607 — Kogarah, New South Wales, Australia
• Investigational Site Number : 3608 — Westmead, New South Wales, Australia
• Investigational Site Number : 3611 — Brisbane, Queensland, Australia
• University of Southern California_Investigational Site Number 84024 — Los Angeles, California, United States

Full record on ClinicalTrials.gov

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