🇮🇪Ireland
16°C Partly Cloudy · Dublin
Live Updates
--:--:-- IST
Writer Login
Latest
Clinical Trials in the USA / NCT04562766
Active, not recruiting Phase 3

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

NCT04562766 · tracked via the Priya Life Science USA tracker
Sponsor
Principia Biopharma, a Sanofi Company
Phase
Phase 3
Started
2020-12-14
Last updated
2026-02-23

Condition(s) studied

Immune Thrombocytopenia

Investigational drug(s) / intervention(s)

RilzabrutinibPlacebo

Rilzabrutinib: 400mg Caplet

Placebo: 400mg Caplet

Study summary

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.

Eligibility

Sex
ALL
Min age
10 Years
Max age
Healthy volunteers
No
Inclusion Criteria: 1. 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. 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 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

Primary outcome measure(s)

Trial sites (155)

FacilityCityRegionStatus
University of Southern California_Investigational Site Number 84024 Los Angeles California
UCSF Benioff Children's Hospital San Francisco_Investigational Site Number 84020 San Francisco California
Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center_Investigational Site Number 84037 Torrance California
The Oncology Institute of Hope and Innovation_Investigational Site Number 84031 Whittier California
Children's Hospital Colorado_Investigational Site Number 84025 Aurora Colorado
IMMUNOe International Research Centers_Investigational Site Number 84028 Centennial Colorado
ASCLEPES Research Centers_Investigational Site Number 84023 Weeki Wachee Florida
Children's Healthcare of Atlanta_Investigational Site Number 84034 Atlanta Georgia
Rush University Medical Center_Investigational Site Number 84029 Chicago Illinois
University of Louisville - James Graham Brown Cancer Center_Investigational Site Number 84033 Louisville Kentucky
Massachusetts General Hospital Site Number : 84038 Boston Massachusetts
Montefiore Medical Center_Investigational Site Number 84032 The Bronx New York
University Hospitals Cleveland Medical Center Site Number : 84036 Cleveland Ohio
Cleveland Clinic_Investigational Site Number 84026 Cleveland Ohio
The Children's Hospital of Philadelphia (CHOP)_Investigational Site Number 84027 Philadelphia Pennsylvania
University of Utah-Huntsman Cancer Institute_Investigational Site Number 84035 Salt Lake City Utah
University of Washington Medical Centre Site Number : 84041 Seattle Washington
Investigational Site Number : 3206 Capital Federal Buenos Aires
Investigational Site Number : 3211 La Plata Buenos Aires
Investigational Site Number : 3205 Córdoba Córdoba Province
Investigational Site Number : 3209 Corrientes Argentina
Investigational Site Number : 3208 San Juan Argentina
Investigational Site Number : 3607 Kogarah New South Wales
Investigational Site Number : 3608 Westmead New South Wales
Investigational Site Number : 3611 Brisbane Queensland
Investigational Site Number : 3609 Adelaide South Australia
Investigational Site Number : 3606 Frankston Victoria
Investigational Site Number : 3610 Perth Western Australia
Investigational Site Number : 4005 Graz Austria
Investigational Site Number : 4004 Leoben Austria
Investigational Site Number : 4001 Linz Austria
Investigational Site Number : 4003 Steyr Austria
Investigational Site Number : 4002 Vienna Austria
Hospital Sao Rafael Instituto D'Or da Bahia Site Number : 7608 Salvador Estado de Bahia
Uniao Oeste Paranaense de Estudos e Combates ao Cancer Site Number : 7610 Cascavel Paraná
Hospital De Clinicas De Porto Alegre Site Number : 7605 Porto Alegre Rio Grande do Sul
Hospital Santa Marcelina Site Number : 7611 São Paulo São Paulo
CEMEC Oncologica do Brasil Site Number : 7606 Belém Brazil
HEMORIO - Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti Site Number : 7609 Rio de Janeiro Brazil
Hospital do Servidor Publico Estadual de Sao Paulo Site Number : 7607 São Paulo Brazil

+ 115 more sites — see the full list on the official registry below.

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04562766 on ClinicalTrials.gov ↗ ← All trials in the USA