Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
Condition(s) studied
Investigational drug(s) / intervention(s)
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
Primary outcome measure(s)
- DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in Regions Except European Union and United Kingdom — Up to 24 weeks
Durable platelet response per guidance in regions except European Union and United Kingdom was defined as platelet counts at or above 50,000/mcL for \>=two-thirds of at least 8 non-missing weekly scheduled platelet measurements during the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy, provided that at least 2 non-missing weekly scheduled platelet measurements were at or above 50,000/mcL during the last 6 weeks of the 24-week blinded treatment period. - DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in European Union and United Kingdom — Up to 24 weeks
Durable platelet response per guidance in European Union and United Kingdom was defined as platelet counts at or above 50,000/mcL for at least 8 out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy.
Trial sites (155)
| Facility | City | Region | Status |
|---|---|---|---|
| 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
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 Netherlands