The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
Condition(s) studied
Investigational drug(s) / intervention(s)
Rilzabrutinib: Pharmaceutical form:Tablet -Route of administration:Oral
Placebo: Pharmaceutical form:Tablet -Route of administration:Oral
Study summary
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of rilzabrutinib in participants with sickle-cell disease (SCD).
Study details include:
* Study duration: a 52-week double-blind period (Part A), followed by an open-label LTE period (Part B). Double-blind period has two parts, 50% (adult only) until the interim analysis (a proof-concept part analogous to a phase 2b study), and 50% (adult and children) after the interim analysis. Only the participants who complete double-blind treatment period (Part A) are eligible to continue to the LTE period. The duration of the LTE period (Part B) will be from the first-participant-in (FPI)-LTE (Part B) until the last participant who enters the LTE has completed 52 weeks.
* Treatment duration: 52-week double-blind period (Part A); LTE period (Part B) from the (FPI until the last participant who enters the LTE has completed 52 weeks.
* Visit frequency: Week visits based on the Schedule of Assessments.
Eligibility
Primary outcome measure(s)
- Annualized rate of clinical VOC — At Week 52
Calculated from total number of clinical VOC incidents and total number of days during the observation period
Trial sites (63)
| Facility | City | Region | Status |
|---|---|---|---|
| University of Alabama at Birmingham- Site Number : 8400003 | Birmingham | Alabama | Recruiting |
| Phoenix Children's Hospital- Site Number : 8400028 | Phoenix | Arizona | Recruiting |
| University of California San Francisco- Site Number : 8400040 | Clovis | California | Recruiting |
| David Geffen School of Medicine at UCLA- Site Number : 8400032 | Los Angeles | California | Recruiting |
| MedStar Georgetown University Hospital- Site Number : 8400036 | Washington D.C. | District of Columbia | Recruiting |
| Oncology & Hematology Associates of West Broward- Site Number : 8400029 | Coral Springs | Florida | Recruiting |
| Sylvester Comprehensive Cancer Center- Site Number : 8400020 | Miami | Florida | Recruiting |
| University of Illinois-Chicago - College of Medicine- Site Number : 8400054 | Chicago | Illinois | Recruiting |
| Indiana University Health Riley Hospital for Children- Site Number : 8400056 | Indianapolis | Indiana | Recruiting |
| Louisiana State University Health Sciences Center - Shreveport- Site Number : 8400037 | Shreveport | Louisiana | Recruiting |
| University of Maryland School of Medicine - Baltimore- Site Number : 8400041 | Baltimore | Maryland | Recruiting |
| University of Michigan Health System - Ann Arbor- Site Number : 8400035 | Ann Arbor | Michigan | Recruiting |
| Southern Specialty Research- Site Number : 8400059 | Flowood | Mississippi | Recruiting |
| Richmond University Medical Center- Site Number : 8400038 | Staten Island | New York | Recruiting |
| Montefiore Medical Center - Moses Campus- Site Number : 8400057 | The Bronx | New York | Recruiting |
| Oregon Health & Science University (OHSU)- Site Number : 8400043 | Portland | Oregon | Recruiting |
| Prisma Health- Site Number : 8400051 | Greenville | South Carolina | Recruiting |
| University of Tennessee Health Science Center- Site Number : 8400045 | Memphis | Tennessee | Recruiting |
| Baylor College of Medicine- Site Number : 8400055 | Houston | Texas | Recruiting |
| UT Physicians - Memorial Hermann- Site Number : 8400052 | Houston | Texas | Recruiting |
| VCU Massey Cancer Center: Dalton Oncology Clinic- Site Number : 8400012 | Richmond | Virginia | Recruiting |
| Investigational Site Number : 0560003 | Brussels | Belgium | Recruiting |
| Investigational Site Number : 0560002 | Brussels | Belgium | Recruiting |
| Investigational Site Number : 0560001 | Leuven | Belgium | Recruiting |
| Investigational Site Number : 0560004 | Liège | Belgium | Recruiting |
| Hospital Santa Izabel- Site Number : 0760006 | Salvador | Estado de Bahia | Recruiting |
| Fundação Bahiana de Infectologia- Site Number : 0760009 | Salvador | Estado de Bahia | Recruiting |
| Universidade Federal de Goias- Site Number : 0760002 | Goiânia | Goiás | Recruiting |
| Fundação Faculdade Regional de Medicina de São José do Rio Preto- Site Number : 0760001 | São José do Rio Preto | São Paulo | Recruiting |
| Hospital Samaritano De Sao Paulo- Site Number : 0760005 | São Paulo | Brazil | Recruiting |
| Investigational Site Number : 2500002 | Créteil | France | Recruiting |
| Investigational Site Number : 2500005 | Marseille | France | Recruiting |
| Investigational Site Number : 2500001 | Paris | France | Recruiting |
| Investigational Site Number : 2500004 | Toulouse | France | Recruiting |
| Investigational Site Number : 2760005 | Berlin | Germany | Recruiting |
| Investigational Site Number : 2760002 | Essen | Germany | Recruiting |
| Investigational Site Number : 2760004 | Stuttgart | Germany | Recruiting |
| Investigational Site Number : 3000001 | Athens | Greece | Recruiting |
| Investigational Site Number : 3000003 | Athens | Greece | Recruiting |
| Investigational Site Number : 3000002 | Pátrai | Greece | Recruiting |
+ 23 more sites — see the full list on the official registry below.
More Sanofi trials in France
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 NCT06975865 on ClinicalTrials.gov ↗ ← All trials in France