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 the USA
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 the USA