The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
Start Date
8/12/2025
Completion Date
12/29/2028
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 Criteria
Age Range: 10 years to 65 years
Interventions
Rilzabrutinib
Placebo
Conditions
Locations
University of Alabama at Birmingham- Site Number : 8400003
Birmingham, Alabama 35233
United States
Phoenix Children's Hospital- Site Number : 8400028
Phoenix, Arizona 85016
United States
University of California San Francisco- Site Number : 8400040
Fresno, California 93701
United States
Oncology & Hematology Associates of West Broward- Site Number : 8400029
Coral Springs, Florida 33065
United States
Sylvester Comprehensive Cancer Center- Site Number : 8400020
Miami, Florida 33136
United States
University of Illinois-Chicago - College of Medicine- Site Number : 8400054
Chicago, Illinois 60612
United States
Indiana University Health Riley Hospital for Children- Site Number : 8400056
Indianapolis, Indiana 46202
United States
Louisiana State University Health Sciences Center - Shreveport- Site Number : 8400037
Shreveport, Louisiana 71130
United States
University of Maryland School of Medicine - Baltimore- Site Number : 8400041
Baltimore, Maryland 21201
United States
University of Michigan Health System - Ann Arbor- Site Number : 8400035
Ann Arbor, Michigan 48109
United States
Southern Specialty Research- Site Number : 8400059
Flowood, Mississippi 39232
United States
Richmond University Medical Center- Site Number : 8400038
Staten Island, New York 10310
United States
Montefiore Medical Center - Moses Campus- Site Number : 8400057
The Bronx, New York 10467
United States
Prisma Health- Site Number : 8400051
Greenville, South Carolina 29605
United States
Baylor College of Medicine- Site Number : 8400055
Houston, Texas 77030
United States
VCU Massey Cancer Center: Dalton Oncology Clinic- Site Number : 8400012
Richmond, Virginia 23298
United States
Investigational Site Number : 0560003
Brussels, 1020
Belgium
Investigational Site Number : 0560002
Brussels, 1070
Belgium
Investigational Site Number : 0560001
Leuven, 3000
Belgium
Hospital Santa Izabel- Site Number : 0760006
Salvador, Estado de Bahia 40050-410
Brazil
Universidade Federal de Goias- Site Number : 0760002
Goiânia, Goiás 74605-020
Brazil
Fundação Faculdade Regional de Medicina de São José do Rio Preto- Site Number : 0760001
São José do Rio Preto, São Paulo 15090-000
Brazil
Pontifícia Universidade Católica do Rio de Janeiro- Site Number : 0760009
Rio de Janeiro, 22451-900
Brazil
Hospital Samaritano De Sao Paulo- Site Number : 0760005
São Paulo, 01232-010
Brazil
Investigational Site Number : 2500002
Créteil, 94010
France
Investigational Site Number : 2500005
Marseille, 13885
France
Investigational Site Number : 2500001
Paris, 75015
France
Investigational Site Number : 2500004
Toulouse, 31100
France
Investigational Site Number : 2760002
Essen, 45147
Germany
Investigational Site Number : 2760004
Stuttgart, 70736
Germany
Investigational Site Number : 3000001
Athens, 115 27
Greece
Investigational Site Number : 3000003
Athens, 115 27
Greece
Investigational Site Number : 3000002
Pátrai, 265 04
Greece
Investigational Site Number : 3760001
Afula, 1834111
Israel
Investigational Site Number : 3760002
Afula, 1834111
Israel
Investigational Site Number : 3760005
Haifa, 3109601
Israel
Investigational Site Number : 3760006
Haifa, 3109601
Israel
Azienda Ospedaliero Universitaria Careggi SOD Ematologia-Site Number : 3800006
Florence, Firenze 50134
Italy
Investigational Site Number : 3800004
Milan, Milano 20122
Italy
Azienda Ospedaliera Universitaria, Università della Campania "Luigi Vanvitelli" Napoli-Site Number : 3800005
Naples, Napoli 80138
Italy
IRCCS Ospedale Pediatrico Bambino Gesù-Site Number : 3800001
Rome, Roma 00165
Italy
Azienda Ospedaliera Universitaria San Luigi Gonzaga, SSD Microcitemie Malattie Rare Ematologiche-Site Number : 3800007
Orbassano, Torino 10043
Italy
Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello-Site Number : 3800002
Palermo, 90146
Italy
Centro Ricerche Cliniche Verona s.r.l. presso Ospedale G.B. Rossi Borgo Roma-Site Number : 3800003
Verona, 37134
Italy
Investigational Site Number : 5280002
Rotterdam, 3015 CE
Netherlands
Investigational Site Number : 5120001
Muscat, 123
Oman
Investigational Site Number : 7240002
Madrid, 28007
Spain
Investigational Site Number : 7240001
Madrid, 28046
Spain
Investigational Site Number : 7920001
Adana, 01330
Turkey (Türkiye)
Investigational Site Number : 7920002
Adana, 01330
Turkey (Türkiye)
Investigational Site Number : 7920003
Mersin, 33330
Turkey (Türkiye)
Investigational Site Number : 8260002
London, Harrow NW10 7NS
United Kingdom
Investigational Site Number : 8260001
London, London, City of SE5 9RL
United Kingdom