The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
Mostrando el original en inglés
A 52-week, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group, Flexible-adaptive, Group Sequential Study to Evaluate the Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
Fase
PHASE3
Inscripción
192 (estimado)
Rango de edad
10 Years to 65 Years
Sexo
ALL
Resumen
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.
Mostrando el original en inglés
Afecciones estudiadas
Período de inscripción
12 ago 2025 – finalización estimada 23 jul 2027
Patrocinadores
Ubicaciones del estudio
University of Alabama at Birmingham- Site Number : 8400003
Birmingham, Alabama
Phoenix Children's Hospital- Site Number : 8400028
Phoenix, Arizona
University of California San Francisco- Site Number : 8400040
Fresno, California
Oncology & Hematology Associates of West Broward- Site Number : 8400029
Coral Springs, Florida
Sylvester Comprehensive Cancer Center- Site Number : 8400020
Miami, Florida
University of Illinois-Chicago - College of Medicine- Site Number : 8400054
Chicago, Illinois
Indiana University Health Riley Hospital for Children- Site Number : 8400056
Indianapolis, Indiana
Louisiana State University Health Sciences Center - Shreveport- Site Number : 8400037
Shreveport, Louisiana
University of Maryland School of Medicine - Baltimore- Site Number : 8400041
Baltimore, Maryland
University of Michigan Health System - Ann Arbor- Site Number : 8400035
Ann Arbor, Michigan
Southern Specialty Research- Site Number : 8400059
Flowood, Mississippi
Richmond University Medical Center- Site Number : 8400038
Staten Island, New York
Montefiore Medical Center - Moses Campus- Site Number : 8400057
The Bronx, New York
Prisma Health- Site Number : 8400051
Greenville, South Carolina
Baylor College of Medicine- Site Number : 8400055
Houston, Texas
VCU Massey Cancer Center: Dalton Oncology Clinic- Site Number : 8400012
Richmond, Virginia
Investigational Site Number : 0560003
Brussels
Investigational Site Number : 0560002
Brussels
Investigational Site Number : 0560001
Leuven
Hospital Santa Izabel- Site Number : 0760006
Salvador, Estado de Bahia
Universidade Federal de Goias- Site Number : 0760002
Goiânia, Goiás
Fundação Faculdade Regional de Medicina de São José do Rio Preto- Site Number : 0760001
São José do Rio Preto, São Paulo
Pontifícia Universidade Católica do Rio de Janeiro- Site Number : 0760009
Rio de Janeiro
Hospital Samaritano De Sao Paulo- Site Number : 0760005
São Paulo
Investigational Site Number : 2500002
Créteil
Investigational Site Number : 2500005
Marseille
Investigational Site Number : 2500001
Paris
Investigational Site Number : 2500004
Toulouse
Investigational Site Number : 2760002
Essen
Investigational Site Number : 2760004
Stuttgart
Investigational Site Number : 3000001
Athens
Investigational Site Number : 3000003
Athens
Investigational Site Number : 3000002
Pátrai
Investigational Site Number : 3760001
Afula
Investigational Site Number : 3760002
Afula
Investigational Site Number : 3760005
Haifa
Investigational Site Number : 3760006
Haifa
Azienda Ospedaliero Universitaria Careggi SOD Ematologia-Site Number : 3800006
Florence, Firenze
Investigational Site Number : 3800004
Milan, Milano
Azienda Ospedaliera Universitaria, Università della Campania "Luigi Vanvitelli" Napoli-Site Number : 3800005
Naples, Napoli
IRCCS Ospedale Pediatrico Bambino Gesù-Site Number : 3800001
Rome, Roma
Azienda Ospedaliera Universitaria San Luigi Gonzaga, SSD Microcitemie Malattie Rare Ematologiche-Site Number : 3800007
Orbassano, Torino
Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello-Site Number : 3800002
Palermo
Centro Ricerche Cliniche Verona s.r.l. presso Ospedale G.B. Rossi Borgo Roma-Site Number : 3800003
Verona
Investigational Site Number : 5280002
Rotterdam
Investigational Site Number : 5120001
Muscat
Investigational Site Number : 7240002
Madrid
Investigational Site Number : 7240001
Madrid
Investigational Site Number : 7920001
Adana
Investigational Site Number : 7920002
Adana
Investigational Site Number : 7920003
Mersin
Investigational Site Number : 8260002
London, Harrow
Investigational Site Number : 8260001
London, London, City of