A 52-week, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group, Group Sequential Study to Evaluate the Efficacy and Safety of Rilzabrutinib in Patients Aged 10 to 65 Years With Sickle-cell Disease
Status: Recruiting
Location: See all (34) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, 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
Participation Requirements
Sex: All
Minimum Age: 10
Maximum Age: 65
Healthy Volunteers: f
View:
• Participants who have been diagnosed with SCD.
• Participants who have had between ≥2 to ≤10 episodes of documented acute clinical VOC within 12 months of the screening visit.
• Participants who are either not on hydroxyurea and/or L-glutamine at the Screening Visit and does not plan to receive them during the course of the study or has received HU and/or L-glutamine for a minimum of 6 months. Participants on hydroxyurea and/or L-glutamine must have been on a stable weight-based dose level (mg/kg) for at least 3 months prior to the Screening Visit, with the intent to continue at the same weight-based dose level for the duration of the study, except for safety reasons.
• Participants with Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower.
• Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
• For participants ≥10 to \<18 years of age: the parent(s)/legal guardian(s) must provide written informed consent prior to any study-related procedures being performed.
Locations
United States
Alabama
University of Alabama at Birmingham- Site Number : 8400003
RECRUITING
Birmingham
Florida
Oncology & Hematology Associates of West Broward- Site Number : 8400029
RECRUITING
Coral Springs
Louisiana
LSU Health Sciences Center- Site Number : 8400037
RECRUITING
Shreveport
New York
Richmond University Medical Center- Site Number : 8400038
RECRUITING
Staten Island
Virginia
Virginia Commonwealth University- Site Number : 8400012
RECRUITING
Richmond
Other Locations
Belgium
Investigational Site Number : 0560002
RECRUITING
Brussels
Investigational Site Number : 0560003
RECRUITING
Brussels
Brazil
Universidade Federal de Goias- Site Number : 0760002
RECRUITING
Goiânia
Universidade Federal da Bahia - Site Number : 0760009
RECRUITING
Salvador
Fundação Faculdade Regional de Medicina de São José do Rio Preto- Site Number : 0760001
RECRUITING
São José Do Rio Preto
Hospital Samaritano De Sao Paulo- Site Number : 0760005
RECRUITING
São Paulo
France
Investigational Site Number : 2500002
RECRUITING
Créteil
Investigational Site Number : 2500005
RECRUITING
Marseille
Investigational Site Number : 2500004
RECRUITING
Toulouse
Ghana
Investigational Site Number : 2880004
ACTIVE_NOT_RECRUITING
Kintampo
Investigational Site Number : 2880002
ACTIVE_NOT_RECRUITING
Navrongo
Greece
Investigational Site Number : 3000001
RECRUITING
Athens
Investigational Site Number : 3000003
RECRUITING
Athens
Israel
Investigational Site Number : 3760002
RECRUITING
Afula
Investigational Site Number : 3760005
RECRUITING
Haifa
Investigational Site Number : 3760006
RECRUITING
Haifa
Italy
Investigational Site Number : 3800004
RECRUITING
Milan
Investigational Site Number : 3800007
RECRUITING
Orbassano
Investigational Site Number : 3800001
RECRUITING
Roma
Investigational Site Number : 3800003
RECRUITING
Verona
Netherlands
Investigational Site Number : 5280002
RECRUITING
Rotterdam
Spain
Investigational Site Number : 7240001
RECRUITING
Madrid
Investigational Site Number : 7240002
RECRUITING
Madrid
Turkey
Investigational Site Number : 7920001
RECRUITING
Adana
Investigational Site Number : 7920002
RECRUITING
Adana
Investigational Site Number : 7920003
RECRUITING
Mersin
United Kingdom
Investigational Site Number : 8260001
RECRUITING
London
Investigational Site Number : 8260002
RECRUITING
London
United Republic of Tanzania
Investigational Site Number : 8340003
ACTIVE_NOT_RECRUITING
Mwanza
Contact Information
Primary
Trial Transparency email recommended (Toll free for US & Canada)