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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
192
Pharmaceutical form:Tablet -Route of administration:Oral
Pharmaceutical form:Tablet -Route of administration:Oral
University of Alabama at Birmingham- Site Number : 8400003
Birmingham, Alabama, United States
RECRUITINGPhoenix Children's Hospital- Site Number : 8400028
Phoenix, Arizona, United States
RECRUITINGUniversity of California San Francisco- Site Number : 8400040
Fresno, California, United States
RECRUITINGOncology & Hematology Associates of West Broward- Site Number : 8400029
Coral Springs, Florida, United States
Annualized rate of clinical VOC
Calculated from total number of clinical VOC incidents and total number of days during the observation period
Time frame: At Week 52
Time to first clinical VOC incidence
Time frame: Until Week 52
Annualized rate of visits due to SCD-related complications as assessed by the Investigator
Time frame: At Week 52
Annualized rate of home-managed VOCs as reported in the Sickle Cell Pain Crisis (SCPC) eDiary
Time frame: At Week 52
Change in fatigue as measured by the PROMIS SF v1.0 Fatigue 13a total score (adults)
Time frame: From baseline to Week 52
Change in Hb levels
Time frame: From baseline to Week 52
Change in fatigue as measured by the PedsQL Multidimensional Fatigue Scale total score (pediatric participants)
Time frame: From baseline to Week 52
Incidence of treatment emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs) and adverse events leading to discontinuation
Time frame: Until Week 52
Incidence of potentially clinically significant laboratory, vital signs, and ECG abnormalities
Time frame: Until Week 52
Absolute number of simple and exchange blood transfusion
Time frame: Until Week 52
Number of days requiring acetaminophen, NSAID and/or short-acting opioid usage
Time frame: Until Week 52
Trial Transparency email recommended (Toll free for US & Canada)
CONTACT
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Sylvester Comprehensive Cancer Center- Site Number : 8400020
Miami, Florida, United States
RECRUITINGUniversity of Illinois-Chicago - College of Medicine- Site Number : 8400054
Chicago, Illinois, United States
RECRUITINGIndiana University Health Riley Hospital for Children- Site Number : 8400056
Indianapolis, Indiana, United States
RECRUITINGLouisiana State University Health Sciences Center - Shreveport- Site Number : 8400037
Shreveport, Louisiana, United States
RECRUITINGUniversity of Maryland School of Medicine - Baltimore- Site Number : 8400041
Baltimore, Maryland, United States
RECRUITINGUniversity of Michigan Health System - Ann Arbor- Site Number : 8400035
Ann Arbor, Michigan, United States
RECRUITING...and 43 more locations