The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
Sanofi
Study ID
NCT06975865
Phase
PHASE3
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
10 Years - 65 Years
Healthy Volunteers
Not accepted

Interventions

  • Rilzabrutinib — DRUG
    Pharmaceutical form:Tablet -Route of administration:Oral
  • Placebo — DRUG
    Pharmaceutical form:Tablet -Route of administration:Oral

Study Details

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.

Key Dates

Start date
Aug 12, 2025
Status verified
Apr 2026
Primary completion
Jul 23, 2027
Completion
Dec 29, 2028

Study Design

Enrollment
192 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Rilzabrutinib
    Rilzabrutinib
  • Placebo Comparator: Placebo
    Placebo

Primary Outcome Measure

Annualized rate of clinical VOC [ Time Frame: At Week 52 ]

Central Contacts

  • Trial Transparency email recommended (Toll free for US & Canada)
    800-633-1610
    [email protected]

Locations (14)

FacilityCityStateZIPSite coordinators
University of Alabama at Birmingham- Site Number : 8400003BirminghamAlabama35233-
Phoenix Children's Hospital- Site Number : 8400028PhoenixArizona85016-
University of California San Francisco- Site Number : 8400040FresnoCalifornia93701-
Oncology & Hematology Associates of West Broward- Site Number : 8400029Coral SpringsFlorida33065-
Sylvester Comprehensive Cancer Center- Site Number : 8400020MiamiFlorida33136-
University of Illinois-Chicago - College of Medicine- Site Number : 8400054ChicagoIllinois60612-
Indiana University Health Riley Hospital for Children- Site Number : 8400056IndianapolisIndiana46202-
Louisiana State University Health Sciences Center - Shreveport- Site Number : 8400037ShreveportLouisiana71130-
University of Maryland School of Medicine - Baltimore- Site Number : 8400041BaltimoreMaryland21201-
University of Michigan Health System - Ann Arbor- Site Number : 8400035Ann ArborMichigan48109-
Southern Specialty Research- Site Number : 8400059FlowoodMississippi39232-
Richmond University Medical Center- Site Number : 8400038Staten IslandNew York10310-
Baylor College of Medicine- Site Number : 8400055HoustonTexas77030-
VCU Massey Cancer Center: Dalton Oncology Clinic- Site Number : 8400012RichmondVirginia23298-

Find similar trials in Birmingham, AL

By condition
Sickle cell disease
By research site
University of Alabama at Birmingham· Birmingham, ALPhoenix Children's Hospital· Phoenix, AZUniversity of California San Francisco· Fresno, CAOncology & Hematology Associates of West Broward· Coral Springs, FLSylvester Comprehensive Cancer Center· Miami, FLUniversity of Illinois-Chicago - College of Medicine· Chicago, IL

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