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

NCT06975865 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 192

Last updated 2026-05-01

No results posted yet for this study

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.

Conditions

Interventions

DRUG

Rilzabrutinib

Pharmaceutical form:Tablet -Route of administration:Oral

DRUG

Placebo

Pharmaceutical form:Tablet -Route of administration:Oral

Sponsors & Collaborators

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
10 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-08-12
Primary Completion
2027-07-23
Completion
2028-12-29
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Brazil
  • France
  • Germany
  • Ghana
  • Greece
  • Israel
  • Italy
  • Netherlands
  • Oman
  • Spain
  • Tanzania
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06975865 on ClinicalTrials.gov