Rilzabrutinib for Sickle Cell Disease
(LIBRA Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called rilzabrutinib for individuals with sickle cell disease (SCD). The goal is to evaluate its effectiveness and safety for patients who frequently experience painful episodes known as vaso-occlusive crises (2 to 10 times a year). Participants should have SCD and either not be on certain other medications or be stable on them for at least three months. The trial involves taking the treatment or a placebo (a non-active substance) for a year, with regular check-ins to monitor progress. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment for SCD.
Will I have to stop taking my current medications?
If you are taking hydroxyurea or L-glutamine, you can continue them at a stable dose during the study. However, if you are taking crizanlizumab or voxelotor, you must stop them before joining the trial.
Is there any evidence suggesting that rilzabrutinib is likely to be safe for humans?
Research has shown that rilzabrutinib was safe in earlier studies. It was tested in individuals with immune thrombocytopenia (ITP), where it was well-tolerated and improved platelet levels. This suggests rilzabrutinib could be safe for humans.
Additionally, rilzabrutinib has received orphan drug status for sickle cell disease, indicating promising evidence of its safety and potential benefits. However, while these early results are encouraging, ongoing studies are essential to confirm the safety of rilzabrutinib specifically for sickle cell disease.12345Why do researchers think this study treatment might be promising for sickle cell disease?
Rilzabrutinib is unique because it targets Bruton’s tyrosine kinase (BTK), a mechanism not typically addressed by current sickle cell disease treatments. While most treatments focus on managing pain or increasing red blood cell count, Rilzabrutinib aims to reduce inflammation and improve blood flow by inhibiting BTK. This approach could potentially offer a new way to alleviate the complications of sickle cell disease, sparking excitement among researchers for its innovative action in tackling the disease's underlying issues.
What evidence suggests that rilzabrutinib might be an effective treatment for sickle cell disease?
Research suggests that rilzabrutinib, which participants in this trial may receive, may help treat sickle cell disease. Studies in mice have shown that this drug can reduce blood vessel blockages, a major issue in sickle cell disease. Rilzabrutinib also decreases inflammation and improves blood flow by reducing cell adhesion. Although limited data exist on its effects in humans with sickle cell disease, these findings indicate that rilzabrutinib might improve symptoms related to blood flow and inflammation.12567
Are You a Good Fit for This Trial?
This trial is for patients aged 10 to 65 with Sickle-cell Disease who've had 2-10 acute clinical VOC episodes in the past year. They should have an ECOG status of ≤2, use contraception, and either not be on hydroxyurea/L-glutamine or have been on a stable dose for at least 6 months.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive either rilzabrutinib or placebo during a 52-week double-blind period
Open-label extension (LTE)
Participants who complete the double-blind period may continue to receive rilzabrutinib in an open-label extension
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Rilzabrutinib
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sanofi
Lead Sponsor
Paul Hudson
Sanofi
Chief Executive Officer since 2019
Degree in Economics from Manchester Metropolitan University
Christopher Corsico
Sanofi
Chief Medical Officer
MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University