The Efficacy and Safety of Rilzabrutinib in Patients Aged 10 to 65 Years With Sickle-cell Disease
Phase 3
192
about 3.4 years
10–65
14 sites in AL, AZ, CA +10
What this study is about
Researchers are testing a treatment called rilzabrutinib for people with sickle cell disease. The trial will last about 1235 days, starting with a double-blind period of 52 weeks followed by an open-label extension period. Participants who complete the double-blind phase are eligible to continue in the open-label extension.
Simplified from trial records by PatientMatch.
What you may be asked to do
- 1.Take Placebo
- 2.Take Rilzabrutinib
Participation Burden
What's physically and logistically required of participants.
Requires travel to a study site
How treatment is administered
You may get a placebo/standard care, and you won't know which.
Extracted study details
Pulled from the trial record to show what is being tested and what the study is measuring.
kinase inhibitor
oral (Oral Tablet)
Secondary: Annualized rate of home-managed VOCs as reported in the Sickle Cell Pain Crisis (SCPC) eDiary, Change in fatigue as measured by the PROMIS SF v1.0 Fatigue 13a total score (adults), Change in fatigue as measured by the PedsQL Multidimensional Fatigue Scale total score (pediatric participants), Incidence of treatment emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs) and adverse events leading to discontinuation