Phase 23 Study of Oral Osivelotor for Treating Sickle Cell Disease in Adolescents and Adults

What this Trial Is About

Researchers are evaluating the safety, tolerability, effectiveness, and how the body processes and responds to osivelotor in people with sickle cell disease SCD. This multicenter, Phase 23 study focuses on both adults and adolescents with SCD, aiming to determine the best dose and assess the drugs effects over time. The study has three parts. Part A tests safety, tolerability, and dose-finding in adults with SCD, starting with randomization to different daily doses of osivelotor, ranging from 100 mg to potentially 200 mg, over 12 weeks. Part B compares osivelotor to placebo in adults and adolescents over 48 weeks, with adults receiving an initial 300 mg daily dose for 7 days followed by 150 mg daily, while adolescent dosing will be defined later. The Open Label Extension OLE offers long-term open-label osivelotor treatment for up to two years after Part B. Participants will be monitored throughout the study with regular visits to assess safety, blood responses, and how well they tolerate the medication. The main results will be reviewed through 12 weeks in Part A, 48 weeks in Part B, and approximately 24 months in the OLE. The study includes blood tests, monitoring of vaso-occlusive crises, and other health evaluations to understand osivelotors effects and safety over time.

A Phase 2/3 Study in Adult and Adolescent Participants With SCD