Dose Escalation Study to Evaluate the Safety, Tolerability, PK and PD of Voxelotor in Patients With SCD
Status:
Recruiting
Trial end date:
2021-12-01
Target enrollment:
Participant gender:
Summary
This is a dose escalation study to evaluate the safety and tolerability of voxelotor at daily
doses of 1500 mg to 3000 mg in participants with sickle cell disease (SCD). The study is
designed to include 2 separate cohorts. In Cohort A participants will undergo up to 4 periods
of voxelotor administration at progressively higher dose levels from 1500 mg until either a
maximum tolerated dose (MTD) or 3000 mg orally daily is reached. Cohort B (following Cohort
A) will assess the tolerability and safety of doses higher than 1500 mg administered without
up-titration.