Overview
Open-label extension study of voxelotor for participants with Sickle Cell Disease who have participated in voxelotor clinical trials.
Description
Open-label extension (OLE) study of voxelotor for participants with Sickle Cell Disease who have participated in voxelotor clinical trials. Up to approximately 600 participants with sickle cell disease (SCD), will be enrolled at approximately 70 clinical sites globally. All participants will receive voxelotor once daily, administered orally as tablets, dispersible tablets, or powder for oral suspension formulation. The objective of this OLE is to assess the safety of, and SCD-related complications of, long-term treatment with voxelotor, in participants who have completed treatment in a Global Blood Therapeutics (GBT)-sponsored voxelotor clinical study.
Eligibility
Inclusion Criteria:
- Male or female participant with SCD, who participated and received study drug in a GBT-sponsored voxelotor clinical study.
Note: Participants who discontinued study drug due to an AE, but who remained on study, may
be eligible for treatment in this study provided the AE does not pose a risk for treatment
with voxelotor.
Note: Participants who discontinued Study GBT440-032 as the result of an abnormal
transcranial Doppler (TCD) flow velocity assessment (≥ 200 cm/sec) are eligible for
treatment in this study.
- Participant has provided written consent/assent (for pediatric participants, both the
consent of the participant's legal representative or legal guardian and the participant's
assent [where applicable] must be obtained).
Exclusion Criteria:
- Female participant who is breastfeeding or pregnant
- Participant withdrew consent from a GBT-sponsored voxelotor clinical study
- Known hypersensitivity to voxelotor or any other components of the study drug
- Use of St. John's wort, sensitive cytochrome P450 (CYP) 3A4 substrates with a narrow
therapeutic index, or moderate or strong CYP3A4 inducers within 30 days of Day 1