Haematological Disorders Sickle Cell Disease
Conditions
Interventions
Sponsors
Global Blood Therapeutics inc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: A. Participants who meet all of the following criteria will be eligible for study enrollment: 1. 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. 2. Female participants of childbearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1. Note: Female participants who become childbearing during the study must be willing to have a negative urine pregnancy test to remain in the study. 3. If sexually active, female participants of childbearing potential must use highly effective methods of contraception until 30 days after the last dose of study drug. If sexually active, male participants must use barrier methods of contraception until 30 days after the last dose of study drug. 4. 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
Exclusion criteria: 1. Female participant who is breastfeeding or pregnant. 2. Participant withdrew consent from a GBT-sponsored voxelotor clinical study. 3. Participant was lost to follow-up from a GBT-sponsored voxelotor clinical study. 4. Participant has any medical, psychological, safety, or behavioral conditions that, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent. 5. Active symptomatic coronavirus disease of 2019 (COVID-19) infection. 6. Known hypersensitivity to voxelotor or any other components of the study drug. 7. 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. TEAEs and SAEs [ Time Frame: Throughout entire study ] Treatment Emergent Adverse Events and Serious Adverse Events 2. Sickle Cell Disease-Related Complications [ Time Frame: Throughout entire study ] Frequency of SCD-related complications | — |
Secondary
| Measure | Time frame |
|---|---|
| Not Provided | — |
Countries
Ghana, Kenya, Nigeria
Contacts
Public ContactAimee Enriquez
Senior Clinical Trial Manager. Global Blood Therapeutics
Outcome results
None listed