GBT440-034 - OLE GBT440 in Patients with Sickle Cell Disease
Research type
Research Study
Full title
An Open Label Extension Study of GBT440 Administered Orally to Patients with Sickle Cell Disease Who Have Participated in GBT440 Clinical Trials
IRAS ID
240315
Contact name
Rachel Kesse-Adu
Contact email
Sponsor organisation
Global Blood Therapeutics, Inc.
Eudract number
2017-004045-25
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
121,691, IND
Duration of Study in the UK
1 years, 10 months, 1 days
Research summary
The main purpose of this study is to assess the effect of safety and efficacy of long-term treatment of Sickle Cell Disease (SCD) with GBT440 (voxelotor, study drug) in patients who have participated in the GBT440-031 (HOPE) study. All participants in GBT440-031, dependent on their Group randomisation, who have completed treatment (per 031 protocol criteria) are eligible for this study. All participants will receive the active drug. This is an open-label study. Tablets will be administered orally to all participants, once daily in the amount equal to the selected dose(s) for the GBT440-031 study.
Participants will initially visit the clinic every 12 weeks for study visits, plus two possible additional visits at Week 4 and Week 8. If participants continue the study beyond 48 weeks, they will have visits every 12 weeks to receive study drug and every 24 weeks for study assessments.
Participants may receive the study drug as long they continue to receive clinical benefit, which outweighs risk as determined by the Investigator and/or until the participant has access to GBT440 from an alternative source (i.e., commercialisation or through a managed access program).REC name
London - Central Research Ethics Committee
REC reference
18/LO/0235
Date of REC Opinion
29 Mar 2018
REC opinion
Further Information Favourable Opinion