Open Label Extension Study with SCD Patients from GBT440-001
Research type
Research Study
Full title
An open label single arm extension study to further evaluate the safety, tolerability and treatment response of GBT440 in patients with Sickle Cell Disease who participated in Phase 1 Study GBT440-001
IRAS ID
208060
Contact name
Timothy Mant
Contact email
Sponsor organisation
Global Blood Therapeutics, Inc.
Eudract number
2016-002123-38
Duration of Study in the UK
0 years, 8 months, 31 days
Research summary
GBT440 is a drug that may be useful in the treatment of Sickle Cell Disease. It works by making haemoglobin (the protein that carries oxygen in red blood cells) bind to oxygen more tightly. This has an effect on oxygen levels, and even more important, it is thought that the GBT440 bound to sickle haemoglobin will not clump and will prevent sickling.
To date GBT440 has been generally well tolerated in patients with SCD for up to 90 days. Given the significant unmet need in SCD, there is an interest in determining the potential for further development of GBT440 by generating safety and efficacy data with longer term dosing (greater than 90 days) as quickly as possible while still safeguarding the safety of subjects. To do this safely and efficiently only those subjects in ongoing cohorts 17 and 15 in the GBT440-001 study will be enrolled in this proposed extension study.
This study involves 4 groups as follows:
Group 1
Patients from Cohort 17 in the GBT440-001 study who have received GBT440 900mg per day for 4 months will continue receiving GBT440 900mg per day for a further 2 months in this study.Group 2
Patients from Cohort 17 in the GBT440-001 study who have received placebo for 4 months will receive GBT440 900mg per day for 6 months in this study.Group 3
Patients from Cohort 15 in the GBT440-001 study who have received GBT440 600mg per day for two months will continue receiving GBT440 600mg for a further 4 months in this study.Group 4
Patients from Cohort 15 in the GBT440-001 study who have received placebo for 2 months will receive GBT440 600mg per day for 6 months in this study.All Subjects will receive a total of 6 months treatment with GBT440 which will include treatment with GBT440 in the GBT440-001 study.
REC name
London - Harrow Research Ethics Committee
REC reference
16/LO/1047
Date of REC Opinion
7 Jul 2016
REC opinion
Further Information Favourable Opinion