SAD and MAD study of GBT440 in healthy volunteers and SCD Patients
Research type
Research Study
Full title
A Phase I randomised, placebo-controlled, double-blind, single and multiple ascending dose study of the tolerability and pharmacokinetics of GBT440 in healthy subjects and patients with Sickle Cell Disease.
IRAS ID
165047
Contact name
Tim Mant
Contact email
Sponsor organisation
Global Blood Therapeutics, Inc.
Eudract number
2014-003555-62
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 1 months, 21 days
Research summary
GBT440 is an experimental oral drug being developed for patients with Sickle Cell Disease (SCD). SCD is genetic disorder affecting haemoglobin, the major protein in red blood cells (RBCs) that carries oxygen to tissues. This makes haemoglobin clump together and damages RBCs. GBT440 binds both normal and sickle haemoglobin (HbS) making it hold oxygen more tightly. By keeping more HbS bound to oxygen GBT440 may prevent HbS from clumping thus preventing long-term adverse effects (pain, organ failure and early death). The study has 2 parts: Part A single dose (6 cohorts of healthy volunteers [HV] and 1 cohort of SCD volunteers) and part B multiple dose (3 cohorts of HVs and 2 cohorts of SCD volunteers). Outpatient visits to Quintiles Research Unit (QRU) include, in part A: 2 for HV cohorts, 8 for HVs participating in the food effect cohort; 7 for SCD cohort; in part B, 8 for 1st cohort of HVs, 15 for 2nd and 3rd cohorts of HVs and 37 for SCD cohorts.
Screening visit:
Informed consent, medical history, physical examination, ECG, urine and blood tests.
Study visit:
Part A: HVs admitted to QRU for 16 days (day-1 to day 15) or 5 days (day-1 to day 4) for SCD volunteers; oral GBT440 or placebo given on day 1.
Par B: : First HV cohort admitted to QRU for 17 days (day-1 to day 16), oral GBT440 or placebo given day 1 to day 15; 2nd and 3rd admitted to QRU for 30 days (day-1 to day 29), oral GBT440 or placebo given day 1 to 28. Two cohorts of SCD volunteers will have 2 admissions for total of 7 days (day -1 to day 4 and day 28 to day 29); oral GBT440 or placebo given day 1 to 28.REC name
London - Harrow Research Ethics Committee
REC reference
14/LO/1834
Date of REC Opinion
24 Nov 2014
REC opinion
Further Information Favourable Opinion