Voxelotor Brain Oxygenation and Neurocognitive Study
Research type
Research Study
Full title
An Open Label, Single Arm, Multicenter Study to Evaluate the Effect of Voxelotor on Cerebral Blood Flow and Neurocognitive Function in Adolescents and Adults with Sickle Cell Disease.
IRAS ID
1007461
Contact name
Sandrine Mwewa
Contact email
Sponsor organisation
Pfizer, Rives de Paris
Clinicaltrials.gov Identifier
Research summary
We know that blood flow in the brain is raised in sickle cell disease to compensate for the anaemia. This may mean that that blood flow in the brain is close to the maximum where no extra blood can be supplied at some points in time when it might be needed. This may leave the brain at risk of being short of oxygen for a while, which can affect thinking skills over the long term. Improving the anaemia may reduce the blood flow so that it can increase if needed. It is now possible to safely measure blood flow to the brain using MRI and a technique called near-infrared spectroscopy (NIRS) which measures changes in a light beam through the skull. This is a study to evaluate the effect of a medication called voxelotor, which reduces sickling and may improve anaemia, on blood flow in the brain and how the brain takes in and uses information. Adolescent and adult participants with sickle cell disease age 12-30 years of age receiving NHS care will be eligible. Participants will all receive the drug for about 6 months and will have MRI and NIRS scans and play iPad games as well as having blood and urine tests at the beginning, the middle and the end of the study.
This study is being done in the UK and the USA across several hospitals by a company called Global Blood Therapeutics, Inc. (GBT; Sponsor), a wholly owned subsidiary of Pfizer, Inc.REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
23/EM/0144
Date of REC Opinion
18 Sep 2023
REC opinion
Further Information Favourable Opinion