Safety of voxelotor with long-term treatment in pediatric population
Research type
Research Study
Full title
An Open-Label Extension Study of Voxelotor Administered Orally to Pediatric Participants with Sickle Cell Disease Who Have articipated in Voxelotor Clinical Trials
IRAS ID
273172
Contact name
Paul Telfer
Contact email
Sponsor organisation
Global Blood Therapeutics, Inc.
Eudract number
2019-003144-76
Clinicaltrials.gov Identifier
121, 691, IND Number
Duration of Study in the UK
5 years, 8 months, 15 days
Research summary
The purpose of this study is to learn more about how safe voxelotor is to take and to see how it works in young people with sickle cell disease, an inherited blood disorder. The study will assess the continued treatment with voxelotor in participants who have received it for 48 weeks on GBT440-007 HOPE Kids 1 study. The main side effects that people experienced in the GBT440-007 included diarrhea, headache, upset stomach, vomiting and rash.
People with sickle cell disease have abnormal hemoglobin (the part of your red blood cells that carries oxygen), called sickle hemoglobin in their red blood cells. Red blood cells that contain normal hemoglobin are disc shaped. This shape allows the cells to be flexible so that they can move through large and small blood vessels to deliver oxygen. Sickle hemoglobin can form stiff rods within the red cell, changing it into a crescent or “sickle” shape. Sickle-shaped cells are not flexible and can stick to vessel walls, causing a blockage that slows or stops the flow of blood. When this happens, oxygen can’t reach nearby tissues. In addition, the stiff rods of sickle hemoglobin damage red blood cells, causing them to be destroyed faster than normal. This causes low blood counts (anemia) which can cause lower energy levels and also can damage blood vessels leading to problems like strokes and leg ulcers.
Approximately 50 participants at about 20 study sites in the US, England, and Lebanon may enroll into this study. This study is open to male and female participants, aged 4 to 18.
The study drug will be provided as tablets. The participants will be invited to the visits where they will be asked questions about their health, the examinations will be performed (vital signs, physical examination) and blood and urine sample will be taken.
The study will be continued until the study drug is available commercially in UK.REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
19/LO/1764
Date of REC Opinion
10 Jan 2020
REC opinion
Further Information Favourable Opinion