A Phase 2/3 Study in Adult and Pediatric Participants with SCD Part B and Part C
Research type
Research Study
Full title
A Phase 2/3 Randomized, Multicenter Study of Osivelotor Administered Orally to Participants with Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants with Sickle Cell Disease
IRAS ID
1008778
Contact name
Dimitrios Mandilis
Contact email
Sponsor organisation
Global Blood Therapeutics, Inc. a wholly owned subsidiary of Pfizer
ISRCTN Number
n/a
Clinicaltrials.gov Identifier
Research summary
Patients with Sickle Cell Disease (SCD) have an inherited blood disorder which causes them to have an abnormal haemoglobin (part of the red blood cells that carries oxygen), called sickle haemoglobin in their red blood cell. With this condition, red blood cells in patients have a different shape (sickle shape), which means the cells are not flexible and can stick to blood vessel walls, causing a blockage that slows or stops the flow of blook. When this happens oxygen can not reach nearby tissues leading to lower energy levels, low blood counts (anaemia) and damaged blood vessels. SCD is a lifelong illness.
Global Blood Therapeutics Inc., is developing a drug called osivelotor that could help the haemoglobin in red blood cells to keep their normal shape, so they do not to sickle. This could help better deliver oxygen to the body. The study hypothesis is that taking osivelotor is safe and tolerable and participants receiving osivelotor will demonstrate increase in haemoglobin. The Part B of the study will be evaluating the efficacy of 48 weeks treatment with 150mg dose of Osivelotor (medication) when compared to a placebo (inactive medication).
The main objective is to evaluate the efficacy of the Osivelator in patients with SCD. The secondary objective is to evaluate the Hb response (haemoglobin count).For Part B ; Participants are eligible if they are male or female and aged at 6 months and older with SCD during screening. The study is planned to open at a few sites within the United Kingdom. Genetic confirmation for sickle cell disease, this may be based on history of laboratory testing or must be confirmed by laboratory testing during Screening. Up to 332 participants for Part B and 37 participants for Part C will be enrolled in the study globally. Participants from Part C will not be eligible to enrol in Part B of the study.
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
24/NW/0134
Date of REC Opinion
10 Jun 2024
REC opinion
Further Information Favourable Opinion