Study to Evaluate Mitapivat in Patients with Sickle Cell Disease
Research type
Research Study
Full title
A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease.
IRAS ID
303180
Contact name
Baba Inusa
Contact email
Sponsor organisation
Agios Pharmaceuticals, Inc.
Eudract number
2021-001674-34
Clinicaltrials.gov Identifier
Duration of Study in the UK
9 years, 1 months, 15 days
Research summary
Sickle cell disease (SCD) is a serious and life-threatening genetic disorder where the red blood cells, which carry oxygen around the body, develop abnormally and become rigid and shaped like a crescent (sickle shape). These cells die early, leaving a shortage of healthy red blood cells (anaemia) and block the flow of oxygen through the body resulting in pain, fatigue, strokes, organ damage, and other serious complications.
The overall purpose of this study is to determine the safety and effectiveness of mitapivat in patients with SCD. Mitapivat is a drug that attaches to a specific protein (the red blood cell specific pyruvate kinase (PKR) protein) that may give red blood cells more energy. This may help the red blood cells live longer, may help stop the process that causes red blood cells to sickle, and may help the body to maintain normal red blood cell levels.
The study consists of two portions (Phase 2 and Phase 3). The purpose of Phase 2 is to select the dose to be evaluated in Phase 3 and assess the safety of mitapivat in SCD. The purpose of Phase 3 is to evaluate if mitapivat reduces sickle cell pain crises and/or improves anemia. Participants will be randomised to either receive mitapivat or placebo during a Double-blind period (neither the participants or doctor knows which treatment a participant is receiving) for 12 weeks (Phase 2) or 52 weeks (Phase 3). Following this, participants will have the option to receive mitapivat in the Open-label Extension Period for up to 216 weeks.Approximately 267 participants (69 in Phase 2 and 198 in Phase 3) are planned for this study and can participate for up to 4.5 (Phase 2) or 5.3 years (Phase 3). Approximately 15 participants across 6 sites in the UK are planned to be enrolled.
REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
22/EM/0020
Date of REC Opinion
17 Mar 2022
REC opinion
Further Information Favourable Opinion