AG348 in subjects with transfusion dependent A/B Thalasseima
Research type
Research Study
Full title
A Phase 3, Double-blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE-T)
IRAS ID
298166
Contact name
Martin Besser
Contact email
Sponsor organisation
Agios Pharmaceuticals, Inc.
Eudract number
2021-000212-34
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
7 years, 0 months, 1 days
Research summary
The purpose of the study is to find out how well the study drug mitapivat helps to reduce the need for transfusion in people with transfusion dependent alpha- or beta-thalassemia and how safe it is compared to a placebo. Thalassemia is a blood disorder that affects and destroys the red blood cells that carry oxygen, mitapivat may help the body to maintain normal red blood cell levels. The use of mitapivat in this study is experimental, which means that mitapivat is not yet approved for use by any authorities.
Mitapivat is a tablet for oral administration.
Approximately 240 people will take part in the study around the world. Participants will be randomly assigned in a 2:1 ratio to receive either mitapivat or placebo (a tablet that looks like the study drug but does not contain the study drug), you or the doctors will not know whether you are receiving mitapivat or placebo Approximately 160 participants will receive mitapivat and 80 will receive placebo. Participants will receive either mitapivat or placebo for 48 weeks, after 48 weeks all participants will be given the option to receive mitapivat for an additional 5 years. The total length of study participation is approximately 6 years. For the first 48 weeks, participants will need to attend hospital site visits approximately every 4 weeks which will be aligned where possible to match their transfusion schedule. After 48 weeks, for those choosing to receive mitapivat for the additional 5 years, hospital site visit frequency will be reduced and where possible there may be the option of home visits.
All participants will undergo a number of tests during the study including blood and sample tests, physical examinations, MRI and DXA scans and ECG.Taking part in the study is entirely voluntary.
REC name
East of England - Cambridge East Research Ethics Committee
REC reference
21/EE/0158
Date of REC Opinion
11 Aug 2021
REC opinion
Further Information Favourable Opinion