AG348 in subjects with Non-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 Non Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE)
IRAS ID
298165
Contact name
Martin Besser
Contact email
Sponsor organisation
Agios Pharmaceuticals, Inc.
Eudract number
2021-000211-23
Clinicaltrials.gov Identifier
Duration of Study in the UK
6 years, 7 months, 0 days
Research summary
The purpose of this study is to find out how well the study drug mitapivaat helps to improve hemaglobin levels in people with non-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 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 171 participants will take part in this study around the world. For the double-blind period, study visits will occur approximately every 4 weeks. If allowed by participant’s doctor, and local institutional guidelines, at certain visits, he/she will have the option for at-home study visits instead of in person at the clinical study center. These at-home visits will include a visit conducted by the study staff and a visit with a nurse or home healthcare provider. After the 24-week double-blind period, participant will be provided the option to receive mitapivat for an additional 5 years. The frequency of study visits in the open-label extension period will be reduced. If permitted, certain visits may be conducted at home, in the same manner as the double-blind period. After about 2 years on mitapivat, these at-home visits will include a visit conducted by the study staff only. The following tests and procedures will be performed during the study: a complete physical examination (including weight), vital signs, an ECG, blood samples, urine sample collection, DXA scan, liver Ferriscan. Patient’s participation in the study is voluntary and he/she can decide to stop at any time and for any reason.
REC name
East of England - Cambridge East Research Ethics Committee
REC reference
21/EE/0157
Date of REC Opinion
11 Aug 2021
REC opinion
Further Information Favourable Opinion