Transfusion-Dependent Alpha- or Beta-Thalassemia research hub

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β-Transfusion-Dependent Thalassemia (ENERGIZEKids-T)

This clinical study tests whether a medicine called mitapivat helps children and teenagers with thalassaemia need fewer blood transfusions. Researchers want to find out if taking this daily tablet safely cuts the amount of transfused blood they need by half or more, reducing hospital visits and iron build-up.