Status: Not yet recruiting
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
The primary objective of this study is to compare the effect of mitapivat versus placebo on transfusion burden in pediatric participants with α- or β-transfusion-dependent thalassemia.
Participants needed: 54
Trial details
Phase: Phase 3Age: 1-17Biological sex: AllType: InterventionalSponsor: Agios Pharmaceuticals, Inc.Updated: Jun 23, 2026
Eligibility criteria
Written informed consent/assent from the participant (or their legally authorize... [+5]
Pregnant or breastfeeding. [+23]