A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia

Phase 3 Not yet recruiting 45 enrolled Agios Pharmaceuticals, Inc.

RandomizedParallel-groupQuadruple-blindTreatment

Summary

The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusion-dependent thalassemia.

Timeline

Start: 2026-09
Primary completion: 2029-03
Completion: 2032-03

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Mitapivat	Small molecule	—	Oral

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia

Summary

Timeline

Drugs

Indications