NCT07517133

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

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-Non-Transfusion-Dependent Thalassemia

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 45 (estimated)
• Sponsor: Agios Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 1 Year – 17 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Non-Transfusion-dependent Alpha-Thalassemia
• Non-Transfusion-dependent Beta-Thalassemia

Interventions

Type	Name	Description
DRUG	Mitapivat	Tablets or Granules
DRUG	Placebo Matching Mitapivat	Tablets or Granules

Timeline

• Start date: 2026-09-01
• Primary completion: 2029-03-01
• Completion: 2032-03-01
• First posted: 2026-04-08
• Last updated: 2026-04-08

Regulatory

• FDA-regulated drug study