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Study identifier: NCT07506863 Synced from ClinicalTrials.gov · August 03, 2026
● 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)

Condition: Transfusion-dependent Alpha-Thalassemia · Transfusion-dependent Beta-Thalassemia  ·  Sponsor: Agios Pharmaceuticals, Inc.

PhasePhase 3
Planned participants54
Who can joinAll sexes, 1 Year to 17 Years
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Agios Medical Affairs  ·  833-228-8474  ·  medinfo@agios.com

Always discuss trial participation with your own doctor first.

Locations (1)

University of Texas Southwestern Medical CenterDallas, Texas, United StatesRecruiting

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