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)

Recruiting Phase 3 Interventional Study
Transfusion-dependent Alpha-Thalassemia Transfusion-dependent Beta-Thalassemia
Pivotal Trial This treatment is in the last trial phase before FDA approval. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
1 – 17
Sex
Any
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
54 (estimated)
Sponsor
Agios Pharmaceuticals, Inc. · Industry
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About This Trial

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.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Written informed consent/assent from the participant (or their legally authorized representative, parent(s), or legal guardian) must be obtained before any study-related procedures are conducted and participants must be willing to comply with all study procedures for the durat…
Contacts

Agios Medical Affairs

833-228-8474

medinfo@agios.com

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