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- Transfusion-Dependent Thalassemia

Agios Pharmaceuticals, Inc.

Summary

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.

Eligibility

Age range: 1–17 years
Sex: All
Healthy volunteers: No

Detailed 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 duration of the study. * Aged 1 to \<18 years and weighing at least 7 kilograms (kg) at the time of providing informed consent/assent. * Documented diagnosis of thalassemia (β-thalassemia with or without α-globin gene mutations, hemoglobin E (HbE)/β-thalassemia, or α-thalassemia/hemoglobin H (HbH) diseas…

Interventions

Drug
Mitapivat Matched Placebo
Tablets or Granules
Drug
Mitapivat
Tablets or Granules

Locations (3)

Weill Cornell Medical College/ New York Presbyterian
New York, New York
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania
University of Texas Southwestern Medical Center
Dallas, Texas

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- Transfusion-Dependent Thalassemia

Summary

Eligibility

Interventions

Locations (3)

Compensation

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Details

Conditions