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
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
- DrugMitapivat Matched Placebo
Tablets or Granules
- DrugMitapivat
Tablets or Granules
Location
- University of Texas Southwestern Medical CenterDallas, Texas