clinical trial · NCT07506863
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)
Transfusion-dependent Alpha-ThalassemiaTransfusion-dependent Beta-ThalassemiaMitapivat Matched PlaceboMitapivat
brief 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.
started
Sep 1, 2026
primary completion
Jun 1, 2029
completion
Jun 1, 2032
last updated
May 29, 2026
official title
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
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol