clinical trial · NCT07517133
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
Non-Transfusion-dependent Alpha-ThalassemiaNon-Transfusion-dependent Beta-ThalassemiaMitapivatPlacebo Matching Mitapivat
brief summary
The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusion-dependent thalassemia.
started
Sep 1, 2026
primary completion
Mar 1, 2029
completion
Mar 1, 2032
last updated
Apr 8, 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-Non-Transfusion-Dependent Thalassemia
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol