U.S., April 3 -- ClinicalTrials.gov registry received information related to the study (NCT07506863) titled '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)' on March 26.
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.
Study Start Date: Sept., 2026
Study Type: INTERVENTIONAL
Condition:
Transfusion-dependent Alpha-Thalassemia
Transfusion-dependent Beta-Thalassemia
Intervention:
DRUG: Mitapivat Matched Placebo
Tablets or Granules
DRUG: Mitapi...