Clinical Trial

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

Active, Not Recruiting Phase 3
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Summary
The primary purpose of this study was to compare the effect of mitapivat versus placebo on hemolytic anemia in participants with alpha- or beta-non-transfusion dependent thalassemia (NTDT).
Protocol Amendment History 51 amendments
This ClinicalTrials.gov record has been amended 51 times since 2021-02-22; most recent amendment 2026-06-19.
Status change: Recruiting → Active, Not Recruiting 2023-05-19
Status change: Not Yet Recruiting → Recruiting 2021-10-08
Trial Details
NCT Number NCT04770753
Lead Sponsor Agios Pharmaceuticals, Inc.
Conditions Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
Enrollment 194 participants
Start Date 2021-12-20
Primary Completion 2023-11-13 (estimated)
Study Completion 2028-12 (estimated)
Updated on ClinicalTrials.gov 2026-07-31