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)
← catalyst calendarNCT07506863 · readout ≤ 1,046 d
Sponsored by Agios Pharmaceuticals, Inc. (industry) · AGIO — their whole pipeline →.
Phase
Phase 3
Status
Recruiting
Study type
Interventional
Design
Randomized · Quadruple · Treatment
Enrollment
54estimated
Sites
1
Country
United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | Sep 2026 | estimated | When the study began enrolling |
| Primary completion | Jun 2029 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Jun 2032 | estimated | The whole study's end, after follow-up |
| First posted | 2026-04-02 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-08-03 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Transfusion-dependent Alpha-Thalassemia
- Transfusion-dependent Beta-Thalassemia
Interventions
- Drug: Mitapivat Matched Placebo
- Drug: Mitapivat — also filed as AG-348, Mitapivat sulfate
Primary outcome
what the primary-completion date above is the date OFPercentage of Participants Who Achieved Transfusion Reduction Response (TRR) Through Week 48
measured Baseline, through Week 48
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