A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
← catalyst calendarNCT07517133 · readout ≤ 955 d
Sponsored by Agios Pharmaceuticals, Inc. (industry) · AGIO — their whole pipeline →.
Phase
Phase 3
Status
Recruiting
Study type
Interventional
Design
Randomized · Quadruple · Treatment
Enrollment
45estimated
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 | Mar 2029 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Mar 2032 | estimated | The whole study's end, after follow-up |
| First posted | 2026-04-08 | 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
- Non-Transfusion-dependent Alpha-Thalassemia
- Non-Transfusion-dependent Beta-Thalassemia
Interventions
- Drug: Mitapivat — also filed as AG-348, Mitapivat sulfate
- Drug: Placebo Matching Mitapivat
Primary outcome
what the primary-completion date above is the date OFPercentage of Participants Who Achieved a Hemoglobin (Hb) Response
measured Baseline, Week 12 through Week 24
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