A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
← catalyst calendarSponsored by Agios Pharmaceuticals, Inc. (industry) · AGIO — their whole pipeline →.
Phase
Phase 3
Status
Active, not recruiting
Study type
Interventional
Design
Randomized · Quadruple · Treatment
Enrollment
258actual
Sites
75
Countries
Brazil, Bulgaria, Canada +16
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 | 2021-11-30 | actual | When the study began enrolling |
| Primary completion | 2024-04-11 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Jun 2029 | estimated | The whole study's end, after follow-up |
| First posted | 2021-02-25 | actual | When this record first appeared on the registry |
| Results posted | 2025-05-25 | actual | When the sponsor posted results to the registry |
| Record updated | 2026-07-31 | 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: Placebo Matching Mitapivat
- Drug: Mitapivat — also filed as AG-348, AG-348 sulfate hydrate, Mitapivat sulfate
Primary outcome
what the primary-completion date above is the date OFDouble-blind Period: Percentage of Participants Who Achieved Transfusion Reduction Response (TRR)
measured Double-blind Period: Baseline through Week 48
Posted documents
hosted by the registry — we link, never re-serveStudy Protocolposted 2022-09-28 · 934 KB · Prot_000.pdf
Statistical Analysis Planposted 2022-11-14 · 609 KB · SAP_001.pdf
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