A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
← 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
194actual
Sites
68
Countries
Brazil, Bulgaria, Canada +15
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 | 2021-12-20 | actual | When the study began enrolling |
| Primary completion | 2023-11-13 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Dec 2028 | 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 | 2025-01-24 | 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
- Non-Transfusion-dependent Alpha-Thalassemia
- Non-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 Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With Baseline
measured Double-Blind Period: Baseline up to Week 12 through Week 24
Posted documents
hosted by the registry — we link, never re-serveStudy Protocolposted 2024-08-05 · 946 KB · Prot_000.pdf
Statistical Analysis Planposted 2022-11-14 · 621 KB · SAP_001.pdf
Publications
- PMID 40544857 — linked by the registry
Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.
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