A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period
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Phase
Phase 3
Status
Active, not recruiting
Study type
Interventional
Design
Randomized · Quadruple · Treatment
Enrollment
49actual
Sites
22
Countries
Canada, Czechia, Denmark +7
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 | 2022-06-08 | actual | When the study began enrolling |
| Primary completion | 2024-05-03 | 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 | 2021-12-03 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-07-30 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Pediatric Pyruvate Kinase Deficiency
- Pediatric Hemolytic Anemia
Interventions
- Drug: Mitapivat — also filed as AG-348, AG-348 sulfate hydrate, Mitapivat sulfate
- Drug: Mitapivat-matching placebo
Primary outcome
what the primary-completion date above is the date OFPercentage of Participants Achieving Transfusion Reduction Response (TRR)
measured Week 9 to Week 32
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