NCT07517133Phase 3Recruiting

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia

Study sponsor: Agios Pharmaceuticals, Inc. · AGIO

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

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Primary completion2029-03Estimated

Enrollment · participants45Estimated

Source updated2026-09-28Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

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Posted 2026-08-03 · 1 field changed

Recruitment status changed

− Before

~~Not yet recruiting~~

+ After

Recruiting

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Study details

Conditions studied

Non-Transfusion-dependent Alpha-Thalassemia; Non-Transfusion-dependent Beta-Thalassemia

Treatments or interventions studied

MitapivatDrugAlso known as AG-348, Mitapivat sulfate
Placebo Matching MitapivatDrug

Primary outcomes · main study measures

measure: Percentage of Participants Who Achieved a Hemoglobin (Hb) Response; description: Hb response is defined as a ≥1.0 grams per deciliter (g/dL) increase in average Hb concentration from Week 12 through Week 24 compared with baseline.; timeFrame: Baseline, Week 12 through Week 24

History coverage

Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.

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