NCT07506863Phase 3Recruiting

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

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 completion2029-06Estimated

Enrollment · participants54Estimated

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

Version history

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Last 12 months

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Posted 2026-08-03 · 1 field changed

Recruitment status

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Study details

Conditions studied

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

Treatments or interventions studied

Mitapivat Matched PlaceboDrug
MitapivatDrugAlso known as AG-348, Mitapivat sulfate

Primary outcomes · main study measures

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measure: Percentage of Participants Who Achieved Transfusion Reduction Response (TRR) Through Week 48; description: TRR is defined as ≥50 percent (%) reduction in transfused red blood cells (RBC) volume (normalized by weight) in any consecutive 12-week period through Week 48 compared with historical RBC transfusion volume (normalized by weight) and standardized to 12 weeks.; timeFrame: Baseline, through Week 48

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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