NCT04770779Phase 3Active not recruiting

A Study Evaluating the Efficacy and Safety of Mitapivat in 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 completion2024-04-11Actual

Enrollment · participants258Actual

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

Version history

Changes to this trial

Last 12 months

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Study details

Conditions studied

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

Treatments or interventions studied

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

Primary outcomes · main study measures

Read the study measures

measure: Double-blind Period: Percentage of Participants Who Achieved Transfusion Reduction Response (TRR); description: TRR is defined as ≥50% reduction in transfused red blood cells (RBC) units with a reduction of ≥2 units of transfused RBCs in any consecutive 12-week period through Week 48 compared with baseline transfusion burden standardized to 12 weeks. Baseline transfusion burden standardized to 12 weeks= (12/24) × total number of RBC units transfused during 24-week period before the randomization date for participants randomized and not dosed or the start of study treatment for participants randomized and dosed. Participants withdrawn from the study before Week 12 (Day 85) were considered nonresponders.; timeFrame: Double-blind Period: Baseline through Week 48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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