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Clinical Trial Active — Not Recruiting Phase III NCT04770753

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT) — Active Not Recruiting • Phase III • NCT04770753.

📅 23 Jun 2026 ⏱ 1 min read
Active — Not Recruiting
Check the registry for current status and eligibility criteria.
Status
Active — Not Recruiting
Phase
Phase III
NCT ID
NCT04770753
Start
2021-12-20
Completion
2023-11-13
ClinicaliQ Trial Snapshot
  • A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT) — Active Not Recruiting • Phase III • NCT04770753.
  • Mitapivat reduced anaemia in patients with non-transfusion-dependent thalassemia compared to placebo.

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What This Trial Is Studying

The primary purpose of this study was to compare the effect of mitapivat versus placebo on hemolytic anemia in participants with alpha- or beta-non-transfusion dependent thalassemia (NTDT). Conditions: Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia Interventions: Placebo Matching Mitapivat, Mitapivat Lead Sponsor: Agios Pharmaceuticals, Inc. Planned Enrollment: 194 participants

Full Trial Details
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Eligibility criteria, protocol, and results when available
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