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CLINICAL_TRIALS research · 2026-04-24

Clinical Trial Update: 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 [Phase 3]

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Summary

Phase 3 clinical trial to evaluate the efficacy and safety of mitapivat in pediatric patients with pyruvate kinase deficiency, including a 5-year extension peri…

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Diseases / indications

Drugs mentioned

  • Mitapivat (mitapivat)

Snippet

Sponsor: Agios Pharmaceuticals, Inc. | Conditions: Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia | Phase 3 | Status: ACTIVE_NOT_RECRUITING