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Mitapivat in regularly transfused pediatric pyruvate kinase deficiency
NL-OMON53547Phase 3Recruiting
- Sponsor
- Agios Pharmaceuticals
- Start
- May 2023
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial of mitapivat in pyruvate kinase deficiency (PKD) and Hemolytic anemia (HA) (3 arms), sponsored by Agios Pharmaceuticals. Primary endpoint: Transfusion reduction response. Started May 2023; status: recruiting.
02
Drugs
mitapivat
03
Conditions
pyruvate kinase deficiency (PKD)Hemolytic anemia (HA)
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