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Ex vivo AG-348 effects in pyruvate kinase deficiency
NL-OMON42151Active, not recruiting
- Sponsor
- Agios Pharmaceuticals
- Start
- May 2015
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Open-label trial of mitapivat in pyruvate kinase deficiency (PKD) and Congenital nonspherocytic hemolytic anemia (CNSHA) (3 arms), sponsored by Agios Pharmaceuticals. Primary endpoints: Pyruvate kinase activity, Pyruvate kinase protein stability and 7 more. Started May 2015; status: active, not recruiting.
02
Drugs
mitapivat
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Conditions
pyruvate kinase deficiency (PKD)Congenital nonspherocytic hemolytic anemia (CNSHA)
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