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PUMA study of pyruvate kinase activation in rare anemias
NL-OMON51728Recruiting
- Sponsor
- University Medical Center Utrecht
- Start
- Jul 2022
- Modality
- Small Molecules
- Countries
- 1
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Overview
Open-label trial of mitapivat in sickle cell disease (SCD), hereditary spherocytosis (HS), Xerocytosis (HX) and 5 more (8 cohorts), sponsored by University Medical Center Utrecht. Primary endpoints: Pyruvate kinase activity and Pyruvate kinase thermostability. Started Jul 2022; status: recruiting.
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Drugs
mitapivat
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Conditions
sickle cell disease (SCD)hereditary spherocytosis (HS)Xerocytosis (HX)erythrocyte enzyme deficiencyThalassaemia
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