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Sapropterin treatment in hyperphenylalaninemia and its effects on amino acid and fatty acid profiles
ISRCTN77098312Phase 4Completed
- Sponsor
- Instituto Aragonés de Ciencias de la Salud
- Start
- Mar 2009
- Modality
- Allosteric Modulators → Classical Binding Small Molecules → Small Molecules, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
- Countries
- 1
01
Overview
Phase 4, open-label trial of sapropterin in hyperphenylalaninemia (HPA) (2 arms), sponsored by Instituto Aragonés de Ciencias de la Salud. Primary endpoints: Biochemical analysis of total amino acids and essential fatty acids, Amino acid measurement by HPLC and 1 more. Started Mar 2009; status: completed.
02
Drugs
sapropterin
03
Conditions
hyperphenylalaninemia (HPA)
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