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Sapropterin treatment for hyperphenylalaninemia and its influence on amino acid and fatty acid profiles
2008-005394-35Phase 3Completed
- 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 3, open-label trial of sapropterin in hyperphenylalaninemia (HPA) and Biopterin Deficiency (2 arms), sponsored by Instituto Aragonés de Ciencias de la Salud. Primary endpoints: Biochemical control parameters and Prevalence of sapropterin-responsive hyperphenylalaninemia. Started Mar 2009; status: completed.
02
Drugs
sapropterin
03
Conditions
hyperphenylalaninemia (HPA)Biopterin Deficiency
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