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BH4 (sapropterin) in children and adolescents with phenylalanine hydroxylase deficiency
2006-000648-15Phase 3Active
- Sponsor
- Orphanetics Pharma Entwicklungs
- Start
- Apr 2006
- 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, randomised, double-blind trial in Phenylalanine hydroxylase deficiency (PAHD), classic phenylketonuria (PKU), phenylketonuria (PKU) and 1 more comparing sapropterin with placebo (2 arms). Primary endpoints: Phenylalanine tolerance under optimal blood phenylalanine control and Long-term safety over 12 months. Started Apr 2006; status: active.
02
Drugs
sapropterin
03
Conditions
Phenylalanine hydroxylase deficiency (PAHD)classic phenylketonuria (PKU)phenylketonuria (PKU)hyperphenylalaninemia (HPA)
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