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Kuvan in infants and young children with phenylketonuria
NL-OMON26957Phase 3Recruiting
- Sponsor
- Amsterdam University Medical Center
- Start
- May 2011
- 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, open-label trial of sapropterin in phenylketonuria (PKU), sponsored by Amsterdam University Medical Center. Primary endpoint: Dietary phenylalanine tolerance at 26 weeks. Started May 2011; status: recruiting.
02
Drugs
sapropterin
03
Conditions
phenylketonuria (PKU)
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