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Kuvan response and safety study in children and adolescents with phenylketonuria
2015-001650-15Phase 3
- Sponsor
- Merck Serono
- Start
- Jun 2015
- 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, single-arm trial of sapropterin in phenylketonuria (PKU), sponsored by Merck Serono. Primary endpoint: Percentage of participants with response to Kuvan treatment.
02
Drugs
sapropterin
03
Conditions
phenylketonuria (PKU)
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