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BH4 treatment in mild phenylketonuria (PKU) with common Danish PAH mutation
2004-002365-21Phase 3Completed
- Sponsor
- Kennedy Krieger Institute
- Start
- Jul 2008
- 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, single-arm trial of sapropterin in phenylketonuria (PKU), sponsored by Kennedy Krieger Institute. Primary endpoint: BH4 responsiveness completion in target genotype group. Started Jul 2008; status: completed.
02
Drugs
sapropterin
03
Conditions
phenylketonuria (PKU)
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