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Phenoptin (sapropterin) to increase phenylalanine tolerance in phenylketonuric children
2005-003777-24Phase 3Completed
- Sponsor
- BioMarin Pharmaceutical Inc.
- Start
- Dec 2005
- 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 phenylketonuria (PKU) comparing Phenoptin 20 mg/kg/day with Placebo (2 arms). Primary endpoint: Dietary phenylalanine tolerance. Started Dec 2005; status: completed.
02
Drugs
sapropterin
03
Conditions
phenylketonuria (PKU)
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