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BH4 Response in PKU Patients with Y414C Mutation
NCT00260000Phase 2Completed
- Sponsor
- Kennedy Institute - National Eye Clinic
- Start
- Apr 2005
- Modality
- Allosteric Modulators, Metabolically Activated Prodrugs
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of sapropterin in phenylketonuria (PKU), sponsored by Kennedy Institute - National Eye Clinic. Primary endpoint: Fasting Serum Phenylalanine at Day 7. Started Apr 2005; status: completed.
02
Drugs
sapropterin
03
Conditions
phenylketonuria (PKU)
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