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Phenoptin in Phenylketonuria
NCT00104247Phase 3
Key facts
- Status
- Completed
- Start
- Mar 2005
- Countries
- 1
- Sponsor
- BioMarin Pharmaceutical
- Drugs
- sapropterin
- Conditions
- phenylketonuria (PKU)
01
Overview
Phase 3, randomised, double-blind trial in phenylketonuria (PKU) comparing Phenoptin with Placebo (2 arms). Primary endpoint: Blood Phenylalanine at 6 weeks. Started Mar 2005; status: completed.
02
Design
2 arms- Study design
- RandomizedDouble-blind
Phenoptin
Sapropterin dihydrochloride (10 mg/kg/day)
Placebo
Matching placebo
03
Who it enrols
Patients 8 years and older with PKU who responded to Phenoptin in study PKU-001 (>=30% reduction in Phe) and have elevated phenylalanine levels (>=450 μmol/L).
04
Endpoints
Primary
Blood Phenylalanine
05
Results
Primary endpoints met
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