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Phase 3 Randomized Double-Blind Placebo-Controlled Study of Phenoptin (sapropterin) in Phenylketonuria
2004-004512-23Phase 3Completed
- Sponsor
- BioMarin Pharmaceutical Inc.
- Start
- Feb 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 with Placebo (2 arms). Primary endpoint: Blood phenylalanine level at Week 6. Started Feb 2005; status: completed.
02
Drugs
sapropterin
03
Conditions
phenylketonuria (PKU)
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