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Phase 2 open-label 8-day Phenoptin response study in phenylketonuria
2004-002071-16Phase 2Completed
- Sponsor
- BioMarin Pharmaceutical Inc.
- Start
- Oct 2004
- Modality
- Allosteric Modulators → Classical Binding Small Molecules → Small Molecules, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of sapropterin in phenylketonuria (PKU), sponsored by BioMarin Pharmaceutical Inc. Primary endpoints: Percentage of responders on Day 8 and Mean change in blood phenylalanine level at Day 8. Started Oct 2004; status: completed.
02
Drugs
sapropterin
03
Conditions
phenylketonuria (PKU)
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