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Phase 2 Open-label Study of Phenoptin (Sapropterin) in Primary BH4 Deficiency

2005-003778-13Phase 2Completed

Sponsor: BioMarin Pharmaceutical Inc.
Start: Jun 2006
Modality: Allosteric Modulators → Classical Binding Small Molecules → Small Molecules, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
Countries: 1

Overview

Phase 2, open-label, single-arm trial of sapropterin in Biopterin Deficiency and phenylketonuria (PKU), sponsored by BioMarin Pharmaceutical Inc. Primary endpoint: Maintenance of blood phenylalanine levels. Started Jun 2006; status: completed.

Drugs

sapropterin

Conditions

Biopterin Deficiencyphenylketonuria (PKU)

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Available in the Gosset platform where reported.

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Available in the Gosset platform where reported.

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