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Ö-PKU 1 – Evaluation of a Test for Identification of BH4-Responsive PKU Patients
2010-019767-11Phase 4Completed
- Sponsor
- Medical University of Graz
- Start
- Aug 2010
- Modality
- Allosteric Modulators → Classical Binding Small Molecules → Small Molecules, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
- Countries
- 1
01
Overview
Phase 4, open-label, single-arm trial of sapropterin in phenylketonuria (PKU) and hyperphenylalaninemia (HPA), sponsored by Medical University of Graz. Primary endpoint: Consistency of BH4 responsiveness between two tests. Started Aug 2010; status: completed.
02
Drugs
sapropterin
03
Conditions
phenylketonuria (PKU)hyperphenylalaninemia (HPA)
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