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Sapropterin effects on phenylalanine and tyrosine variability in children with phenylketonuria
2010-021343-41Phase 4Active
- Sponsor
- University Medical Center Groningen
- Start
- Nov 2011
- 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 trial of sapropterin in phenylketonuria (PKU) (2 arms), sponsored by University Medical Center Groningen. Primary endpoint: Mean standard deviation of blood phenylalanine concentrations. Started Nov 2011; status: active.
02
Drugs
sapropterin
03
Conditions
phenylketonuria (PKU)
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