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Sapropterin effects on phenylalanine and tyrosine variability in phenylketonuria
NL-OMON44967Active, not recruiting
- Sponsor
- University Medical Center Groningen
- Start
- Jun 2014
- Modality
- Allosteric Modulators → Classical Binding Small Molecules → Small Molecules, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
- Countries
- 1
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Overview
Open-label trial of sapropterin in phenylketonuria (PKU) (2 arms), sponsored by University Medical Center Groningen. Primary endpoint: Diurnal and day-to-day variability of blood phenylalanine. Started Jun 2014; status: active, not recruiting.
02
Drugs
sapropterin
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Conditions
phenylketonuria (PKU)
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