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Tetrahydrobiopterin (BH4) dosing regimens in phenylketonuria
2021-003777-63Phase 4
- Sponsor
- University Medical Center Groningen
- Start
- Nov 2021
- 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 trial of sapropterin in phenylketonuria (PKU) (3 arms), sponsored by University Medical Center Groningen. Primary endpoint: Phenylalanine variability across BH4 dosing regimens.
02
Drugs
sapropterin
03
Conditions
phenylketonuria (PKU)
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