Trial intelligence / Profile preview

BH4 dosing timing study in phenylketonuria

NL-OMON25048Not yet recruiting

Sponsor
University Medical Center Groningen
Start
Oct 2021
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

Randomised, open-label trial of sapropterin in phenylketonuria (PKU) (3 arms), sponsored by University Medical Center Groningen. Primary endpoint: Blood phenylalanine variation. Planned start Oct 2021; status: not yet recruiting.

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Drugs

sapropterin
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Conditions

phenylketonuria (PKU)

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