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Phase III sepiapterin versus sapropterin study in phenylketonuria
ISRCTN79102999Phase 3Completed
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Jan 2024
- Modality
- Allosteric Modulators → Classical Binding Small Molecules → Small Molecules, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules, Small Molecules
01
Overview
Phase 3, randomised, open-label trial of sepiapterin and sapropterin in phenylketonuria (PKU) (2 arms), sponsored by PTC Therapeutics, Inc. Primary endpoint: Mean change in blood phenylalanine levels. Started Jan 2024; status: completed.
02
Drugs
sepiapterinsapropterin
03
Conditions
phenylketonuria (PKU)
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