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Phase 3 study of PTC923 in phenylketonuria
NL-OMON51999Phase 3Active, not recruiting
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Aug 2022
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial of sepiapterin in phenylketonuria (PKU) and hyperphenylalaninemia (HPA) (3 arms), sponsored by PTC Therapeutics, Inc. Primary endpoint: Mean change in blood phenylalanine from baseline to Weeks 5 and 6. Started Aug 2022; status: active, not recruiting.
02
Drugs
sepiapterin
03
Conditions
phenylketonuria (PKU)hyperphenylalaninemia (HPA)
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