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Phase 3 open-label long-term study of PTC923 in phenylketonuria
NL-OMON53510Phase 3Recruiting
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Oct 2023
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of sepiapterin in phenylketonuria (PKU) and hyperphenylalaninemia (HPA), sponsored by PTC Therapeutics, Inc. Primary endpoints: Long-term safety and Change from baseline in dietary phenylalanine/protein consumption. Started Oct 2023; status: recruiting.
02
Drugs
sepiapterin
03
Conditions
phenylketonuria (PKU)hyperphenylalaninemia (HPA)
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