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Phase 3 open-label sepiapterin study in phenylketonuria
2023-509229-31-00Phase 3Completed
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Feb 2024
- Modality
- Small Molecules
- Countries
- 18
01
Overview
Phase 3, open-label, single-arm trial of sepiapterin in phenylketonuria (PKU), sponsored by PTC Therapeutics, Inc. Primary endpoints: Safety of long-term treatment with PTC923 and Change from baseline in dietary phenylalanine/protein consumption. Started Feb 2024; status: completed.
02
Drugs
sepiapterin
03
Conditions
phenylketonuria (PKU)
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