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Phase 3 Open-Label Study of PTC923 (sepiapterin) in Phenylketonuria
jRCT2041230069Phase 3Active, not recruiting
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Dec 2023
- Modality
- Small Molecules
01
Overview
Phase 3, open-label, single-arm trial of sepiapterin in phenylketonuria (PKU), sponsored by PTC Therapeutics, Inc. Primary endpoints: Long-term safety of PTC923 and Change in dietary phenylalanine/protein consumption. Started Dec 2023; status: active, not recruiting.
02
Drugs
sepiapterin
03
Conditions
phenylketonuria (PKU)
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