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Last Updated: 10/31/2025
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2 clinical trials found
A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin
Enrollment Status: Recruiting
Publish Date: September 12, 2025
Intervention Type: Drug
Study Phase: Phase 3
Summary: The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment is initiated in early childhood.
A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria
Who is this study for: Patients with phenylketonuria
Enrollment Status: Recruiting
Publish Date: May 08, 2025
Intervention Type: Drug
Study Drug: PTC923
Study Phase: Phase 3
Summary: The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein consumption.
Showing 1-2 of 2
Last Updated: 10/31/2025