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A Study of Sepiapterin in Participants With Phenylketonuria (PKU) — EPIPHENY  

Phenylketonuria Clinical Trial

Official title:
A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin

Clinical Trial 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.

Clinical Trial Description

The study includes 2 parts: Part 1 and 2. Part 1 is a sepiapterin-responsiveness test, and Part 2 is an open-label treatment period. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06302348
Study type Interventional
Source PTC Therapeutics
Contact Patient Advocacy
Phone 1-866-562-4620
Email medinfo@ptcbio.com
Status Recruiting
Phase Phase 3
Start date March 4, 2024
Completion date February 28, 2031
View Details
See also
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