Clinical Trial

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

Name: A Study of Sepiapterin in Participants With Phenylketonuria (PKU) — clinical trial record (NCT06302348)
Creator: DataLookout
License: https://creativecommons.org/licenses/by/4.0/

Study acronym: EPIPHENY

Recruiting Phase 3

View on ClinicalTrials.gov →

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.

Protocol Amendment History 1 change

notable Trial sites expanded: 5 -> 8 locations 2026-05-07

Trial Details

NCT Number	NCT06302348
Lead Sponsor	PTC Therapeutics
Conditions	Phenylketonuria
Enrollment	56 participants
Start Date	2024-03-04
Primary Completion	2031-02-28 (estimated)
Study Completion	2031-02-28 (estimated)
Updated on ClinicalTrials.gov	2026-07-23