Overview

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

Status:
RECRUITING
Trial end date:
2031-02-28
Target enrollment:
Participant gender:
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.
Phase:
PHASE3
Details
Lead Sponsor:
PTC Therapeutics
Treatments:
sepiapterin