clinical trial · NCT06302348
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
PhenylketonuriaSepiapterin
brief 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.
started
Mar 4, 2024
primary completion
Feb 28, 2031
completion
Feb 28, 2031
last updated
Mar 27, 2026
detailed description
The study includes 2 parts: Part 1 and 2. Part 1 is an open-label sepiapterin-responsiveness test and Part 2 is an open-label treatment period.
official title
A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol