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A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

NCT06302348 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 56

Last updated 2026-05-06

No results posted yet for this study

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.

Conditions

Interventions

DRUG

Sepiapterin

Sepiapterin powder for oral use will be mixed in water or apple juice prior to administration.

Sponsors & Collaborators

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
9 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-03-04
Primary Completion
2031-02-28
Completion
2031-02-28
FDA Drug
Yes

Countries

  • United States
  • Australia
  • France
  • Ireland
  • Poland
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06302348 on ClinicalTrials.gov