Sepiapterin for Phenylketonuria (EPIPHENY Trial)

Phase 3

Recruiting

Research Sponsored by PTC Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, year 4

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

"This trial aims to see if starting sepiapterin treatment early in childhood can help preserve brain function in children with PKU in the long run."

Who is the study for?

This trial is for children with Phenylketonuria (PKU) who have high blood Phe levels but can maintain them within a specific range with diet. They must not have had major surgery recently, be on other trials, or have certain genetic conditions or kidney disease. Girls who can have babies must not be pregnant and agree to use birth control.

What is being tested?

The trial is testing the long-term effects of a drug called Sepiapterin on brain function in kids with PKU when started early in life. It's checking if this drug helps maintain their thinking and problem-solving abilities over time.

What are the potential side effects?

The summary didn't provide specific side effects of Sepiapterin, but common ones for PKU treatments include digestive issues, headaches, and low blood pressure. Participants will be monitored for any adverse reactions throughout the trial.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, year 4

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, year 4

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, year 4 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Mean Change From Baseline in FSIQ Wechsler Intelligence Scale for Children - Fifth Edition (WISC-V) Score

Mean Change From Baseline in Full-scale Intelligence Quotient (FSIQ) Wechsler Preschool and Primary Scale of Intelligence - Fourth Edition (WPPSI-IV) Score

Secondary study objectives

Mean Change From Baseline in FSIQ (WISC-V) Score

Mean Change From Baseline in FSIQ (WPPSI-IV) Score