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Sepiapterin for Phenylketonuria (EPIPHENY Trial)
Phase 3
Waitlist Available
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 up to 6 years
Awards & highlights
EPIPHENY 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.Check my eligibility
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.See study design
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.
EPIPHENY Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline up to 6 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to 6 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Mean Change From Baseline in FSIQ Weschler Intelligence Scale for Children - Fifth Edition (WISC-V) Score
Mean Change From Baseline in Full-scale Intelligence Quotient (FSIQ) Weschler Preschool and Primary Scale of Intelligence - Fourth Edition (WPPSI-IV) Score
Secondary outcome measures
Change From Baseline in Mean Blood Phe Levels
Change From Baseline in Phenylketonuria-Quality of Life (PKU-QOL) Questionnaire Score
Change From Baseline in the European Quality of Life - 5 Dimensions (EQ-5D) Score
+2 moreEPIPHENY Trial Design
1Treatment groups
Experimental Treatment
Group I: SepiapterinExperimental Treatment1 Intervention
Participants ≥1 month of age with average screening blood Phe ≥360 micromoles (μmol)/liter (L) will receive age- and weight-adjusted doses of sepiapterin orally once daily for 4 weeks in Part 1. Participants ≥1 month of age with average screening blood Phe <360 μmol/L will receive an age- and weight-adjusted dose of sepiapterin on Day 1 of Part 1 after protein/Phe load. Participants <1 month of age at screening will receive a single oral dose of sepiapterin on Day 1 of Part 1. Participants who are responsive to sepiapterin in Part 1 will progress to Part 2 and will continue to receive sepiapterin orally once daily for up to 6 years.
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Who is running the clinical trial?
PTC TherapeuticsLead Sponsor
74 Previous Clinical Trials
6,174 Total Patients Enrolled
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What is the safety profile of Sepiapterin in individuals?
"According to our assessment at Power, the safety rating for Sepiapterin is graded as 3. This evaluation aligns with the Phase 3 trial status, indicating a foundation of efficacy data and robust safety records from various evaluations."
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