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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
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
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Trial Design
1Treatment groups
Experimental Treatment
Group I: SepiapterinExperimental Treatment1 Intervention
Participants will receive age- and weight-adjusted doses of 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
6,231 Total Patients Enrolled