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RecruitingPhase 3NCT06302348

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin

Sponsor

PTC Therapeutics

Enrollment

56 participants

Start Date

Mar 4, 2024

Study Type

INTERVENTIONAL

Conditions

Phenylketonuria

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.

Eligibility

Max Age: 9 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Sepiapterin for people with phenylketonuria. The study is currently recruiting participants at 8 locations. People eligible for this study include up to age 9 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGSepiapterin

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

Locations(10)

Indiana University School of Medicine

Indianapolis, Indiana, United States

Women and Children Hospital

North Adelaide, Australia

The Royal Children's Hospital

Parkville, Australia

Centre Hospitalier Universitaire (CHU) de Lille - Hôpital Jeanne de Flandre

Lille, France

Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades

Paris, France

Centre Hospitalier Régional Universitaire (CHRU) de Tours - Hôpital Clocheville

Tours, France

Children's Health Ireland (CHI)

Dublin, Ireland

Pomorski Uniwersytet Medyczny w Szczecinie

Szczecin, Poland

Instytut Matki i Dziecka

Warsaw, Poland

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, United Kingdom

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NCT06302348

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