A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Recruiting now · Phase 3
Conditions studied: Phenylketonuria
In brief
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.
Key facts
- Study ID
- NCT06302348
- Run by
- PTC Therapeutics
- People needed
- 56
- Starts
- 2024-03-04
- Expected to finish
- 2031-02-28
- Last updated by the study team
- 2026-07-23
Who can join
Age: any, up to 9. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Indiana University School of Medicine — Indianapolis, Indiana, United States (enrolling)
- Women and Children Hospital — North Adelaide, Australia (enrolling)
- The Royal Children's Hospital — Parkville, Australia (enrolling)
- Centre Hospitalier Universitaire (CHU) de Lille - Hôpital Jeanne de Flandre — Lille, France (enrolling)
- Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades — Paris, France (enrolling)
- Centre Hospitalier Régional Universitaire (CHRU) de Tours - Hôpital Clocheville — Tours, France (enrolling)
- Children's Health Ireland (CHI) — Dublin, Ireland (enrolling)
- Pomorski Uniwersytet Medyczny w Szczecinie — Szczecin, Poland (enrolling)
- Instytut Matki i Dziecka — Warsaw, Poland (enrolling)
- Birmingham Women's and Children's NHS Foundation Trust — Birmingham, United Kingdom (enrolling)
Full record on ClinicalTrials.gov
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