Condition: Phenylketonuria · Sponsor: PTC Therapeutics
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.
This description comes directly from the study's public registry record.
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| Indiana University School of Medicine | Indianapolis, Indiana, United States | Recruiting |
| Women and Children Hospital | North Adelaide, Australia | Recruiting |
| The Royal Children's Hospital | Parkville, Australia | Recruiting |
| Centre Hospitalier Universitaire (CHU) de Lille - Hôpital Jeanne de Flandre | Lille, France | Recruiting |
| Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades | Paris, France | Recruiting |
| Centre Hospitalier Régional Universitaire (CHRU) de Tours - Hôpital Clocheville | Tours, France | Recruiting |
| Children's Health Ireland (CHI) | Dublin, Ireland | Recruiting |
| Pomorski Uniwersytet Medyczny w Szczecinie | Szczecin, Poland | Recruiting |
| Instytut Matki i Dziecka | Warsaw, Poland | Recruiting |
| Birmingham Women's and Children's NHS Foundation Trust | Birmingham, United Kingdom | Recruiting |
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Source record: clinicaltrials.gov/study/NCT06302348