Can a daily pill preserve IQ in kids with PKU? new trial aims to find out
NCT ID NCT06302348
First seen Oct 31, 2025 · Last updated May 21, 2026 · Updated 20 times
Summary
This study tests whether the drug sepiapterin can help preserve thinking and learning skills in children with phenylketonuria (PKU) when started in early childhood. About 56 children will take the medicine and have their IQ and quality of life measured over time. The goal is to see if early treatment can prevent or reduce long-term brain-related problems.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Birmingham Women's and Children's NHS Foundation Trust
RECRUITINGBirmingham, B4 6NH, United Kingdom
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Centre Hospitalier Régional Universitaire (CHRU) de Tours - Hôpital Clocheville
RECRUITINGTours, 37044, France
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Children's Health Ireland (CHI)
RECRUITINGDublin, D01 YC67, Ireland
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Indiana University School of Medicine
RECRUITINGIndianapolis, Indiana, 46202, United States
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Instytut Matki i Dziecka
RECRUITINGWarsaw, 01-211, Poland
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Pomorski Uniwersytet Medyczny w Szczecinie
RECRUITINGSzczecin, 70-204, Poland
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The Royal Children's Hospital
RECRUITINGParkville, VIC 3052, Australia
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Women and Children Hospital
RECRUITINGNorth Adelaide, SA 5006, Australia
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Conditions
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