Can a daily pill save kids' brains from PKU damage?
NCT ID NCT06302348
First seen Oct 31, 2025 · Last updated May 15, 2026 · Updated 18 times
Summary
This study tests a drug called sepiapterin in 56 children with phenylketonuria (PKU), a genetic condition that can harm the brain if not managed. The goal is to see if starting treatment early can preserve thinking skills like IQ and quality of life over the long term. Children as young as 1 month old can join, and they will take the drug for years while researchers track their development.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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
Explore the condition pages connected to this study.