Could a daily powder shield Kids' brains from PKU damage?
NCT ID NCT06302348
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests whether sepiapterin, a powder taken daily, can preserve intelligence and quality of life in children with phenylketonuria (PKU) when started early in childhood. The trial will follow 56 children for years, measuring IQ and other outcomes. It is an open-label phase 3 study, meaning everyone knows they are getting the drug, which may limit how strong the conclusions are.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
sepiapterin (also called PTC923 or Sephience), a powder mixed in water or apple juice
What this could lead to
If it works, this could show that early treatment with sepiapterin helps children with PKU maintain normal intelligence and quality of life as they grow.
What could go wrong
This is an open-label study with no placebo group, so results may be less reliable. It is also small (56 children) and focuses on long-term outcomes, so benefits may take years to confirm.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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
Contact
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Centre Hospitalier Régional Universitaire (CHRU) de Tours - Hôpital Clocheville
RECRUITINGTours, 37044, France
Contact
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Children's Health Ireland (CHI)
RECRUITINGDublin, D01 YC67, Ireland
Contact
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Indiana University School of Medicine
RECRUITINGIndianapolis, Indiana, 46202, United States
Contact
Contact
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Instytut Matki i Dziecka
RECRUITINGWarsaw, 01-211, Poland
Contact
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Pomorski Uniwersytet Medyczny w Szczecinie
RECRUITINGSzczecin, 70-204, Poland
Contact
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The Royal Children's Hospital
RECRUITINGParkville, VIC 3052, Australia
Contact
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Women and Children Hospital
RECRUITINGNorth Adelaide, SA 5006, Australia
Contact