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Could a daily powder shield Kids' brains from PKU damage?

NCT ID NCT06302348

Recruiting now Disease control Sponsor: PTC Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jul 24, 2026 · Updated 3 times

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

phenylketonuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PHENYLKETONURIA

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades

    RECRUITING

    Paris, 75015, France

  • Birmingham Women's and Children's NHS Foundation Trust

    RECRUITING

    Birmingham, B4 6NH, United Kingdom

  • Centre Hospitalier Régional Universitaire (CHRU) de Tours - Hôpital Clocheville

    RECRUITING

    Tours, 37044, France

  • Centre Hospitalier Universitaire (CHU) de Lille - Hôpital Jeanne de Flandre

    RECRUITING

    Lille, 59800, France

  • Children's Health Ireland (CHI)

    RECRUITING

    Dublin, D01 YC67, Ireland

  • Indiana University School of Medicine

    RECRUITING

    Indianapolis, Indiana, 46202, United States

  • Instytut Matki i Dziecka

    RECRUITING

    Warsaw, 01-211, Poland

  • Pomorski Uniwersytet Medyczny w Szczecinie

    RECRUITING

    Szczecin, 70-204, Poland

  • The Royal Children's Hospital

    RECRUITING

    Parkville, VIC 3052, Australia

  • Women and Children Hospital

    RECRUITING

    North Adelaide, SA 5006, Australia

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