New pill for PKU shows promise in small teen trial

NCT ID NCT06637514

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a new drug called JNT-517 in 14 teenagers with phenylketonuria (PKU). Participants took either JNT-517 or a placebo twice daily for 28 days. The main goal was to check safety and how the drug moves through the body, while also measuring changes in blood phenylalanine levels.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

JNT-517 (a tablet taken twice daily)

What this could lead to

If successful, this could lead to a new oral treatment option to help control phenylalanine levels in teenagers with PKU.

What could go wrong

This is a small, early-phase trial with only 14 participants, so results may not apply to everyone. The main goal is safety, not yet proof of effectiveness.

Disclaimer Read more

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.

phenylketonuria

As listed by the trial registrant

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

Contacts and locations

Locations

  • Children's Medical Center Dallas

    Dallas, Texas, 75235, United States

  • Murdoch Children's Research Institute

    Parkville, Melbourne, Victoria, 3052, Australia

  • Oregon Health and Science University

    Portland, Oregon, 97239, United States

  • University of Florida (UF) Health Shands Hospital

    Gainesville, Florida, 32608, United States

  • University of South Florida

    Tampa, Florida, 33606, United States

  • University of Texas Health (UTHealth) Science Center at Houston

    Houston, Texas, 77030, United States

  • Utah Health - The University of Utah Hospital

    Salt Lake City, Utah, 84112, United States