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
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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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Locations
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Children's Medical Center Dallas
Dallas, Texas, 75235, United States
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Murdoch Children's Research Institute
Parkville, Melbourne, Victoria, 3052, Australia
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Oregon Health and Science University
Portland, Oregon, 97239, United States
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University of Florida (UF) Health Shands Hospital
Gainesville, Florida, 32608, United States
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University of South Florida
Tampa, Florida, 33606, United States
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University of Texas Health (UTHealth) Science Center at Houston
Houston, Texas, 77030, United States
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Utah Health - The University of Utah Hospital
Salt Lake City, Utah, 84112, United States