New PKU drug JNT-517 moves to Long-Term safety trial
NCT ID NCT06628128
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This Phase 3 study is testing the long-term safety of an oral drug called JNT-517 in 240 children and adults with phenylketonuria (PKU). All participants will receive the drug twice daily, with doses based on age and weight. The goal is to see if JNT-517 is safe to use over time and how it affects 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 (oral drug taken twice daily)
What this could lead to
If successful, this could confirm JNT-517 as a safe long-term treatment to help control phenylalanine levels in people with PKU.
What could go wrong
This is an open-label study with no placebo, so results may be biased. Long-term safety and effectiveness are still being evaluated, and side effects are possible.
Disclaimer
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This is a summary of
the original study
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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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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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Children's Health Queensland - Queensland Children's Hospital
RECRUITINGSouth Brisbane, Queensland, 4101, Australia
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Medical Center Dallas
RECRUITINGDallas, Texas, 75235, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Mater Health - Mater Hospital Brisbane
RECRUITINGSouth Brisbane, Queensland, 4101, Australia
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Murdoch Children's Research Institute
RECRUITINGParkville, Victoria, 3052, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Oregon Health and Science University
RECRUITINGPortland, Oregon, 97239, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Royal Adelaide Hospital
RECRUITINGAdelaide, South Australia, 5000, Australia
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Florida (UF) Health Shands Hospital
RECRUITINGGainesville, Florida, 32608, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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University of Pittsburgh Medical Center (UPMC) - Children's Hospital of Pittsburgh
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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University of South Florida
RECRUITINGTampa, Florida, 33606, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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University of Texas Health (UTHealth) Science Center at Houston
RECRUITINGHouston, Texas, 77030, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75390, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Utah Health - The University of Utah Hospital
RECRUITINGSalt Lake City, Utah, 84112, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••