New pill for PKU shows promise in early human testing
NCT ID NCT05781399
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage study tested an experimental oral drug called JNT-517 in healthy volunteers and people with phenylketonuria (PKU), a genetic condition that causes harmful buildup of the amino acid phenylalanine. The main goals were to check safety and how the body processes the drug. In the PKU part, participants received either JNT-517 or a placebo for 4 weeks to see if the drug could lower phenylalanine levels.
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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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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Mater Misericordia Ltd
South Brisbane, Queensland, 4101, Australia
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Nucleus Network Melbourne
Melbourne, Melbourne VIC, 3004, Australia
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Oregon Health & Sciences University
Portland, Oregon, 97239, United States
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Rare Disease Research
Atlanta, Georgia, 30329, United States
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Royal Adelaide Hospital
Adelaide, South Australia, 5000, Australia
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UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
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UT Southwestern Medical Center
Dallas, Texas, 75390, United States
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University of Florida College of Medicine
Gainesville, Florida, 32610, United States
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University of Nebraska Medical Center
Omaha, Nebraska, 68198, United States
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University of South Florida
Tampa, Florida, 33620, United States
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University of Texas Health 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
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Westmead Hospital
Westmead, New South Wales, 2145, Australia