New pill for PKU shows promise in early human testing
NCT ID NCT05781399
First seen May 23, 2026 · Last updated May 23, 2026
Summary
This study tested a new oral drug called JNT-517 in healthy volunteers and people with phenylketonuria (PKU), a genetic condition that causes harmful buildup of phenylalanine. The main goals were to check safety, how the body processes the drug, and its effect on phenylalanine levels. The trial involved 111 participants across multiple parts, including single and multiple doses, and compared the drug to a placebo in people with PKU.
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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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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
Conditions
Explore the condition pages connected to this study.