Shot of hope: new drug may free teens with PKU from strict diet
NCT ID NCT05270837
First seen Oct 31, 2025 · Last updated May 08, 2026 · Updated 30 times
Summary
This study tests a drug called pegvaliase, given as a shot under the skin, in teenagers aged 12-17 with phenylketonuria (PKU) who have trouble keeping their blood Phe levels under control with diet alone. About 55 teens will be randomly assigned to either get the drug or stick with their usual diet-only care. The goal is to see if the drug safely lowers blood Phe levels and helps teens eat more protein.
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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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Contacts and locations
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Locations
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Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Charité - Universitätsmedizin Berlin
Berlin, Germany
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Children's Hospital of Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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IU Health University Hospital
Indianapolis, Indiana, 46202, United States
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McGovern Medical School, University of Texas Health Science Center at Houston (UTHealth)
Houston, Texas, 77030, United States
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Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
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Universitat Mainz
Mainz, Germany
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University of Kentucky College of Medicine
Lexington, Kentucky, 40506, United States
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University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15224, United States
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University of South Florida
Tampa, Florida, 33606, United States
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University of Utah Medical Center
Salt Lake City, Utah, 84132, United States
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University of Virginia School of Medicine
Charlottesville, Virginia, 22903, United States
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Universitätsklinikum Hamburg-Eppendorf
Hamburg, Germany
Conditions
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