New shot could free teens with PKU from strict diet
NCT ID NCT05270837
First seen Jun 25, 2026 · Last updated Jun 26, 2026 · Updated 1 time
Summary
This study tests an injectable drug called pegvaliase in 55 teenagers (ages 12-17) with phenylketonuria (PKU) who have high blood Phe levels despite diet management. Half get the drug, half stick to diet only. The goal is to see if pegvaliase safely lowers Phe levels and reduces the need for a strict low-protein diet.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
pegvaliase
What this could lead to
If it works, this could offer a new treatment option for teens with PKU who struggle to manage their condition with diet alone.
What could go wrong
This is a relatively small Phase 3 trial, and pegvaliase may cause side effects like allergic reactions. It's not a cure, and long-term safety is still being studied.
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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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