Shot of hope for teens with PKU: new drug may ease strict diet burden
NCT ID NCT05270837
First seen Oct 31, 2025 · Last updated May 17, 2026 · Updated 31 times
Summary
This study tests a medicine called pegvaliase, given as a shot under the skin, in teenagers aged 12-17 with phenylketonuria (PKU). The goal is to see if it safely lowers high blood phenylalanine levels better than a strict diet alone. About 55 teens will take part, with half getting the drug and half continuing their usual diet. The drug is not a cure—teens would need to keep using it long-term to control their condition.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for PHENYLKETONURIA (PKU) are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Locations
-
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
-
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
-
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
-
Charité - Universitätsmedizin Berlin
Berlin, Germany
-
Children's Hospital of Colorado
Aurora, Colorado, 80045, United States
-
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226, United States
-
IU Health University Hospital
Indianapolis, Indiana, 46202, United States
-
McGovern Medical School, University of Texas Health Science Center at Houston (UTHealth)
Houston, Texas, 77030, United States
-
Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
-
Universitat Mainz
Mainz, Germany
-
University of Kentucky College of Medicine
Lexington, Kentucky, 40506, United States
-
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15224, United States
-
University of South Florida
Tampa, Florida, 33606, United States
-
University of Utah Medical Center
Salt Lake City, Utah, 84132, United States
-
University of Virginia School of Medicine
Charlottesville, Virginia, 22903, United States
-
Universitätsklinikum Hamburg-Eppendorf
Hamburg, Germany
Conditions
Explore the condition pages connected to this study.