New injection offers hope for teens with rare genetic disorder
NCT ID NCT05270837
Summary
This study is testing whether daily injections of a medication called pegvaliase are safe and effective for teenagers with PKU, a genetic disorder that makes it hard to process protein. About 55 teens will be randomly assigned to receive either the injections or continue managing their PKU with a strict diet alone. The main goal is to see if the injections lower the level of a harmful substance in the blood better than diet management.
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.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
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.