New gene therapy hopes to ease PKU's strict diet for life
NCT ID NCT06332807
First seen Dec 18, 2025 · Last updated Apr 30, 2026 · Updated 16 times
Summary
This early-stage trial tests a one-time gene therapy called NGGT002 in 12 adults with classic PKU, a genetic disorder that makes it hard to break down an amino acid called phenylalanine. The therapy delivers a working copy of the PAH gene to help the body process phenylalanine normally. Participants will be monitored for safety and changes in blood phenylalanine levels for up to 5 years. The goal is to see if this treatment can reduce the need for a strict low-protein diet.
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 PHENYLKETONURIAS 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
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Atlantic Health System
RECRUITINGMorristown, New Jersey, 07960, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
Children's Hospital of Orange County Hospital
RECRUITINGOrange, California, 92868, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
University of Minnesota
RECRUITINGMinneapolis, Minnesota, 55454, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
University of Pittsburgh Medical Center
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
University or Texas, Southwestern medical Center
RECRUITINGDallas, Texas, 75390, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Conditions
Explore the condition pages connected to this study.