One-Time gene fix could ease PKU's strict diet burden
NCT ID NCT06332807
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a gene therapy called NGGT002 for adults with classic PKU, a genetic disorder that makes it hard to break down an amino acid called phenylalanine. The treatment delivers a working copy of the missing gene via a single IV infusion. Researchers will monitor 12 participants for 5 years to check safety and whether blood phenylalanine levels drop enough to relax the strict 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
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
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