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.
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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
Locations
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Atlantic Health System
RECRUITINGMorristown, New Jersey, 07960, United States
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Children's Hospital of Orange County Hospital
RECRUITINGOrange, California, 92868, United States
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University of Minnesota
RECRUITINGMinneapolis, Minnesota, 55454, United States
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University of Pittsburgh Medical Center
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
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University or Texas, Southwestern medical Center
RECRUITINGDallas, Texas, 75390, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a One-Time gene infusion rewrite the fate of PKU?
- Newborn screening study aims to catch rare diseases at birth
- PKU Drug's nutritional impact under the microscope
- PKU diet in childhood may shape adult IQ, study finds
- New PKU drink passes small taste and tolerance test
- New PKU formula shows promise in managing blood levels