One-Shot gene therapy aims to fix rare genetic disorder
NCT ID NCT06332807
Summary
This early-stage study is testing a new one-time gene therapy called NGGT002 for adults with classic phenylketonuria (PKU). The goal is to see if delivering a working copy of the faulty gene is safe and can lower harmful blood levels of phenylalanine, potentially reducing the need for a strict lifelong diet. Up to 12 participants will receive a single infusion and be followed for five years.
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Contacts and locations
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Study contacts
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Contact
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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
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Conditions
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