One-Shot gene therapy trial offers hope for lifelong PKU management
NCT ID NCT06061614
Summary
This early-stage study is testing a single intravenous infusion of an experimental gene therapy called NGGT002 in adults with phenylketonuria (PKU). The main goals are to see if the treatment is safe and if it can lower the high levels of phenylalanine (Phe) in the blood, which is the core problem in PKU. Fifteen participants will be followed closely for five years to monitor their health and diet.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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The First Affiliated Hospital of Bengbu Medical College
RECRUITINGBengbu, Anhui, 233000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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