Gene therapy hope for PKU: first human safety trial launches
NCT ID NCT06061614
First seen Mar 02, 2026 · Last updated May 24, 2026 · Updated 11 times
Summary
This early-stage study tests a single intravenous dose of NGGT002, a gene therapy for adults with phenylketonuria (PKU), a genetic disorder that prevents the breakdown of the amino acid phenylalanine. Fifteen participants will be monitored for safety and changes in blood phenylalanine levels for up to 5 years. The goal is to see if the treatment is safe and can help control the disease, though lifelong management may still be needed.
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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.
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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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