One-Time gene infusion aims to tame PKU for years
NCT ID NCT06061614
First seen Mar 02, 2026 · Last updated May 16, 2026 · Updated 9 times
Summary
This early-phase trial tests a single intravenous dose of NGGT002, a gene therapy for adults with phenylketonuria (PKU). PKU is a genetic condition that causes harmful buildup of phenylalanine (Phe) in the blood, requiring a strict low-protein diet. The study will enroll 15 participants and follow them for 5 years to check safety and whether the treatment can lower blood Phe levels enough to allow a more normal diet.
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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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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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