New gene therapy aims to tame PKU in first human test
NCT ID NCT06061614
First seen Jun 27, 2026 · Last updated Aug 07, 2026 · Updated 2 times
Summary
This early-phase study tests a single IV dose of NGGT002 gene therapy in 15 adults with phenylketonuria (PKU), a genetic disorder that causes harmful buildup of phenylalanine. The main goal is to check safety over one year, with long-term follow-up for five years. Researchers will also measure whether the treatment helps lower blood phenylalanine levels and allows a more normal 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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The First Affiliated Hospital of Bengbu Medical College
Bengbu, Anhui, 233000, China
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Other studies related to the condition(s) this trial covers.
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