Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects
A Phase I/II Study for the Safety and Efficacy of Intravenous Infusion With NGGT002 in Adults Patients With Classic Phenylketonuria
NGGT (Suzhou) Biotechnology Co., Ltd.
18 participants
Jul 25, 2024
INTERVENTIONAL
Conditions
Summary
This is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects with classic Phenylketonuria (PKU). NGGT002 is a rAAV8 based vector carrying a functional copy of the human PAH gene. Participants will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.
Eligibility
Plain Language Summary
Simplified for easier understanding
This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.
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Interventions
adeno-associated viral vector with human phenylalanine hydroxylase gene
Locations(2)
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NCT06687733