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Safety and Efficacy Study of NGGT002 in PKU Adult Subjects

Phenylketonurias Clinical Trial

Official title:
A Clinical Study for the Safety and Efficacy of IV Infusion of NGGT002 in the Treatment of Phenylketonuria

Clinical Trial Summary

This investigator initiated trial is an open-label, dose escalation study to evaluate the safety, tolerability and efficacy of NGGT002, an AAV derived investigational gene therapy product expressing human PAH enzyme in adult Phenylketonuria (PKU) subjects with PAH deficiency. All participants will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.

Clinical Trial Description

This study will evaluate the safety and efficacy of NGGT002 gene therapy with three dose cohorts in adult subjects with diagnosis of PKU characterized by PAH deficiency with confirmed PAH gene mutations. NGGT002 will be administered through intravenous infusion. Dose escalation will be initiated from the low dose of NGGT002. After evaluating the safety and efficacy data, a decision will be made regarding the cohort expansion or the escalation to the next dose level. The same process will be followed for subsequent dose cohorts. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06061614
Study type Interventional
Source First Affiliated Hospital Bengbu Medical College
Contact Huan Zhou, Doctor
Phone 008613665527160
Email zhouhuanbest@vip.163.com
Status Recruiting
Phase Early Phase 1
Start date March 30, 2023
Completion date December 30, 2028
View Details
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