AAV Gene Therapy Clinical Study in Adult Classic PKU

Status Not yet recruiting

Clinical Trial 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 an 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.

Clinical Trial Description

This study will evaluate the safety and efficacy of NGGT002 gene therapy with two dose cohorts in adult subjects with diagnosis of classic PKU, a condition characterized by severe PAH deficiency with no residual enzyme activity. NGGT002 will be administered through intravenous infusion. In Part 1 of the study, subjects will receive NGGT002 at the low dose. Dosing of the first 3 subjects will be staggered. Following evaluation of data from the first 3 subjects, a decision can be made to either escalate to the high dose level or expand the low dose cohort with additional 3 subjects. Upon completion of Part 1 study, based on the evaluation of and safety and efficacy, the study may be stopped or proceed to Part 2. In Part 2, the same process will be conducted with 3 -6 subjects dosed at the high dose. ;

Study Design

Related Conditions & MeSH terms

Phenylketonurias

Clinical Trial Details

NCT number	NCT06332807
Study type	Interventional
Source	NGGT (Suzhou) Biotechnology Co., Ltd.
Contact	Yiting Liu
Phone	(717) 480-0202
Email	yitingliu@nggtbio.com
Status	Not yet recruiting
Phase	Phase 1/Phase 2
Start date	May 22, 2024
Completion date	December 30, 2030

View Details

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