AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)

NCTID	NCT06332807 (View at clinicaltrials.gov)
Description	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. (Show More)
Indication	Phenylketonurias
Compound Name	NGGT002 (rAAV8-hPAH)
Sponsor	NGGT INC.
Funder Type	Industry
Status	Recruiting
Enrollment Count	12

Target Gene/Variant	PAH
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV8
Editor Type	none
Dose 1	6.0E12 vg/kg
Dose 2	1.0E13 vg/kg
Dose 3	2.0E13 vg/kg
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
Recent Updates	Recieved Orphan Drug Designation from the FDA in Jan 2023