Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency

NCTID	NCT02168686 (View at clinicaltrials.gov)
Description	The ADVANCE study is being conducted by Adverum Biotechnologies, Inc. as an open-label, multicenter, dose-escalation study in order to assess the safety and protein expression of ADVM-043 following a single intravenous or intrapleural administration. (Show More)
Development Status	Inactive
Indication	Alpha-1 Antitrypsin Deficiency (AATD)
Disease Ontology Term	DOID:13372
Compound Name	ADVM-043
Sponsor	Adverum Biotechnologies, Inc.
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	6
Results Posted	View Results

Target Gene/Variant	SERPINA1
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	AAVrh10
Editor Type	none
Dose 1	8E13 vg
Dose 2	4E14 vg
Dose 3	1.2E15 vg
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Product failed to show efficacy or dose-response effect; Product development was discontinued in August 2019