Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency

NCTID	NCT01054339 (View at clinicaltrials.gov)
Description	Assessment of the safety and efficacy of intramuscular (IM) administration of a recombinant adenoassociated virus (rAAV) alpha-1 antitrypsin (AAT) vector (rAAV1-CB-hAAT) in AAT-deficient adults at three dosage levels \[6.0 × 10e11, 1.9 × 10e12 and 6.0 × 10e12 vector genome particles (vg) per kg body weight\]. Funding Sources - The FDA Office of Orphan Products Development and NIH National Heart, Lung, and Blood Institute (Show More)
Development Status	Inactive
Indication	Alpha-1 Antitrypsin Deficiency (AATD)
Disease Ontology Term	DOID:13372
Compound Name	AGTC-0106
Compound Description	rAAV1-CB-hAAT
Sponsor	Beacon Therapeutics
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	9
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	Intramuscular
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV1
Editor Type	none
Dose 1	6E11 vg/kg
Dose 2	1.9E12 vg/kg
Dose 3	6E12 vg/kg
Dose 4
Dose 5

No.of Trial Sites	4
Locations	United States,Ireland

Has US IND	True
FDA Designations
Recent Updates	IM route resulted in poor efficacy, product development abandoned