Contact SCGE




Clinical Trial Report

Gene Therapy Trial Report

Summary

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


NCTID NCT01054339 (View at clinicaltrials.gov)
Description
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

Therapy Information


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

Study Record Dates


Current Stage Phase2
Submit Date 2010-01-21
Completion Date 2015-10
Last Update 2019-03-28

Participation Criteria


Eligible Age 18 Years - 75 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 4
Locations United States,Ireland

Regulatory Information


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

Resources/Links