Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency

EARLY_PHASE1CompletedINTERVENTIONAL
Enrollment

12

Participants

Timeline

Start Date

March 31, 2004

Primary Completion Date

October 31, 2006

Study Completion Date

January 31, 2020

Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
GENETIC

rAAV2-CB-hAAT Gene Vector

Participants will attend a 5-day inpatient visit, during which they will receive a series of injections consisting of one of four different doses of rAAV2-CB-hAAT.

Trial Locations (2)

32610

University of Florida, College of Medicine, Department of Pediatrics, Gainesville

01655

UMass Medical School, Worcester

Sponsors

Collaborators (1)

All Listed Sponsors
collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

collaborator

Alpha-1 Foundation

OTHER

collaborator

University of Florida

OTHER

collaborator

National Center for Research Resources (NCRR)

NIH

lead

University of Massachusetts, Worcester

OTHER

NCT00377416 - Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency | Biotech Hunter | Biotech Hunter