drugset / Trial / NCT00377416
Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency
Phase 0
Completed
12 enrolled
University of Massachusetts, Worcester
Alpha-1 Foundation · collabNational Center for Research Resources (NCRR) · collabNational Heart, Lung, and Blood Institute (NHLBI) · collabUniversity of Florida · collab
NaSingle-groupOpen-labelTreatment
Summary
Individuals with a deficiency of the Alpha 1-antitrypsin (AAT) protein are at risk for developing emphysema and liver damage. Researchers have developed a way to introduce normal AAT genes into muscle cells so that the AAT protein is produced at normal levels. This study will evaluate the safety of the experimental gene transfer procedure in individuals with AAT deficiency.
Timeline
- Start
- 2004-03
- Primary completion
- 2006-10
- Completion
- 2020-01
Publications
- Background Flotte TR, Brantly ML, Spencer LT, Byrne BJ, Spencer CT, Baker DJ, Humphries M. Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults. Hum Gene Ther. 2004 Jan;15(1):93-128. doi: 10.1089/10430340460732490.
- Background Song S, Morgan M, Ellis T, Poirier A, Chesnut K, Wang J, Brantly M, Muzyczka N, Byrne BJ, Atkinson M, Flotte TR. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc Natl Acad Sci U S A. 1998 Nov 24;95(24):14384-8. doi: 10.1073/pnas.95.24.14384.
- Remih K, Amzou S, Strnad P. Alpha1-antitrypsin deficiency: New therapies on the horizon. Curr Opin Pharmacol. 2021 Aug;59:149-156. doi: 10.1016/j.coph.2021.06.001. Epub 2021 Jul 10.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | rAAV2-CB-hAAT | Gene therapy (AAV / viral vector) | — | Intramuscular |