drugset
Drugs / rAAV1-CB-hAAT
last change Oct 2015 re-read 3 minutes ago

rAAV1-CB-hAAT

Gene therapy (AAV / viral vector)

Developed for
alpha 1-antitrypsin deficiency
Investigated by
Beacon Therapeutics · National Heart, Lung, and Blood Institute (NHLBI) · Alpha-1 Foundation · National Center for Research Resources (NCRR) · University of Florida · University of Massachusetts, Worcester

Trials 2

today NCT01054339 · Ph 2 NCT00430768 · Ph 1
2006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 11 trial
Phase 1 NCT00430768 Feb 2006 → Jan 2015 alpha 1-antitrypsin deficiency University of Massachusetts, Worcester Completed No outcome recorded
Phase 21 trial
Phase 2 NCT01054339 Jun 2010 → Oct 2011 alpha 1-antitrypsin deficiency Beacon Therapeutics Completed No outcome recorded

Evidence & citations 4 cited values

Every value below carries the sentence it was read from. 2 sources stand behind the page.

FieldValueCited text
Known as rAAV1-CB-hAAT ClinicalTrials.gov intervention name — accepted as the source's own label NCT01054339 ↗
1

NCT00430768 ↗
Action Restore “may be able to carry normal copies of the AAT gene into muscle cells with the expectation that additional AAT would be produced.” NCT00430768 ↗
Modality Gene therapy (AAV / viral vector) “a modified virus, adeno-associated virus (AAV), has been genetically engineered to contain a normal copy of the AAT gene.” NCT00430768 ↗
Route Intramuscular “injections in the deltoid muscle” NCT00430768 ↗