drugset
Drugs / rAAV2-CB-hAAT
last change Jan 2020 re-read 3 minutes ago

rAAV2-CB-hAAT

Gene therapy (AAV / viral vector)

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

Trials 1

today NCT00377416 · Ph 0
200420052006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 0 NCT00377416 Mar 2004 → Oct 2006 alpha 1-antitrypsin deficiency University of Massachusetts, Worcester Completed No outcome recorded

Evidence & citations 4 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as rAAV2-CB-hAAT ClinicalTrials.gov intervention name — accepted as the source's own label NCT00377416 ↗
Action Restore “normal copies of the AAT gene into muscle cells to produce additional AAT.” NCT00377416 ↗
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.” NCT00377416 ↗
Route Intramuscular “Intramuscular Injection” NCT00377416 ↗