CI

At a glance

ClinicalIndex Comparison Record
Phase 1Completed· 9 enrolled
Drug / intervention
rAAV1-CB-hAATbiological
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT00430768
NCT00430768Phase 1Completed
CompareCT.gov

Preclinical & Phase I/II Trials of AAV-AAT Vectors: Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Alpha 1-Antitrypsin (rAAV1-CB-hAAT) Gene Vector to AAT-Deficient Adults

University of Massachusetts, Worcester·interventional·Posted Feb 2, 2007·Updated Dec 15, 2016

In Brief

A Phase 1 clinical trial evaluating rAAV1-CB-hAAT for Alpha 1-Antitrypsin Deficiency. Completed, enrolled 9 participants across 2 sites.

Detailed 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 with the expectation that the AAT protein may be produced at normal levels. This study will evaluate the safety of the experimental gene transfer procedure in individuals with AAT deficiency. The study will also determine what dose may be required to achieve normal levels of AAT.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsAlpha 1-Antitrypsin Deficiency
CountriesUnited States
CollaboratorsNational Heart, Lung, and Blood Institute (NHLBI), Beacon Therapeutics, Alpha-1 Foundation, University of Florida, National Center for Research Resources (NCRR)

Timeline

Phase 1CompletedFinished
2006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
First PostedFeb 2, 2007
Enrollment StartFeb 1, 2006
Primary CompletionJan 1, 2015
TodayJul 2, 2026
Enrollment to primary: 8.9 yearsPosted 19.4 years ago

Interventions

rAAV1-CB-hAATbiological