Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency
Status:
Completed
Trial end date:
2015-10-01
Target enrollment:
Participant gender:
Summary
Assessment of the safety and efficacy of intramuscular (IM) administration of a recombinant
adenoassociated virus (rAAV) alpha-1 antitrypsin (AAT) vector (rAAV1-CB-hAAT) in
AAT-deficient adults at three dosage levels [6.0 × 10e11, 1.9 × 10e12 and 6.0 × 10e12 vector
genome particles (vg) per kg body weight].
Funding Sources - The FDA Office of Orphan Products Development and NIH National Heart, Lung,
and Blood Institute