Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency
Summary
The lay summary the sponsor registered on ClinicalTrials.gov.
Investigators
The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.
- Bruce C. Trapnell, MD
Cincinnati Children's Hospital Medical Center, Cincinnati, OH
Principal InvestigatorKOL profile → - Noel G. McElvaney, MB, BCh, BAO
Beaumont Hospital, Dublin, Ireland
Principal Investigator - Robert A. Sandhaus, MD, PhD
National Jewish Health, Denver, CO
Principal Investigator - Terence R. Flotte, MD
University of Massachusetts Medical School, Worcester, MA
Principal Investigator
A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.
Sites
4 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.
- Beaumont Hospital
Dublin, Ireland
- National Jewish Health
Denver, Colorado, United States
- University of Massachusetts Medical Center
Worcester, Massachusetts, United States
- Cincinnati Children'S Hospital Medical Center
Cincinnati, Ohio, United States
Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.
Drugs studied
The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.
Registration & key dates
Registry attributes as recorded on the ClinicalTrials.gov study record.
- Start
- 30 Jun 2010
- Primary completion
- 31 Oct 2011
- Completion
- 31 Oct 2015
- Last update posted
- 28 Mar 2019
- Registered enrollment
- 9
- Registry id
- NCT01054339
Cross-registrations
Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.