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Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency

NCT01054339 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2019-03-28

Study results available
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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

Conditions

  • Alpha-1 Antitrypsin Deficiency

Interventions

DRUG

rAAV1-CB-hAAT

Recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    collaborator NIH

  • Beacon Therapeutics

    lead INDUSTRY

Principal Investigators

  • Terence R. Flotte, MD · University of Massachusetts Medical School, Worcester, MA

  • Bruce C. Trapnell, MD · Cincinnati Children's Hospital Medical Center, Cincinnati, OH

  • Robert A. Sandhaus, MD, PhD · National Jewish Health, Denver, CO

  • Noel G. McElvaney, MB, BCh, BAO · Beaumont Hospital, Dublin, Ireland

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-06-30
Primary Completion
2011-10-31
Completion
2015-10-31

Countries

  • United States
  • Ireland

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01054339 on ClinicalTrials.gov