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Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency

NCT00430768 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2016-12-15

Study results available
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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.

Conditions

Interventions

BIOLOGICAL

rAAV1-CB-hAAT

Sponsors & Collaborators

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

    collaborator NIH

  • Beacon Therapeutics

    collaborator INDUSTRY

  • Alpha-1 Foundation

    collaborator OTHER

  • University of Florida

    collaborator OTHER

  • National Center for Research Resources (NCRR)

    collaborator NIH

  • University of Massachusetts, Worcester

    lead OTHER

Principal Investigators

  • Terence R. Flotte, MD · UMass Medical School

  • Mark L Brantly, MD · University of Florida

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2006-02-28
Primary Completion
2015-01-31
Completion
2015-01-31

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

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 NCT00430768 on ClinicalTrials.gov