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Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency

NCT00377416 · Status: COMPLETED · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2020-04-10

No results posted yet for this study

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 so that the AAT protein is produced at normal levels. This study will evaluate the safety of the experimental gene transfer procedure in individuals with AAT deficiency.

Conditions

Interventions

GENETIC

rAAV2-CB-hAAT Gene Vector

Participants will attend a 5-day inpatient visit, during which they will receive a series of injections consisting of one of four different doses of rAAV2-CB-hAAT.

Sponsors & Collaborators

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

    collaborator NIH

  • 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

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-03-31
Primary Completion
2006-10-31
Completion
2020-01-31
FDA Drug
Yes

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 NCT00377416 on ClinicalTrials.gov