Status:
COMPLETED
Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency
Lead Sponsor:
University of Massachusetts, Worcester
Collaborating Sponsors:
National Heart, Lung, and Blood Institute (NHLBI)
Alpha-1 Foundation
Conditions:
Alpha 1-Antitrypsin Deficiency
Eligibility:
All Genders
18+ years
Phase:
EARLY_PHASE1
Brief 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...
Detailed Description
AAT deficiency is a genetic disorder in which individuals have inadequate levels of the AAT protein. AAT protects the lungs from white blood cell enzymes that can damage air sacs within the lungs, pot...
Eligibility Criteria
Inclusion
- Diagnosed with AAT deficiency
- Forced expiratory volume in one second (FEV1) greater than 24% of predicted value (post bronchodilator)
- Willing to discontinue AAT protein replacement 4 weeks prior to study entry, and to resume 11 weeks after rAAV2-CB-hAAT has been administered
- Willing to discontinue aspirin, aspirin-containing products, and other drugs that may alter platelet function 7 days prior to study entry, and to resume 24 hours after rAAV2-CB-hAAT has been administered
- Willing to use contraception throughout the study
Exclusion
- Required antibiotic therapy for a respiratory infection in the 28 days prior to rAAV2-CB-hAAT administration
- Required oral or systemic corticosteroids in the 28 days prior to rAAV2-CB-hAAT administration
- Liver disease
- Currently receiving or has received an investigational study agent in the 30 days prior to study entry
- Received gene transfer agents in the 6 months prior to study entry
- Currently smokes cigarettes or uses illegal drugs
- History of immune response to human AAT replacement
- History of platelet dysfunction
- Any other medical condition that the investigator deems unsuitable for study participation
- Pregnant or breastfeeding
Key Trial Info
Start Date :
March 1 2004
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2020
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT00377416
Start Date
March 1 2004
End Date
January 1 2020
Last Update
April 10 2020
Active Locations (2)
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1
University of Florida, College of Medicine, Department of Pediatrics
Gainesville, Florida, United States, 32610
2
UMass Medical School
Worcester, Massachusetts, United States, 01655