Status:
COMPLETED
Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency
Lead Sponsor:
University of Massachusetts, Worcester
Collaborating Sponsors:
National Heart, Lung, and Blood Institute (NHLBI)
Beacon Therapeutics
Conditions:
Alpha 1-Antitrypsin Deficiency
Eligibility:
All Genders
18+ years
Phase:
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 (Group 1) and 8 weeks (Groups 2 and 3) prior to study entry, and to resume 11 weeks after rAAV1-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 rAAV1-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 rAAV1-CB-hAAT administration
- Required oral or systemic corticosteroids in the 28 days prior to rAAV1-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
- Abnormal ECG, heart disease, pulmonary edema, or embolism in the 6 months prior to study entry
- Current or recent facial or chest trauma that makes it medically impossible to perform pulmonary function tests (PFTs)
- Any other medical condition that the investigator deems unsuitable for study participation
- Pregnant or breastfeeding
Key Trial Info
Start Date :
February 1 2006
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2015
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT00430768
Start Date
February 1 2006
End Date
January 1 2015
Last Update
December 15 2016
Active Locations (2)
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1
University of Florida, College of Medicine, Department of Pediatrics
Gainesville, Florida, United States, 32610
2
University of Massachusetts School of Medicine
Worcester, Massachusetts, United States, 01655