Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency

Completed · Early Phase 1

Conditions studied: Alpha 1-Antitrypsin Deficiency

In brief

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.

Key facts

Study ID

NCT00377416

Run by

University of Massachusetts, Worcester

People needed

12

Starts

2004-03-01

Expected to finish

2020-01-01

Last updated by the study team

2020-04-10

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• University of Florida, College of Medicine, Department of Pediatrics — Gainesville, Florida, United States
• UMass Medical School — Worcester, Massachusetts, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.