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

Completed · 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 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.

Key facts

Study ID

NCT00430768

Run by

University of Massachusetts, Worcester

People needed

9

Starts

2006-02-01

Expected to finish

2015-01-01

Last updated by the study team

2016-12-15

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 (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

You may not qualify if…

• 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

Where it is running

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

Full record on ClinicalTrials.gov

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