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

Plain-language summary

This study looks at an experimental gene transfer method (rAAV1-CB-hAAT) that aims to help the body produce the missing protein in patients with alpha 1-antitrypsin deficiency. Patients diagnosed with alpha 1-antitrypsin deficiency whose lung function was above a certain level were able to take part. Sponsored by the University of Massachusetts, Worcester, the study was conducted in Phase 1 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 1

Status

Completed

Sponsor

University of Massachusetts, Worcester

Intervention

rAAV1-CB-hAAT

Start date

2/1/2006

Estimated completion

1/1/2015

Countries

United States

Related genes

Locations

• Gainesville, United States
• Worcester, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* 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 Criteria:

* 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