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Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency
Bu çalışma, alfa 1-antitripsin eksikliği (alpha 1-antitrypsin deficiency) olan hastalarda, eksik olan proteinin üretimini sağlamayı amaçlayan deneysel bir gen aktarım yöntemini (rAAV1-CB-hAAT) incelemektedir. Çalışmaya, alfa 1-antitripsin eksikliği tanısı almış ve akciğer fonksiyonları belirli düzeyin üzerinde olan hastalar katılabilmiştir. University of Massachusetts, Worcester tarafından yürütülen çalışma Faz 1 aşamasında yürütülmüş ve tamamlanmıştır.
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
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