This study examined a gene therapy based on an adeno-associated virus vector carrying the CFTR gene in patients with cystic fibrosis. Patients with cystic fibrosis who had mild to moderate lung disease could take part. The study was carried out 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.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Diagnosis of cystic fibrosis (CF) with mild to moderate lung disease; FVC at least 60% predicted; Schwachman-Kulczycki score at least 65; Sweat chloride greater than 60 mEq/L All CF genotypes allowed No sputum colonization with Burkholderia cepacia or another multiply resistant organism No recurrent or intermittent hemoptysis; At least 1 year since significant hemoptysis requiring transfusion or embolization OR Coughing of greater than 30 mL of blood more often than once a week --Prior/Concurrent Therapy-- At least 30 days since hospitalization or home intravenous antibiotic therapy for pulmonary exacerbation No concurrent investigational drugs or therapy --Patient Characteristics-- Other: No concurrent cigarette smoking; Not pregnant or nursing; Negative pregnancy test; Fertile patients must use effective contraception