Safety and Efficacy of Recombinant Adeno-Associated Virus Containing the CFTR Gene in the Treatment of Cystic Fibrosis
Safety and Efficacy of Recombinant Adeno-Associated Virus Containing the CFTR Gene in the Treatment of Cystic Fibrosis
The purpose of this study is to confirm the improvement in pulmonary function and cytokine
 levels observed in the recently completed multidose aerosol study for the treatment of Cystic
 Fibrosis (CF).
Cystic Fibrosis is an autosomal recessive disorder with an incidence of approximately 1 in
 33000 live births. It is due to defects in the CFTR gene, which is located on chromosome 7.
 Gene Therapy holds the promise of addressing the primary defect in CF by reconstituting the
 CFTR function in the lung. tgAAVCF, which has been genetically engineered to contain the CFTR
 gene, has been extremely well tolerated following single and multiple dose administrations to
 the nose, sinus, and lung. Dose-dependent gene transfer has been demonstrated. Although
 vector gene expression has not been detected, evidence consistent with biological activity
 was observed in maxillary sinus study, and statistically significant changes in the FEV1 and
 IL-8 levels were observed in the recently completed multidose aerosol study. These findings
 are worthy of further investigation.
Eligible subjects will be randomized to two aerosolized doses of either tgAAVCF or placebo
 30 days apart. Subjects will undergo pulmonary function testing every two weeks during the
 active portion of the study (three months) and will be followed for safety for a total of
 seven months.