MULTI-CENTER, DOUBLE-BLIND, PLACEBO-CONTROLLED, PHASE II STUDY OF AEROSOLIZED TG

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The purpose of this study is to confirm the improvement in pulmonary function and cytokine levels observed in a recently completed multidose aerosol study with tgAAVCF (25B01, University of Minnesota GCRC #819). tgAAVCF is being developed as Gene Therapy for CF and the previously completed Phase II study showed a good safety profile and mild, short-term improvement in pulmonary function. The proposed study is powered to detect changes in FEV1 similar to those observed in the multidose aerosol trial at Day 30, but similar analyses of pulmonary function will be conducted at other time points to determine the time course and durability of changes in pulmonary function after two doses of study drug. This will be a randomized, double blind, placebo controlled trial of aerosolized tgAAVCF versus matching placebo. Study drug will be administered on 2 separate occasions, one month apart. Subjects participation in the study will be for a total of 224 days, with clinic visits to the General Clinical Research Center (GCRC) of the University of Minnesota every 2 weeks for a total of 8 visits and phone follow up 150 and 210 days after first administration of study drug. Subjects enrolled into the trial will be asked to complete a screening visit at the GCRC to verify entry criteria and then will be randomized to either aerosolized tgAAVCF or matching placebo. Subjects will return to the GCRC 2 weeks later for pulmonary function testing, induced sputum, and serum antibodies testing. Two weeks later the subjects will return for a second, and last, dose of study agent. Subjects will then be seen at the GCRC every 2 weeks for the next 2 months. Subjects will be contacted by phone 2 and 4 months after the last visit to assess for any adverse events.