Safety and Efficacy of Recombinant Adeno-Associated Virus Containing the CFTR Gene in the Treatment of Cystic Fibrosis
- Conditions
- Cystic Fibrosis
- Registration Number
- NCT00073463
- Lead Sponsor
- Targeted Genetics Corporation
- Brief Summary
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).
- Detailed Description
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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 100
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (11)
University of Florida
🇺🇸Gainesville, Florida, United States
University of Pittsburgh Medical Center
🇺🇸Pittsburgh, Pennsylvania, United States
UC San Diego
🇺🇸San Diego, California, United States
UAB-Childrens Health System
🇺🇸Birmingham, Alabama, United States
Mass General Hospital
🇺🇸Boston, Massachusetts, United States
Stanford University Medical Center
🇺🇸Palo Alto, California, United States
University of Colorado-The Childrens Hospital
🇺🇸Denver, Colorado, United States
Childrens Hospital
🇺🇸Boston, Massachusetts, United States
The Minnesota CF Center
🇺🇸Minneapolis, Minnesota, United States
University of Nebraska
🇺🇸Omaha, Nebraska, United States
The Johns Hopkins Hospital
🇺🇸Baltimore, Maryland, United States