A Multicenter, Double-Blind, Placebo Controlled, Phase II Study of Aerosolized tgAAVCF for the Treatment of Cystic Fibrosis
Data Collection
Cystic Fibrosis+6
+ Digestive System Diseases
+ Fibrosis
Treatment Study
Summary
Study start date: June 1, 2003
Actual date on which the first participant was enrolled.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.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.100 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Over 12 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Criteria
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.
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 11 locations
Stanford University Medical Center
Palo Alto, United StatesUC San Diego
San Diego, United StatesUniversity of Colorado-The Childrens Hospital
Denver, United States