Clinical trial NCT00073463: Safety and Efficacy of Recombinant Adeno-Associated Virus Containing the CFTR Gene in the Treatment of Cystic Fibrosis
Official title: A Multicenter, Double-Blind, Placebo Controlled, Phase II Study of Aerosolized tgAAVCF for the Treatment of Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
PHASE3
Conditions: Cystic Fibrosis
Keywords: Gene Transfer
Pulmonary function
Lead sponsor: Targeted Genetics Corporation
Sponsor class: INDUSTRY
Collaborators: name: Cystic Fibrosis Foundation
First posted: 2003-11-25
Enrollment: 100
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility 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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: UAB-Childrens Health System
state: Alabama
city: Palo Alto
country: United States
facility: Stanford University Medical Center
state: California
city: San Diego
country: United States
facility: UC San Diego
state: California
city: Denver
country: United States
facility: University of Colorado-The Childrens Hospital
state: Colorado
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Baltimore
country: United States
facility: The Johns Hopkins Hospital
state: Maryland
city: Boston
country: United States
facility: Mass General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Childrens Hospital
state: Massachusetts
city: Minneapolis
country: United States
facility: The Minnesota CF Center
state: Minnesota
city: Omaha
country: United States
facility: University of Nebraska
state: Nebraska
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
Study officials:
References: