A Multicenter, Double-Blind, Placebo Controlled, Phase II Study of Aerosolized tgAAVCF for the Treatment of Cystic Fibrosis

Targeted Genetics Corporation11 sites in 1 country100 target enrollmentJune 2003

ConditionsCystic Fibrosis

Overview

Phase: Phase 2
Intervention: Not specified
Conditions: Cystic Fibrosis
Sponsor: Targeted Genetics Corporation
Enrollment: 100
Locations: 11
Status: Terminated
Last Updated: 18 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

June 2003

End Date

October 2005

Last Updated

18 years ago

Study Type

Interventional

Study Design

Parallel