Safety and Efficacy of Recombinant Adeno-Associated Virus Containing the CFTR Gene in the Treatment of Cystic Fibrosis

Cystic Fibrosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00073463
• Other study ID #: 25E01
• Secondary ID: RAC-0301-569
• Status: Terminated
• Phase: Phase 2/Phase 3
• First received: November 21, 2003
• Last updated: January 24, 2008
• Start date: June 2003
• Est. completion date: October 2005

Study information

• Verified date: January 2008
• Source: Targeted Genetics Corporation
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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).

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 information / eligibility

• Status: Terminated
• Enrollment: 100
• Est. completion date: October 2005
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Years and older

Eligibility

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 Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double-Blind, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Genetic:
• tgAAVCF

Locations

Country	Name	City	State
United States	The Johns Hopkins Hospital	Baltimore	Maryland
United States	UAB-Childrens Health System	Birmingham	Alabama
United States	Childrens Hospital	Boston	Massachusetts
United States	Mass General Hospital	Boston	Massachusetts
United States	University of Colorado-The Childrens Hospital	Denver	Colorado
United States	University of Florida	Gainesville	Florida
United States	The Minnesota CF Center	Minneapolis	Minnesota
United States	University of Nebraska	Omaha	Nebraska
United States	Stanford University Medical Center	Palo Alto	California
United States	University of Pittsburgh Medical Center	Pittsburgh	Pennsylvania
United States	UC San Diego	San Diego	California

Sponsors (2)

Lead Sponsor	Collaborator
Targeted Genetics Corporation	Cystic Fibrosis Foundation Therapeutics

Country where clinical trial is conducted

United States,