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

This study has been terminated.

First Received on November 21, 2003. Last Updated on January 24, 2008 History of Changes

Sponsor:	Targeted Genetics Corporation
Collaborator:	Cystic Fibrosis Foundation
Information provided by:	Targeted Genetics Corporation
ClinicalTrials.gov Identifier:	NCT00073463

Purpose

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).

Condition	Intervention	Phase
Cystic Fibrosis	Genetic: tgAAVCF	Phase II Phase III

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double-Blind Primary Purpose: Treatment
Official Title:	A Multicenter, Double-Blind, Placebo Controlled, Phase II Study of Aerosolized tgAAVCF for the Treatment of Cystic Fibrosis

Resource links provided by NLM:

Genetics Home Reference related topics: cystic fibrosis Help Me Understand Genetics

MedlinePlus related topics: Cystic Fibrosis

U.S. FDA Resources

Further study details as provided by Targeted Genetics Corporation:

Estimated Enrollment:	100
Study Start Date:	June 2003
Estimated Study Completion Date:	October 2005

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

Ages Eligible for Study:	12 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00073463

Locations

United States, Alabama
UAB-Childrens Health System
Birmingham, Alabama, United States, 35233
United States, California
Stanford University Medical Center
Palo Alto, California, United States, 94304
UC San Diego
San Diego, California, United States, 92123
United States, Colorado
University of Colorado-The Childrens Hospital
Denver, Colorado, United States, 80209
United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
United States, Maryland
The Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Childrens Hospital
Boston, Massachusetts, United States, 02115
Mass General Hospital
Boston, Massachusetts, United States, 02114
United States, Minnesota
The Minnesota CF Center
Minneapolis, Minnesota, United States, 55455
United States, Nebraska
University of Nebraska
Omaha, Nebraska, United States, 68198
United States, Pennsylvania
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States, 15213

Sponsors and Collaborators

Targeted Genetics Corporation

Cystic Fibrosis Foundation

More Information

No publications provided

ClinicalTrials.gov Identifier:	NCT00073463 History of Changes
Other Study ID Numbers:	25E01, RAC-0301-569
Study First Received:	November 21, 2003
Last Updated:	January 24, 2008
Health Authority:	United States: Food and Drug Administration

Keywords provided by Targeted Genetics Corporation:

Gene Transfer
Pulmonary function

Additional relevant MeSH terms:

Cystic Fibrosis
Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases

Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on February 09, 2012