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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00073463
Recruitment Status : Terminated
First Posted : November 25, 2003
Last Update Posted : January 28, 2008
Cystic Fibrosis Foundation
Information provided by:
Targeted Genetics Corporation

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).

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Genetic: tgAAVCF Phase 2 Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Enrollment : 100 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double
Primary Purpose: Treatment
Official Title: A Multicenter, Double-Blind, Placebo Controlled, Phase II Study of Aerosolized tgAAVCF for the Treatment of Cystic Fibrosis
Study Start Date : June 2003
Study Completion Date : October 2005

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00073463

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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
Mass General Hospital
Boston, Massachusetts, United States, 02114
Childrens Hospital
Boston, Massachusetts, United States, 02115
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
Layout table for additonal information Identifier: NCT00073463    
Other Study ID Numbers: 25E01
First Posted: November 25, 2003    Key Record Dates
Last Update Posted: January 28, 2008
Last Verified: January 2008
Keywords provided by Targeted Genetics Corporation:
Gene Transfer
Pulmonary function
Additional relevant MeSH terms:
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Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases