Phase I Pilot Study of Ad5-CB-CFTR, an Adenovirus Vector Containing the Cystic Fibrosis Transmembrane Conductance Regulator Gene, in Patients With Cystic Fibrosis

This study has been completed.

First Received: February 24, 2000 Last Updated: June 23, 2005 History of Changes

Sponsors and Collaborators:	National Center for Research Resources (NCRR) University of North Carolina
Information provided by:	Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:	NCT00004779

Purpose

OBJECTIVES: I. Assess the safety and efficacy of gene transfer into the nasal epithelium using Ad5-CB-CFTR, an E1-deleted adenovirus vector containing the cystic fibrosis transmembrane conductance regulator gene, in patients with cystic fibrosis (CF).

II. Determine whether ion transport abnormalities in CF airway cells can be corrected.

Condition	Intervention	Phase
Cystic Fibrosis	Gene Transfer: Ad5-CB-CFTR	Phase I

Genetics Home Reference related topics: cystic fibrosis

MedlinePlus related topics: Cystic Fibrosis

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Safety/Efficacy Study

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment:	12
Study Start Date:	January 1993

Detailed Description:

PROTOCOL OUTLINE:

Groups of 3 patients receive 1 of 4 doses of Ad5-CB-CFTR, a recombinant E1-deleted adenovirus serotype 5 vector containing the cystic fibrosis transmembrane conductance regulator gene. Ad5-CB-CFTR is administered to 1 nasal cavity and the vehicle alone is administered to the opposite nasal cavity as the control.

Eligibility

Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics-- Cystic fibrosis No mild genetic mutations, i.e., normal nasal chloride ion permeability At least 2 weeks since decrease in pulmonary function --Prior/Concurrent Therapy-- At least 3 months since systemic cortisone At least 1 month since other therapeutic research study, e.g., DNAse --Patient Characteristics-- Other: Adequate endocrine, liver, kidney, and cardiac function Adenovirus antibody seropositive No pregnant or nursing women Negative pregnancy test required of fertile women Adequate contraception required of fertile patients

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004779

Sponsors and Collaborators

National Center for Research Resources (NCRR)

University of North Carolina

Investigators

Study Chair:

Michael R. Knowles

University of North Carolina

More Information

No publications provided

Study ID Numbers:	199/11829, UNCCH-921
Study First Received:	February 24, 2000
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00004779 History of Changes
Health Authority:	United States: Federal Government

Keywords provided by Office of Rare Diseases (ORD):

cardiovascular and respiratory diseases
cystic fibrosis
genetic diseases and dysmorphic syndromes
rare disease

Study placed in the following topic categories:

Digestive System Diseases
Genetic Diseases, Inborn
Respiratory Tract Diseases
Cystic Fibrosis
Fibrosis
Lung Diseases

Adenoviridae Infections
Respiration Disorders
Rare Diseases
Infant, Newborn, Diseases
Pancreatic Diseases

Additional relevant MeSH terms:

Pathologic Processes
Digestive System Diseases
Genetic Diseases, Inborn
Respiratory Tract Diseases
Cystic Fibrosis

Fibrosis
Lung Diseases
Infant, Newborn, Diseases
Pancreatic Diseases

ClinicalTrials.gov processed this record on May 07, 2009