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.
Sponsor:
Collaborator:
University of North Carolina
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00004779
First received: February 24, 2000
Last updated: June 23, 2005
Last verified: December 2001
  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
Genetic: Ad5-CB-CFTR
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Primary Purpose: Treatment

Resource links provided by NLM:


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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

Sponsors and Collaborators
University of North Carolina
Investigators
Study Chair: Michael R. Knowles University of North Carolina
  More Information

No publications provided

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

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 July 23, 2014