Back to resultsPhase I Pilot Study of Ad5-CB-CFTR, an Adenovirus Vector Containing the Cystic Fibrosis Transmembrane Conductance Regulator Gene, in Patients With Cystic Fibrosis
Primary Purpose
Cystic Fibrosis
Status
Completed
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
Ad5-CB-CFTR
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis focused on measuring cardiovascular and respiratory diseases, cystic fibrosis, genetic diseases and dysmorphic syndromes, rare disease
Eligibility 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
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT00004779
First Posted
February 24, 2000
Last Updated
June 23, 2005
Sponsor
National Center for Research Resources (NCRR)
Collaborators
University of North Carolina
1. Study Identification
Unique Protocol Identification Number
NCT00004779
Brief Title
Phase I Pilot Study of Ad5-CB-CFTR, an Adenovirus Vector Containing the Cystic Fibrosis Transmembrane Conductance Regulator Gene, in Patients With Cystic Fibrosis
Study Type
Interventional
2. Study Status
Record Verification Date
December 2001
Overall Recruitment Status
Completed
Study Start Date
January 1993 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Name of the Sponsor
National Center for Research Resources (NCRR)
Collaborators
University of North Carolina
4. Oversight
5. Study Description
Brief Summary
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis
Keywords
cardiovascular and respiratory diseases, cystic fibrosis, genetic diseases and dysmorphic syndromes, rare disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Enrollment
12 (false)
8. Arms, Groups, and Interventions
Intervention Type
Genetic
Intervention Name(s)
Ad5-CB-CFTR
10. Eligibility
Sex
All
Minimum Age & Unit of Time
0 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Michael R. Knowles
Organizational Affiliation
University of North Carolina
Official's Role
Study Chair
12. IPD Sharing Statement
Learn more about this trial
Phase I Pilot Study of Ad5-CB-CFTR, an Adenovirus Vector Containing the Cystic Fibrosis Transmembrane Conductance Regulator Gene, in Patients With Cystic Fibrosis
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