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 Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

1993-01-31

Brief Summary

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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

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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.

Conditions

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Cystic Fibrosis

Keywords

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

Study Design

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Primary Study Purpose

TREATMENT

Interventions

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Ad5-CB-CFTR

Intervention Type GENETIC

Eligibility Criteria

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Inclusion 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

Minimum Eligible Age

0 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Principal Investigators

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Michael R. Knowles

Role: STUDY_CHAIR

University of North Carolina

Other Identifiers

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UNCCH-921

Identifier Type: -

Identifier Source: secondary_id

199/11829