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Drug & research development » Clinical trials » NCT00004779

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

Official title: Not provided

Clinical Trials gov number: NCT00004779

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Purpose: To assess the safety and efficacy of gene transfer into the nasal epithelium using Ad5-CB-CFTR, an E1-deleted adenovirus vector containing the CF transmembrane conductance regulator gene, in CF patients.

To determine whether ion transport abnormalities in CF airway cells can be corrected.

Phase: 1

Type: Interventional

Study sponsor: National Center for Research Resources (NCRR); University of North Carolina

Disease characteristics

    -No mild genetic mutations, i.e., normal nasal chloride ion permeability

    -≥2 weeks since decrease in pulmonary function

Prior/concurrent therapy

    -≥3 months since systemic cortisone

    -≥1 month since other therapeutic research study, e.g., DNAse

Patient characteristics

    -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

Ad5-CB-CFTR, Genetic

Geographical Location
No contacts or locations provided

Number of Participants
≤50 (Not Available)

Primary Endpoint

Not provided

Secondary Endpoint

Not provided

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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.

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