This trial is active, not recruiting.

Condition cystic fibrosis
Treatment adeno-associated virus-cftr vector
Phase phase 1
Sponsor National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Collaborator University of Florida
Start date August 1999
Trial size 19 participants
Trial identifier NCT00004533, 199/14137, TGC-AAV-9502, UF-G-037-96, UF-G-37-1996



I. Determine the maximum tolerated dose of recombinant adeno-associated virus-CFTR vector in patients with cystic fibrosis.

II. Assess the safety of this gene therapy in these patients.

III. Assess the in vivo gene transfer of this vector in these patients.

IV. Assess the CFTR gene expression and physiologic activity following gene transfer in these patients.

V. Assess the clinical impact of CFTR gene expression following gene transfer in these patients.

VI. Monitor patient immune response directed against CFTR or vector components following vector administration.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation randomized
Masking double-blind
Primary purpose treatment

Eligibility Criteria

Male or female participants at least 15 years old.

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Diagnosis of cystic fibrosis (CF) with mild to moderate lung disease; FVC at least 60% predicted; Schwachman-Kulczycki score at least 65; Sweat chloride greater than 60 mEq/L All CF genotypes allowed No sputum colonization with Burkholderia cepacia or another multiply resistant organism No recurrent or intermittent hemoptysis; At least 1 year since significant hemoptysis requiring transfusion or embolization OR Coughing of greater than 30 mL of blood more often than once a week --Prior/Concurrent Therapy-- At least 30 days since hospitalization or home intravenous antibiotic therapy for pulmonary exacerbation No concurrent investigational drugs or therapy --Patient Characteristics-- Other: No concurrent cigarette smoking; Not pregnant or nursing; Negative pregnancy test; Fertile patients must use effective contraception

Additional Information

Description PROTOCOL OUTLINE: This is a randomized, dose escalation, double blind, placebo controlled, multicenter study. Patients are randomized to receive either adeno-associated virus-CFTR (AAV-CFTR) vector or placebo. Patients undergo tests on days -10 to -4 to rule out adenovirus and adeno-associated virus infections. Patients then receive AAV-CFTR vector intranasally to the right or left inferior turbinates and placebo to the other side of the nose. The next day, patients receive an endobronchial dose of AAV-CFTR vector to the superior segment of the right lower lobe. Cohorts of 2-4 patients each receive escalating doses of AAV-CFTR vector until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose level immediately preceding the dose at which at least 2 subjects develop dose limiting toxicity. Patients are followed at day 10, then at 1, 2, 3, 6, 9, and 12 months.
Trial information was received from ClinicalTrials.gov and was last updated in June 2005.
Information provided to ClinicalTrials.gov by Office of Rare Diseases (ORD).