Phase I Study of Liposome-Mediated Gene Transfer in Patients With Cystic Fibrosis

OBJECTIVES:

Evaluate the efficacy and safety of lipid-mediated transfer of the cystic fibrosis transmembrane conductance regulator gene to nasal epithelium in patients with cystic fibrosis.

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Genetic: Cystic fibrosis transmembrane conductance regulator

Detailed Description

PROTOCOL OUTLINE: Cystic fibrosis transmembrane conductance regulator (CFTR) gene complexed with lipid is administered intranasally to the right inferior turbinate. Lipid without CFTR is administered to the left nostril as a control.

Cohorts of 3 patients are given successively lower doses of CFTR, each dose complexed with 500 micrograms of lipid.

Patients are followed at days 2-12, 15, and 21, then every 3-4 days for 3 weeks, every 2-3 weeks for 10 weeks, and every 3 months thereafter.

• Study Type: Interventional
• Enrollment: 9
• Phase: Phase 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics-- Cystic fibrosis (CF), i.e.: Sweat chloride greater than 60 mEq/L Clinical manifestations of CF Homozygous delta F508 mutation preferred Compound heterozygotes for F508 mutation with pancreatic insufficiency eligible FEV1 greater than 40% of predicted PO2 at least 60 mm Hg on room air No acute or recurrent sinusitis No obstructive nasal polyposis No pneumothorax or hemoptysis, e.g., more than 250 mL blood within 24-hour period, within past year No unstable lung disease with worsening pulmonary symptoms, arterial blood gas, or pulmonary function tests within 2 weeks prior to entry No pulmonary exacerbation within 4 weeks prior to entry --Prior/Concurrent Therapy-- At least 2 months since participation in any other clinical study At least 2 months since DNase At least 4 months since corticosteroids --Patient Characteristics-- Cardiac: No severe cardiac disease Other: No other severe organ system disease, e.g., juvenile-type diabetes mellitus No pregnant women

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment

Collaborators and Investigators

• Sponsor: University of Alabama at Birmingham
• Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Investigators: Study Chair: Eric J. Sorscher, University of Alabama at Birmingham

Study record dates

Study Major Dates

• Study Start: June 1, 1995
• Primary Completion (Actual): November 1, 2002
• Study Completion (Actual): November 1, 2002

Study Registration Dates

• First Submitted: February 24, 2000
• First Submitted That Met QC Criteria: February 24, 2000
• First Posted (Estimate): February 25, 2000

Study Record Updates

• Last Update Posted (Estimate): March 30, 2011
• Last Update Submitted That Met QC Criteria: March 29, 2011
• Last Verified: March 1, 2011

More Information

Terms related to this study

• Keywords: rare disease; cystic fibrosis; cardiovascular and respiratory diseases; genetic diseases and dysmorphic syndromes
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: 199/11983; UAB-11983