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

Cystic Fibrosis Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00004806
• Other study ID #: 199/11983
• Secondary ID: UAB-11983
• Status: Completed
• Phase: Phase 1
• First received: February 24, 2000
• Last updated: March 29, 2011
• Start date: June 1995
• Est. completion date: November 2002

Study information

• Verified date: March 2011
• Source: University of Alabama at Birmingham
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 9
• Est. completion date: November 2002
• Est. primary completion date: November 2002
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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 Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Genetic:
• Cystic fibrosis transmembrane conductance regulator

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
University of Alabama at Birmingham	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)