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

This study has been completed.
Sponsor:
Collaborator:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Information provided by:
University of Alabama at Birmingham
ClinicalTrials.gov Identifier:
NCT00004806
First received: February 24, 2000
Last updated: March 29, 2011
Last verified: March 2011
History of Changes
  Purpose

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.


Condition Intervention Phase
Cystic Fibrosis
 Genetic: Cystic fibrosis transmembrane conductance regulator
 Phase 1

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:

MedlinePlus related topics: Cystic Fibrosis
U.S. FDA Resources

Further study details as provided by University of Alabama at Birmingham:

Estimated Enrollment: 9
Study Start Date: June 1995
Study Completion Date: November 2002
Primary Completion Date: November 2002 (Final data collection date for primary outcome measure)
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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00004806

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

No publications provided

ClinicalTrials.gov Identifier: NCT00004806     History of Changes
Other Study ID Numbers: 199/11983, UAB-11983
Study First Received: February 24, 2000
Last Updated: March 29, 2011
Health Authority: United States: Federal Government

Keywords provided by University of Alabama at Birmingham:
cardiovascular and respiratory diseases
cystic fibrosis
genetic diseases and dysmorphic syndromes
rare disease

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
 Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on May 16, 2013