Phase I Pilot Study of Gene Therapy for Cystic Fibrosis Using Cationic Liposome Mediated Gene Transfer

This study has been completed.

First Received on October 18, 1999. Last Updated on March 28, 2011 History of Changes

Sponsor:	University of Alabama at Birmingham
Information provided by:	University of Alabama at Birmingham
ClinicalTrials.gov Identifier:	NCT00004471

Purpose

OBJECTIVES:

Determine whether copies of the cystic fibrosis gene (pGT-1) can be delivered to the cells lining the nose of cystic fibrosis patients using cationic liposome (DMRIE/DOPE) mediated gene transfer.

Condition	Intervention	Phase
Cystic Fibrosis	Genetic: pGT-1 gene lipid complex	Phase I

Study Type:	Interventional
Study Design:	Primary Purpose: Treatment

Resource links provided by NLM:

Genetics Home Reference related topics: cystic fibrosis Help Me Understand Genetics

MedlinePlus related topics: Cystic Fibrosis

Drug Information available for: Lipids

U.S. FDA Resources

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

Estimated Enrollment:	9
Study Start Date:	August 1995
Primary Completion Date:	May 2001 (Final data collection date for primary outcome measure)

Detailed Description:

PROTOCOL OUTLINE: Under direct visualization, patients receive lipid/DNA formulation (pGT-1 lipid complex) by syringe instillation over 30 minutes to the right inferior nasal turbinate.

Patients are followed daily for 7 days, twice a week for 3 weeks, every 2-3 weeks for 10 weeks, then every 3 months thereafter.

Eligibility

Ages Eligible for Study:	16 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Cystic fibrosis patients
Not pregnant Fertile patients must use effective contraception

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004471

Sponsors and Collaborators

University of Alabama at Birmingham

Investigators

Study Chair:

Eric J. Sorscher

University of Alabama at Birmingham

More Information

No publications provided

ClinicalTrials.gov Identifier:	NCT00004471 History of Changes
Other Study ID Numbers:	199/13941, UAB-6097, UAB-F930923001
Study First Received:	October 18, 1999
Last Updated:	March 28, 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 February 09, 2012