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

This study is ongoing, but not recruiting participants.

First Received: October 18, 1999 Last Updated: June 8, 2006 History of Changes

Sponsor:	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Collaborator:	University of Alabama at Birmingham
Information provided by:	Office of Rare Diseases (ORD)
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	Gene Transfer: pGT-1 gene lipid complex	Phase I

Study Type:	Interventional
Study Design:	Treatment

Resource links provided by NLM:

Genetics Home Reference related topics: cystic fibrosis

MedlinePlus related topics: Cystic Fibrosis

U.S. FDA Resources

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment:	9
Study Start Date:	August 1995

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

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

University of Alabama at Birmingham

Investigators

Study Chair:

Eric J. Sorscher

University of Alabama at Birmingham

More Information

No publications provided

Study ID Numbers:	199/13941, UAB-6097, UAB-F930923001
Study First Received:	October 18, 1999
Last Updated:	June 8, 2006
ClinicalTrials.gov Identifier:	NCT00004471 History of Changes
Health Authority:	United States: Federal Government

Keywords provided by Office of Rare Diseases (ORD):

cardiovascular and respiratory diseases
cystic fibrosis
genetic diseases and dysmorphic syndromes
rare disease

Additional relevant MeSH terms:

Pathologic Processes
Digestive System Diseases
Genetic Diseases, Inborn
Respiratory Tract Diseases
Cystic Fibrosis

Fibrosis
Lung Diseases
Infant, Newborn, Diseases
Pancreatic Diseases

ClinicalTrials.gov processed this record on November 05, 2009