We are updating the design of this site. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00004471
First Posted: October 19, 1999
Last Update Posted: March 29, 2011
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by:
University of Alabama at Birmingham
  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 1

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:


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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   16 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria
  • Cystic fibrosis patients
  • Not pregnant Fertile patients must use effective contraception
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004471


Sponsors and Collaborators
University of Alabama at Birmingham
Investigators
Study Chair: Eric J. Sorscher University of Alabama at Birmingham
  More Information

ClinicalTrials.gov Identifier: NCT00004471     History of Changes
Other Study ID Numbers: 199/13941
UAB-6097
UAB-F930923001
First Submitted: October 18, 1999
First Posted: October 19, 1999
Last Update Posted: March 29, 2011
Last Verified: March 2011

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