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

This study has been completed.
Information provided by:
University of Alabama at Birmingham Identifier:
First received: October 18, 1999
Last updated: March 28, 2011
Last verified: March 2011


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.


Ages Eligible for Study:   16 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
  • Cystic fibrosis patients
  • Not pregnant Fertile patients must use effective contraception
  Contacts and Locations
Please refer to this study by its identifier: NCT00004471

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

No publications provided 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
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes processed this record on April 16, 2014