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

This study has been completed.
Sponsor:
Information provided by:
University of Alabama at Birmingham
ClinicalTrials.gov Identifier:
NCT00004471
First received: October 18, 1999
Last updated: March 28, 2011
Last verified: March 2011
  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

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
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, see Learn About Clinical Studies.

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 August 21, 2014