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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
History of Changes
  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:

MedlinePlus related topics: Cystic Fibrosis
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 May 22, 2013