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

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. Identifier: NCT00004471
Recruitment Status : Completed
First Posted : October 19, 1999
Last Update Posted : March 29, 2011
Information provided by:
University of Alabama at Birmingham

Brief Summary:


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 or disease Intervention/treatment Phase
Cystic Fibrosis Genetic: pGT-1 gene lipid complex Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Enrollment : 9 participants
Primary Purpose: Treatment
Study Start Date : August 1995
Actual Primary Completion Date : May 2001

Resource links provided by the National Library of Medicine

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
  • Cystic fibrosis patients
  • Not pregnant Fertile patients must use effective contraception

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 identifier (NCT number): NCT00004471

Sponsors and Collaborators
University of Alabama at Birmingham
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Study Chair: Eric J. Sorscher University of Alabama at Birmingham
Layout table for additonal information Identifier: NCT00004471    
Other Study ID Numbers: 199/13941
First Posted: October 19, 1999    Key Record Dates
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:
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Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases