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

Phase 1

Completed

• Conditions: Cystic Fibrosis

• Registration Number: NCT00004471
• Lead Sponsor: University of Alabama at Birmingham

Brief Summary

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.

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.

Study Timeline

• Study Start: 1995-08
• Study First Submitted: 1999-10-18
• Study First Submitted QC: 1999-10-18
• Study First Posted: 1999-10-19
• Primary Completion: 2001-05
• Status Verified: 2011-03
• Last Update Submitted: 2011-03-28
• Last Update Posted: 2011-03-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: Not specified