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

NCT ID: NCT00004471

Last Updated: 2011-03-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

9 participants

Study Classification

INTERVENTIONAL

Study Start Date

1995-08-31

Brief Summary

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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

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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.

Conditions

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Cystic Fibrosis

Study Design

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Primary Study Purpose

TREATMENT

Interventions

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pGT-1 gene lipid complex

Intervention Type GENETIC

Eligibility Criteria

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Inclusion Criteria

* Cystic fibrosis patients
* Not pregnant Fertile patients must use effective contraception
Minimum Eligible Age

16 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University of Alabama at Birmingham

OTHER

Sponsor Role lead

Principal Investigators

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Eric J. Sorscher

Role: STUDY_CHAIR

University of Alabama at Birmingham

Other Identifiers

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UAB-6097

Identifier Type: -

Identifier Source: secondary_id

UAB-F930923001

Identifier Type: -

Identifier Source: secondary_id

199/13941

Identifier Type: -

Identifier Source: org_study_id

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