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

University of Alabama at Birmingham9 enrolled

Overview

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.

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 Type

INTERVENTIONAL

Purpose

TREATMENT

Enrollment

Conditions

Cystic Fibrosis

Interventions

pGT-1 gene lipid complexGENETIC

Eligibility

Sex: ALLMin age: 16 Years

Medical Language ↔ Plain English

* Cystic fibrosis patients * Not pregnant Fertile patients must use effective contraception

Data from ClinicalTrials.gov

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