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Drug & research development » Clinical trials » NCT00004471

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

Official title: Not provided

Clinical Trials gov number: NCT00004471

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Purpose: To 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.

Phase: 1

Type: Interventional

Study sponsor: University of Alabama at Birmingham

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


Geographical Location
No contacts or locations provided

Number of Participants
≤50 (≥16 years)

Primary Endpoint

Not provided

Secondary Endpoint

Not provided

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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.

View Trial Results