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Drug & research development » Clinical trials » NCT00004806

Phase I Study of Liposome-Mediated Gene Transfer in Patients With Cystic Fibrosis

Official title: Not provided

Clinical Trials gov number: NCT00004806

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Purpose: To evaluate the efficacy and safety of lipid-mediated transfer of the CF transmembrane conductance regulator gene to nasal epithelium in CF patients

Phase: 1

Type: Interventional

Study sponsor: University of Alabama at Birmingham; National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

      • Sweat chloride >60 mEq/L
      • Clinical manifestations of CF
      • Homozygous delta F508 mutation preferred
      • Compound heterozygotes for F508 mutation with pancreatic insufficiency eligible
      • FEV1 >40% of predicted PO2 ≥60 mm Hg on room air
      • No acute or recurrent sinusitis
      • No obstructive nasal polyposis
      • No pneumothorax or hemoptysis, e.g., >250 mL blood within 24-hour period, within past year
      • No unstable lung disease with worsening pulmonary symptoms, arterial blood gas, or pulmonary function tests within 2 weeks prior to entry
      • No pulmonary exacerbation within 4 weeks prior to entry

Prior/Concurrent Therapy

      • ≥2 months since participation in any other clinical study
      • ≥2 months since DNase
      • ≥4 months since corticosteroids

Patient Characteristics

    • Cardiac: No severe cardiac disease
    • Other: No other severe organ system disease, e.g., juvenile-type diabetes mellitus
    • No pregnant women


Geographical Location
No contacts or locations provided

Number of Participants
≤50 (≥18 years)

Primary Endpoint

Not provided

Secondary Endpoint

Not provided

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Not provided

View Trial Results