MDT gene therapy

Repeated application of gene therapy in CF patients

Design and Objective

Design: randomised, double-blind placebo-controlled study. Randomisation will be on a 1:1 basis, stratified for centre, age and FEV1. Objective: To assess clinical efficacy, safety & tolerability and gene expression of repeated doses of gene therapy.

Key inclusion criteria

1. Cystic fibrosis confirmed by sweat testing or genetic analysis 2. Males and females >=12 yrs 3. FEV1 between 50 & 90% predicted inclusive 4. Clinical stability at screening 5. Effective contraception 6. Written informed consent obtained 7. Permission to inform GP of participation in study

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