MDT gene therapy
Trial Details
- Trial statusCompleted
- Chief InvestigatorEric Alton
- SponsorImperial College London
- ISRCTN number71164341
- Phase2B
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