Repeated Application of Gene Therapy in CF Patients
Status:
Completed
Trial end date:
2014-05-01
Target enrollment:
Participant gender:
Summary
Cystic fibrosis is a genetic condition where epithelial cells, including from the respiratory
tract, have an abnormal function of a surface protein, the cystic fibrosis transmembrane
conductance regulator (CFTR) protein resulting from abnormal gene expression. The trial will
assess the clinical efficacy, safety & tolerability and gene expression following repeated
nebulised doses of a gene product coding for a normal CFTR protein, with the primary outcome
of the trial assessing lung function.
Phase:
Phase 2
Details
Lead Sponsor:
Imperial College London
Collaborators:
Royal Brompton & Harefield NHS Foundation Trust University of Edinburgh University of Oxford