Date: 2012-03-16
Type of information: Initiation of patient enrollment
phase: 2
Announcement: initiation of patient enrollment
Company: Oxford University (UK) Imperial College London (UK) The Edinburgh University (UK)
Product: cystic fibrosis (CF) gene included in a plasmid and administered via a nebulizer
Action
mechanism: gene therapy
Disease: cystic fibrosis
Therapeutic area: Rare diseases - Genetic diseases
Country: UK
Trial
details: The phase II clinical trial will involve 130 cystic fibrosis patients using an inhaler to breathe in a working copy of the cystic fibrosis gene once a month for a year. Patients will receive the treatment by inhaling molecules of DNA wrapped in fat globules that deliver the replacement gene into the cells in the lung lining. Half the participants will receive the real treatment and half a placebo in a double-blind study. Patients aged 12 and over at Royal Brompton Hospital, London and Western General and Royal Hospital for Sick Children in Edinburgh, will receive one dose a month for one year. Over 30 patients have each received a single dose of the gene therapy in the Consortium’s previous studies, looking at how effective the therapy is at replacing the protein encoded by the defective CF gene. By delivering multiple doses over the course of a year, the researchers aim to determine whether the therapy can improve symptoms for CF patients.
Latest
news: A consortium of researchers from Oxford University, Imperial College London and the University of Edinburgh will start enrolling patients on the trial this month. The trial will go ahead thanks to £3 million in funding from the Medical Research Council (MRC) and the National Institute for Health Research (NIHR) that is announced today. Additional laboratory studies to develop a potentially more efficient delivery method for the gene therapy will receive a further £1.2 million in support from the MRC. This second, lab-based study will investigate a more advanced version of the therapy using a modified virus to carry the replacement gene into the lungs, which could in future lead to a more efficient delivery mechanism.
The outcome of the phase II trial will be known in spring 2014 and regular progress reports will be posted on the UK Cystic Fibrosis Gene Therapy Consortium’s website.
