New Publication, Alton et al., 2015 - Single Dose Clinical Trial

Friday, January 29th 2016

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A Phase I/IIa Safety and Efficacy Study of Nebulized Liposome-mediated Gene Therapy for Cystic Fibrosis Supports a Multidose Trial. American Journal of Respiratory and Critical Care Medicine Volume 192, Pages 1389-1392.

The results of the Consortium's first - single dose clinical trial of our GL67a/pGM169 formulation have been published.

This trial preceeded our multi dose clinical trial the results of which were published in July 2015 (Alton et al., 2015).

The aim of the Pilot study was to assess the safety of a single dose of the Wave 1 product. A total of 35 patients received a single nebulised dose of GL67a/pGM169 in either a 5 ml, 10 ml or 20 ml volume.

The different volumes of the formation were used to assess the optimal dose going forward to the multi-dose clinical trial. The results of this were encouraging and demonsrated that the 5 ml dose was optimal for the patients and this dose was selected for the multi dose clinical trial that followed on from this work.

Publication of these results was delayed because our clinical team were working flat out on the multi dose trial for so long, however the Consortium would like to thank all the patients who volunteered to be part of this important study.

The trial was supported by the UK Cystic Fibrosis Trust and the National Institute for Health Research Respiratory Biomedical Research Unit at the Royal Brompton & Harefield NHS Foundation Trust and Imperial College London and the Biomedical Research Centre, based at Imperial College Healthcare NHS Trust and Imperial College London.