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Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
Open accessAbstract
BACKGROUND: Lung delivery of plasmid DNA encoding the CFTR gene complexed with a cationic liposome is a potential treatment option for patients with cystic fibrosis. We aimed to assess the efficacy of non-viral CFTR gene therapy in patients with cystic fibrosis. METHODS: We did this randomised, double-blind, placebo-controlled, phase 2b trial in two cystic fibrosis centres with patients recruited from 18 sites in the UK. Patients (aged ≥12 years) with a forced expiratory volume in 1 s (FEV1) of 50-90% predicted and any combination of CFTR mutations, were randomly assigned, via a computer-based randomisation system, to receive 5 mL of either nebulised pGM169/GL67A gene-liposome complex or 0.9% saline (placebo) every 28 days (plus or minus 5 days) for 1 year. Randomisation was stratified by % predicted FEV1 (<70 vs ≥70%), age (<18 vs ≥18 years), inclusion in the mechanistic substudy, and dosing site (London or Edinburgh). Participants and investigators were masked to treatment allocation. The primary endpoint was the relative change in % predicted FEV1. The primary analysis was per protocol. This trial is registered with ClinicalTrials.gov, number NCT01621867. FINDINGS: Between June 12, 2012, and June 24, 2013, we randomly assigned 140 patients to receive placebo (n=62) or pGM169/GL67A (n=78), of whom 116 (83%) patients comprised the per-protocol population. We noted a significant, albeit modest, treatment effect in the pGM169/GL67A group versus placebo at 12 months' follow-up (3.7%, 95% CI 0.1-7.3; p=0.046). This outcome was associated with a stabilisation of lung function in the pGM169/GL67A group compared with a decline in the placebo group. We recorded no significant difference in treatment-attributable adverse events between groups. INTERPRETATION: Monthly application of the pGM169/GL67A gene therapy formulation was associated with a significant, albeit modest, benefit in FEV1 compared with placebo at 1 year, indicating a stabilisation of lung function in the treatment group. Further improvements in efficacy and consistency of response to the current formulation are needed before gene therapy is suitable for clinical care; however, our findings should also encourage the rapid introduction of more potent gene transfer vectors into early phase trials. FUNDING: Medical Research Council/National Institute for Health Research Efficacy and Mechanism Evaluation Programme.
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Cites
Intermittent Administration of Inhaled Tobramycin in Patients with Cystic Fibrosis
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CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression
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Self-citationRepeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis
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Ginn, S. L., Amaya, A. K., Alexander, I. E., Edelstein, M., & Abedi, M. R. (2018) · The Journal of Gene Medicine · doi:10.1002/jgm.3015 · Cited by 903
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Metadata
- Authors
- Alton, E. W. F. W., Armstrong, D. K., Ashby, D., Bayfield, K. J., Bilton, D., Bloomfield, E. V., Boyd, A. C., Brand, J., Buchan, R., Calcedo, R., Carvelli, P., Chan, M., Cheng, S. H., Collie, D. D. S., Cunningham, S., Davidson, H. E., Davies, G., Davies, J. C., Davies, L. A., Dewar, M. H., Doherty, A., Donovan, J., Dwyer, N. S., Elgmati, H. I., Featherstone, R. F., Gavino, J., Gea-Sorli, S., Geddes, D. M., Gibson, J. S. R., Gill, D. R., Greening, A. P., Griesenbach, U., Hansell, D. M., Harman, K., Higgins, T. E., Hodges, S. L., Hyde, S. C., Hyndman, L., Innes, J. A., Jacob, J., Jones, N., Keogh, B. F., Limberis, M. P., Lloyd-Evans, P., Maclean, A. W., Manvell, M. C., McCormick, D., McGovern, M., McLachlan, G., Meng, C., Montero, M. A., Milligan, H., Moyce, L. J., Murray, G. D., Nicholson, A. G., Osadolor, T., Parra-Leiton, J., Porteous, D. J., Pringle, I. A., Punch, E. K., Pytel, K. M., Quittner, A. L., Rivellini, G., Saunders, C. J., Scheule, R. K., Sheard, S., Simmonds, N. J., Smith, K., Smith, S. N., Soussi, N., Soussi, S., Spearing, E. J., Stevenson, B. J., Sumner-Jones, S. G., Turkkila, M., Ureta, R. P., Waller, M. D., Wasowicz, M. Y., Wilson, J. M., & Wolstenholme-Hogg, P.
- Venue
- The Lancet Respiratory Medicine
- Publisher
- Elsevier BV
- Published
- 2015-09
- Volume
- 3
- Issue
- 9
- Pages
- 684-691
- ISSN
- 2213-2600 (ISSN-L)
- OpenAlex
- W1922064584
- Cited by
- 428
- References
- 21
- FWCI
- 38.53
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- Alton, E. W. F. W., Armstrong, D. K., Ashby, D., Bayfield, K. J., Bilton, D., Bloomfield, E. V., Boyd, A. C., Brand, J., Buchan, R., Calcedo, R., Carvelli, P., Chan, M., Cheng, S. H., Collie, D. D. S., Cunningham, S., Davidson, H. E., Davies, G., Davies, J. C., Davies, L. A., . . . Wolstenholme-Hogg, P. (2015). Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial. The Lancet Respiratory Medicine. https://doi.org/10.1016/s2213-2600(15)00245-3
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