A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis

Eric W F W Alton; A. Christopher Boyd; Seng H. Cheng; Steve Cunningham; Jane C. Davies; Deborah R. Gill; Uta Griesenbach; Tracy Higgins; Stephen C. Hyde; J. Alastair Innes; Gordon D. Murray; David J. Porteous

doi:10.1136/thoraxjnl-2013-203309

A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis

Eric W F W Alton, A. Christopher Boyd, Seng H. Cheng, Steve Cunningham, Jane C. Davies^*, Deborah R. Gill, Uta Griesenbach, Tracy Higgins, Stephen C. Hyde, J. Alastair Innes, Gordon D. Murray, David J. Porteous

^*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

Abstract / Description of output

The UK Cystic Fibrosis Gene Therapy Consortium has been working towards clinical gene therapy for patients with cystic fibrosis for several years. We have recently embarked on a large, multi-dose clinical trial of a nonviral, liposome-based formulation powered for the first time to detect clinical benefit. The article describes the details of the protocol.

Original language	English
Pages (from-to)	1075-1077
Number of pages	3
Journal	Thorax
Volume	68
Issue number	11
DOIs	https://doi.org/10.1136/thoraxjnl-2013-203309
Publication status	Published - 1 Nov 2013

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10.1136/thoraxjnl-2013-203309

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Alton, E. W. F. W., Boyd, A. C., Cheng, S. H., Cunningham, S., Davies, J. C., Gill, D. R., Griesenbach, U., Higgins, T., Hyde, S. C., Innes, J. A., Murray, G. D., & Porteous, D. J. (2013). A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis. Thorax, 68(11), 1075-1077. https://doi.org/10.1136/thoraxjnl-2013-203309

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abstract = "The UK Cystic Fibrosis Gene Therapy Consortium has been working towards clinical gene therapy for patients with cystic fibrosis for several years. We have recently embarked on a large, multi-dose clinical trial of a nonviral, liposome-based formulation powered for the first time to detect clinical benefit. The article describes the details of the protocol.",

author = "Alton, {Eric W F W} and Boyd, {A. Christopher} and Cheng, {Seng H.} and Steve Cunningham and Davies, {Jane C.} and Gill, {Deborah R.} and Uta Griesenbach and Tracy Higgins and Hyde, {Stephen C.} and Innes, {J. Alastair} and Murray, {Gordon D.} and Porteous, {David J.}",

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doi = "10.1136/thoraxjnl-2013-203309",

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Alton, EWFW, Boyd, AC, Cheng, SH, Cunningham, S, Davies, JC, Gill, DR, Griesenbach, U, Higgins, T, Hyde, SC, Innes, JA , Murray, GD & Porteous, DJ 2013, 'A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis', Thorax, vol. 68, no. 11, pp. 1075-1077. https://doi.org/10.1136/thoraxjnl-2013-203309

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AU - Alton, Eric W F W

AU - Boyd, A. Christopher

AU - Cheng, Seng H.

AU - Cunningham, Steve

AU - Davies, Jane C.

AU - Gill, Deborah R.

AU - Griesenbach, Uta

AU - Higgins, Tracy

AU - Hyde, Stephen C.

AU - Innes, J. Alastair

AU - Murray, Gordon D.

AU - Porteous, David J.

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AB - The UK Cystic Fibrosis Gene Therapy Consortium has been working towards clinical gene therapy for patients with cystic fibrosis for several years. We have recently embarked on a large, multi-dose clinical trial of a nonviral, liposome-based formulation powered for the first time to detect clinical benefit. The article describes the details of the protocol.

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A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis

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A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis

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