Imperial College London
Alternate

ProfessorUtaGriesenbach

Faculty of Medicine, National Heart & Lung Institute

Professor of Molecular Medicine
 
 
 
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Contact

 

+44 (0)20 7594 7927u.griesenbach

 
 
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Assistant

 

Miss Samia Soussi +44 (0)20 7594 7980

 
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Location

 

Emmanuel Kaye BuildingRoyal Brompton Campus

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Summary

 

Publications

Citation

BibTex format

@article{Alton:2015:10.1016/S2213-2600(15)00245-3,
author = {Alton, EWFW and Armstrong, DK and Ashby, D and Alton, EWFW and Armstrong, DK and Ashby, D and Bayfield, KJ and Bilton, D and Bloomfield, EV and Boyd, AC and Brand, J and Buchan, R and Calcedo, R and Carvelli, P and Chan, M and Cheng, SH and Collie, DDS and Cunningham, S and Davidson, HE and Davies, G and Davies, JC and Davies, LA and Dewar, MH and Doherty, A and Donovan, J and Dwyer, NS and Elgmati, HI and Featherstone, RF and Gavino, J and Gea-Sorli, S and Geddes, DM and Gibson, JSR and Gill, DR and Greening, AP and Griesenbach, U and Hansell, DM and Harman, K and Higgins, TE and Hodges, SL and Hyde, SC and Hyndman, L and Innes, JA and Jacob, J and Jones, N and Keogh, BF and Limberis, MP and Lloyd-Evans, P and Maclean, AW and Manvell, MC and McCormick, D and McGovern, M and McLachlan, G and Meng, C and Montero, MA and Milligan, H and Moyce, LJ and Murray, GD and Nicholson, AG and Osadolor, T and Parra-Leiton, J and Porteous, DJ and Pringle, IA and Punch, EK and Pytel, KM and Quittner,},
doi = {10.1016/S2213-2600(15)00245-3},
journal = {The Lancet Respiratory Medicine},
pages = {684--691},
title = {Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial},
url = {http://dx.doi.org/10.1016/S2213-2600(15)00245-3},
volume = {3},
year = {2015}
}
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TY  - JOUR
AB  - BackgroundLung 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.MethodsWe 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.FindingsBetween 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.InterpretationMonthly application of the pGM169/GL67A gene therapy formulation was associated with a significant, albeit modest, benefit in FEV1 compared with placebo at 1 yea
AU  - Alton,EWFW
AU  - Armstrong,DK
AU  - Ashby,D
AU  - Alton,EWFW
AU  - Armstrong,DK
AU  - Ashby,D
AU  - Bayfield,KJ
AU  - Bilton,D
AU  - Bloomfield,EV
AU  - Boyd,AC
AU  - Brand,J
AU  - Buchan,R
AU  - Calcedo,R
AU  - Carvelli,P
AU  - Chan,M
AU  - Cheng,SH
AU  - Collie,DDS
AU  - Cunningham,S
AU  - Davidson,HE
AU  - Davies,G
AU  - Davies,JC
AU  - Davies,LA
AU  - Dewar,MH
AU  - Doherty,A
AU  - Donovan,J
AU  - Dwyer,NS
AU  - Elgmati,HI
AU  - Featherstone,RF
AU  - Gavino,J
AU  - Gea-Sorli,S
AU  - Geddes,DM
AU  - Gibson,JSR
AU  - Gill,DR
AU  - Greening,AP
AU  - Griesenbach,U
AU  - Hansell,DM
AU  - Harman,K
AU  - Higgins,TE
AU  - Hodges,SL
AU  - Hyde,SC
AU  - Hyndman,L
AU  - Innes,JA
AU  - Jacob,J
AU  - Jones,N
AU  - Keogh,BF
AU  - Limberis,MP
AU  - Lloyd-Evans,P
AU  - Maclean,AW
AU  - Manvell,MC
AU  - McCormick,D
AU  - McGovern,M
AU  - McLachlan,G
AU  - Meng,C
AU  - Montero,MA
AU  - Milligan,H
AU  - Moyce,LJ
AU  - Murray,GD
AU  - Nicholson,AG
AU  - Osadolor,T
AU  - Parra-Leiton,J
AU  - Porteous,DJ
AU  - Pringle,IA
AU  - Punch,EK
AU  - Pytel,KM
AU  - Quittner,AL
AU  - Rivellini,G
AU  - Saunders,CJ
AU  - Scheule,RK
AU  - Sheard,S
AU  - Simmonds,NJ
AU  - Smith,K
AU  - Smith,SN
AU  - Soussi,N
AU  - Soussi,S
AU  - Spearing,EJ
AU  - Stevenson,BJ
AU  - Sumner-Jones,SG
AU  - Turkkila,M
AU  - Ureta,RP
AU  - Waller,MD
AU  - Wasowicz,MY
AU  - Wilson,JM
AU  - Wolstenholme-Hogg,P
DO  - 10.1016/S2213-2600(15)00245-3
EP  - 691
PY  - 2015///
SN  - 2213-2600
SP  - 684
TI  - Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
T2  - The Lancet Respiratory Medicine
UR  - http://dx.doi.org/10.1016/S2213-2600(15)00245-3
UR  - http://gateway.webofknowledge.com/gateway/Gateway.cgi?GWVersion=2&SrcApp=PARTNER_APP&SrcAuth=LinksAMR&KeyUT=WOS:000360981500006&DestLinkType=FullRecord&DestApp=ALL_WOS&UsrCustomerID=1ba7043ffcc86c417c072aa74d649202
UR  - http://hdl.handle.net/10044/1/28157
VL  - 3
ER  -
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