Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE1/PHASE2
14 participants
INTERVENTIONAL
2011-10-31
2017-10-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
\- Secondary Objective: To identify any therapeutic benefit as evidenced by a slowing down of the retinal degeneration assessed by functional and anatomical methods in the treated eye compared to the control eye 24 months after gene delivery.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial, The Lancet, Volume 383, Issue 9923, Pages 1129 - 1137 (29 March 2014).
Links: www.thelancet.com/journals/lancet/article/PIIS0140-6736(13)62117-0/abstract ; http://dx.doi.org/doi:10.1016/S0140-6736(13)62117-0
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Keywords
Explore important study keywords that can help with search, categorization, and topic discovery.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Dose 1
Dose 1 = single subretinal injection of vector suspension containing approximately 10e10 rAAV2.REP1 genome particles. Six patients have now received Dose 1.
rAAV2.REP1
Single subretinal injection of rAAV2.REP1 vector suspension containing 10e12 genome particles per ml. Dose 1 = dose containing approximately 10e10 rAAV2.REP1 genome particles. Dose 2 = dose containing approximately 10e11 rAAV2.REP1 genome particles.
Dose 2
Dose 2 = single subretinal injection of vector suspension containing approximately 10e11 rAAV2.REP1 genome particles. Three patients thus far have received Dose 2.
rAAV2.REP1
Single subretinal injection of rAAV2.REP1 vector suspension containing 10e12 genome particles per ml. Dose 1 = dose containing approximately 10e10 rAAV2.REP1 genome particles. Dose 2 = dose containing approximately 10e11 rAAV2.REP1 genome particles.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
rAAV2.REP1
Single subretinal injection of rAAV2.REP1 vector suspension containing 10e12 genome particles per ml. Dose 1 = dose containing approximately 10e10 rAAV2.REP1 genome particles. Dose 2 = dose containing approximately 10e11 rAAV2.REP1 genome particles.
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Male aged 18 years or above,
* Diagnosed with choroideraemia and in good health,
* Active disease with SLO changes visible within the macula region,
* Willing to allow his or her General Practitioner and consultant, if appropriate, to be notified of participation in the study,
* Vision at least 6/60 or better in the study eye.
Exclusion Criteria
* Men unwilling to use barrier contraception methods, if relevant,
* Previous history of retinal surgery or ocular inflammatory disease (uveitis),
* Grossly asymmetrical disease or other ocular morbidity which might confound use of the fellow eye as a long-term control,
* Any other significant disease or disorder which, in the opinion of the Investigator, may either put the participants at risk because of participation in the study, or may influence the result of the study, or the participant's ability to participate in the study,
* Participants who have participated in another research study involving an investigational product in the previous 12 weeks.
18 Years
MALE
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Oxford University Hospitals NHS Trust
OTHER
Moorfields Eye Hospital NHS Foundation Trust
OTHER
University College, London
OTHER
Manchester University NHS Foundation Trust
OTHER_GOV
University of Manchester
OTHER
University Hospital Southampton NHS Foundation Trust
OTHER
University of Southampton
OTHER
University of Oxford
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Robert E MacLaren, MB ChB DPhil
Role: STUDY_CHAIR
University of Oxford, Oxford Radcliffe Hospitals NHS Trust and Moorfields Eye Hospital
Miguel C Seabra, MD PhD
Role: PRINCIPAL_INVESTIGATOR
Imperial College London
Andrew R Webster, MD
Role: PRINCIPAL_INVESTIGATOR
UCL Institute of Ophthalmology and Moorfields Eye Hospital
Susan M Downes, MD
Role: PRINCIPAL_INVESTIGATOR
Oxford University Hospitals NHS Trust
Graeme C Black, MB BCh DPhil
Role: PRINCIPAL_INVESTIGATOR
University of Manchester and Central Manchester University Hospitals NHS Foundation Trust
Andrew J Lotery, MD
Role: PRINCIPAL_INVESTIGATOR
University of Southampton and Southampton University Hospitals Trust
Len W Seymour, PhD
Role: PRINCIPAL_INVESTIGATOR
University of Oxford
Tanya Tolmachova, PhD
Role: PRINCIPAL_INVESTIGATOR
Imperial College London
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Moorfields Eye Hospital NHS Foundation Trust
London, , United Kingdom
St Mary's Hospital, Central Manchester University Hospitals NHS Foundation Trust
Manchester, , United Kingdom
Oxford Radcliffe Hospitals NHS Trust
Oxford, , United Kingdom
Eye Unit, Southampton University Hospitals NHS Trust
Southampton, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Simunovic MP, Jolly JK, Xue K, Edwards TL, Groppe M, Downes SM, MacLaren RE. The Spectrum of CHM Gene Mutations in Choroideremia and Their Relationship to Clinical Phenotype. Invest Ophthalmol Vis Sci. 2016 Nov 1;57(14):6033-6039. doi: 10.1167/iovs.16-20230.
Xue K, Oldani M, Jolly JK, Edwards TL, Groppe M, Downes SM, MacLaren RE. Correlation of Optical Coherence Tomography and Autofluorescence in the Outer Retina and Choroid of Patients With Choroideremia. Invest Ophthalmol Vis Sci. 2016 Jul 1;57(8):3674-84. doi: 10.1167/iovs.15-18364.
Seitz IP, Zhour A, Kohl S, Llavona P, Peter T, Wilhelm B, Zrenner E, Ueffing M, Bartz-Schmidt KU, Fischer MD. Multimodal assessment of choroideremia patients defines pre-treatment characteristics. Graefes Arch Clin Exp Ophthalmol. 2015 Dec;253(12):2143-50. doi: 10.1007/s00417-015-2976-4. Epub 2015 Mar 7.
MacLaren RE, Groppe M, Barnard AR, Cottriall CL, Tolmachova T, Seymour L, Clark KR, During MJ, Cremers FP, Black GC, Lotery AJ, Downes SM, Webster AR, Seabra MC. Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. Lancet. 2014 Mar 29;383(9923):1129-37. doi: 10.1016/S0140-6736(13)62117-0. Epub 2014 Jan 16.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2009-014617-27
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
CHM09/01
Identifier Type: -
Identifier Source: org_study_id