Trial record 14 of 50 for:
Open Studies | "Blindness"
Gene Therapy for Blindness Caused by Choroideremia
This study is currently recruiting participants.
Verified October 2011 by University of Oxford
Sponsor:
University of Oxford
Collaborators:
Oxford University Hospitals NHS Trust
Moorfields Eye Hospital NHS Foundation Trust
University College, London
Central Manchester University Hospitals NHS Foundation Trust
University of Manchester
University Hospital Southampton NHS Foundation Trust.
University of Southampton
Information provided by (Responsible Party):
University of Oxford
ClinicalTrials.gov Identifier:
NCT01461213
First received: October 21, 2011
Last updated: October 27, 2011
Last verified: October 2011
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Purpose
- Primary objective: To assess the safety and tolerability of the AAV.REP1 vector, administered at two different doses to the retina in 12 patients with a diagnosis of choroideremia.
- 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.
| Condition | Intervention | Phase |
|---|---|---|
|
Choroideremia |
Drug: rAAV2.REP1 |
Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | An Open Label Dose Escalation Phase 1 Clinical Trial of Retinal Gene Therapy for Choroideraemia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1) |
Resource links provided by NLM:
Genetics Home Reference related topics:
choroideremia
Lenz microphthalmia syndrome
oculofaciocardiodental syndrome
Peters plus syndrome
retinitis pigmentosa
U.S. FDA Resources
Further study details as provided by University of Oxford:
Primary Outcome Measures:
- Visual acuity [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]Best corrected visual acuity, following cataract surgery if indicated
Secondary Outcome Measures:
- Microperimetry, OCT and fundus autofluorescence [ Time Frame: 24 months ] [ Designated as safety issue: No ]Structure function correlations at the margins of the retinal degeneration
| Estimated Enrollment: | 12 |
| Study Start Date: | October 2011 |
| Estimated Study Completion Date: | June 2016 |
| Estimated Primary Completion Date: | December 2014 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Dose 1
Injection of up to 10e10 genome particles
|
Drug: rAAV2.REP1
Single subretinal injection of vector suspension containing 10e12 genome particles per ml
Other Name: Adeno-associated viral vector
|
|
Experimental: Dose 2
Injection of up to 10e11 genome particles
|
Drug: rAAV2.REP1
Single subretinal injection of vector suspension containing 10e12 genome particles per ml
Other Name: Adeno-associated viral vector
|
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Participant is willing and able to give informed consent for participation in the study,
- 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:
- Female and child participants (under the age of 18),
- 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.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01461213
Contacts
| Contact: Robert E MacLaren | enquiries@eye.ox.ac.uk |
Locations
| United Kingdom | |
| Moorfields Eye Hospital NHS Foundation Trust | Recruiting |
| London, United Kingdom, EC1V 2PD | |
| Contact 020 7566 2260 | |
| Principal Investigator: Andrew R Webster | |
| St Mary's Hospital, Central Manchester University Hospitals NHS Foundation Trust | Recruiting |
| Manchester, United Kingdom, M13 9WL | |
| Contact 0161 276 6269 | |
| Principal Investigator: Graeme C Black | |
| Oxford Radcliffe Hospitals NHS Trust | Recruiting |
| Oxford, United Kingdom, OX3 9DU | |
| Contact 01865 231578 | |
| Principal Investigator: Susan Downes | |
| Eye Unit, Southampton University Hospitals NHS Trust | Recruiting |
| Southampton, United Kingdom, SO16 6YD | |
| Contact 023 8079 4590 | |
| Principal Investigator: Andrew J Lotery | |
Sponsors and Collaborators
University of Oxford
Oxford University Hospitals NHS Trust
Moorfields Eye Hospital NHS Foundation Trust
University College, London
Central Manchester University Hospitals NHS Foundation Trust
University of Manchester
University Hospital Southampton NHS Foundation Trust.
University of Southampton
Investigators
| Study Chair: | Robert E MacLaren, MB ChB DPhil | University of Oxford, Oxford Radcliffe Hospitals NHS Trust and Moorfields Eye Hospital |
| Principal Investigator: | Miguel C Seabra, MD PhD | Imperial College London |
| Principal Investigator: | Andrew R Webster, MD | UCL Institute of Ophthalmology and Moorfields Eye Hospital |
| Principal Investigator: | Susan M Downes, MD | Oxford University Hospitals NHS Trust |
| Principal Investigator: | Graeme C Black, MB BCh DPhil | University of Manchester and Central Manchester University Hospitals NHS Foundation Trust |
| Principal Investigator: | Andrew J Lotery, MD | University of Southampton and Southampton University Hospitals Trust |
| Principal Investigator: | Len W Seymour, PhD | University of Oxford |
| Principal Investigator: | Tanya Tolmachova, PhD | Imperial College London |
More Information
No publications provided
| Responsible Party: | University of Oxford |
| ClinicalTrials.gov Identifier: | NCT01461213 History of Changes |
| Other Study ID Numbers: | CHM09/01, 2009-014617-27 |
| Study First Received: | October 21, 2011 |
| Last Updated: | October 27, 2011 |
| Health Authority: | United Kingdom: Medicines and Healthcare Products Regulatory Agency United Kingdom: National Health Service United Kingdom: National Institute for Health Research |
Keywords provided by University of Oxford:
|
Tapetoretinal degeneration, choroideraemia, X-linked retinitis pigmentosa |
Additional relevant MeSH terms:
|
Choroideremia Eye Diseases, Hereditary Eye Diseases Choroid Diseases |
Uveal Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |
ClinicalTrials.gov processed this record on June 18, 2013