Gene Therapy for Blindness Caused by Choroideremia

This study is currently recruiting participants.
Verified October 2011 by University of Oxford
Sponsor:
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
  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:


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 by University of Oxford

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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 April 23, 2014