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REP1 Gene Replacement Therapy for Choroideremia (REGENERATE)

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2017 by University of Oxford
Sponsor:
Collaborators:
Moorfields Eye Hospital NHS Foundation Trust
University College, London
Information provided by (Responsible Party):
University of Oxford
ClinicalTrials.gov Identifier:
NCT02407678
First received: March 10, 2015
Last updated: February 9, 2017
Last verified: February 2017
  Purpose
The assessment of the efficacy (with respect to preservation of visual function and retinal structure) and safety of a single subretinal injection of AAV2.REP1 in participants with a confirmed diagnosis of choroideremia, as evaluated by various functional and anatomical outcomes measured over a number of time points up to 24 months post-treatment.

Condition Intervention Phase
Choroideremia
Genetic: AAV-mediated REP1 gene replacement
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
Official Title: An Open Label Phase 2 Clinical Trial of Retinal Gene Therapy for Choroideremia 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:
  • Change from baseline in best corrected visual acuity in the treated eye [ Time Frame: 2 years ]

Secondary Outcome Measures:
  • Change from baseline in the central visual field in the treated eye as determined by microperimetry [ Time Frame: 2 years ]
  • Change from baseline in the area of surviving retinal pigment epithelium in the treated eye as measured by fundus autofluorescence, compared to the untreated fellow eye (control eye) after randomisation of treatment to one eye or the other [ Time Frame: 2 years ]

Estimated Enrollment: 30
Study Start Date: August 2016
Estimated Primary Completion Date: August 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment
Treated eye undergoes AAV-mediated REP1 gene replacement. AAV vector is delivered by subretinal injection.
Genetic: AAV-mediated REP1 gene replacement
AAV vector carrying human REP1 gene is delivered into the treated eye by subretinal injection
No Intervention: Control
Untreated eye

  Eligibility

Ages Eligible for Study:   18 Years to 90 Years   (Adult, Senior)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Candidate is willing and able to give informed consent for participation in the study.
  2. Male aged 18 years or above.
  3. Genetic or molecular confirmed diagnosis of choroideremia (REP1 protein deficiency).
  4. Active disease visible clinically within the macula region.
  5. Best corrected visual acuity equal to or worse than 6/6 (20/20; Decimal 1.0; LogMAR 0) but better than or equal to 6/60 (20/200; Decimal 0.1; LogMAR 1.0) in the study eye.

Exclusion Criteria:

  1. Any female, or a male aged below 18 years.
  2. An additional cause for sight loss (e.g. amblyopia) in the eye to be treated.
  3. Any other significant ocular and non-ocular disease or disorder which, in the opinion of the investigator, may put the participants at risk because of participation in the study.
  4. Inability to take systemic prednisolone for a minimum of 3 weeks.
  5. Unwillingness to use barrier contraception methods for a period of three months following gene therapy surgery.
  6. Participation in another research study involving an investigational product in the preceding 12 weeks.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02407678

Contacts
Contact: Marco Bellini, PhD trials@eye.ox.ac.uk

Locations
United Kingdom
Moorfields Eye Hospital NHS Foundation Trust Recruiting
London, United Kingdom, EC1V 2PD
Contact: Lyndon da Cruz, MD DPhil         
Contact: Marco Bellini, PhD       trials@eye.ox.ac.uk   
Principal Investigator: Lyndon da Cruz, MD DPhil         
Oxford University Hospitals NHS Foundation Trust Recruiting
Oxford, United Kingdom, OX3 9DU
Contact: Marco Bellini, PhD       trials@eye.ox.ac.uk   
Principal Investigator: Robert E MacLaren, MB ChB DPhil         
Sponsors and Collaborators
University of Oxford
Moorfields Eye Hospital NHS Foundation Trust
University College, London
Investigators
Principal Investigator: Robert E MacLaren, MB ChB DPhil University of Oxford
  More Information

Responsible Party: University of Oxford
ClinicalTrials.gov Identifier: NCT02407678     History of Changes
Other Study ID Numbers: REGEN2015
Study First Received: March 10, 2015
Last Updated: February 9, 2017

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 21, 2017