REP1 Gene Replacement Therapy for Choroideremia (REGENERATE)

August 4, 2021 updated by: University of Oxford

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)

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

30

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
      • London, United Kingdom, EC1V 2PD
        • Moorfields Eye Hospital NHS Foundation Trust
      • Oxford, United Kingdom, OX3 9DU
        • Oxford University Hospitals NHS Foundation Trust

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years to 88 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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 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 period of 45 days.
  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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change from baseline in best corrected visual acuity in the treated eye
Time Frame: 2 years
2 years

Secondary Outcome Measures

Outcome Measure
Time Frame
Change from baseline in the central visual field in the treated eye as determined by microperimetry
Time Frame: 2 years
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
2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Oxford

Collaborators

University College, London
Moorfields Eye Hospital NHS Foundation Trust

Investigators

  • Principal Investigator: Robert E MacLaren, MB ChB DPhil, University of Oxford

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 16, 2016

Primary Completion (Actual)

July 23, 2021

Study Completion (Actual)

July 23, 2021

Study Registration Dates

First Submitted

March 10, 2015

First Submitted That Met QC Criteria

March 30, 2015

First Posted (Estimate)

April 3, 2015

Study Record Updates

Last Update Posted (Actual)

August 5, 2021

Last Update Submitted That Met QC Criteria

August 4, 2021

Last Verified

July 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • REGEN2015

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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