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Completed

NCT Number: NCT02407678

REP1 Gene Replacement Therapy for Choroideremia

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.

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Key information

Conditions

Choroideremia Choroid Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Genetic Diseases, X-Linked Uveal Diseases

Age range

18 year–90 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2

Primary location

Moorfields Eye Hospital NHS Foundation Trust, London, United Kingdom

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Candidate is willing and able to give informed consent for participation in the study.
  • Male aged 18 years or above.
  • Genetic or molecular confirmed diagnosis of choroideremia (REP1 protein deficiency).
  • Active disease visible clinically within the macula region.
  • 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

  • Any female, or a male aged below 18 years.
  • An additional cause for sight loss (e.g. amblyopia) in the eye to be treated.
  • 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.
  • Inability to take systemic prednisolone for a period of 45 days.
  • Unwillingness to use barrier contraception methods for a period of three months following gene therapy surgery.
  • Participation in another research study involving an investigational product in the preceding 12 weeks.

Treatment and study plan

AAV-mediated REP1 gene replacement

Genetic

AAV vector carrying human REP1 gene is delivered into the treated eye by subretinal injection

Primary outcomes

  1. Change from baseline in best corrected visual acuity in the treated eye

    Time frame: 2 years

Secondary outcomes

  1. Change from baseline in the central visual field in the treated eye as determined by microperimetry

    Time frame: 2 years

  2. 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

Sponsors and collaborators

Lead sponsor

University of Oxford

Other

Collaborators

  • Moorfields Eye Hospital NHS Foundation Trust
  • University College, London

Registry information

Official study 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)

Acronym: REGENERATE

Important dates

Study start
2016
Primary completion
2021
Study completion
2021
First posted
Apr 3, 2015
Registry last updated
Aug 5, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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