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This is a multi-centre, non-randomised trial of gene therapy in those with the condition choroidaemia.

BRIEF SUMMARY

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.

Primary Outcome 

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

Secondary Outcome 

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


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