Developing patient-relevant outcome measures for clinical trials of sight-restoring interventions in very low and ultra-low vision

Promising new technologies and treatments for restoring vision are rapidly evolving, including pharmaceutical treatments, gene therapy, stem cells and retinal visual prostheses. As interventional trials are limited to persons who have little or no vision (so called ultra-low vision) any improvements in visual function are expected to be small and unlikely to be captured in conventional clinical measures such as letter chart visual acuity. Therefore, the evaluation of therapeutic efficacy in sight-restoration trials should –at least partly- rely on standardized, validated measures of vision-related quality of life (VRQoL) and instrumental activities of daily living (IDL). Our aim is to develop patient-relevant outcome measures which can be used as clinical endpoints in interventional clinical trials of sight restoring interventions in participants with ultra-low vision in the area of VRQoL and IADLs. A cohort up to 150 very low to ultra-low vision patients will be followed for 2 years. All participants will be interviewed with the Impact of Vision Impairment – Very Low Vision (IVI-VLV) questionnaire and complete the IADL-VLV under observation. We will assess IVI-VLV’s and IADL-VLV’s test-retest reliability and refine and shorten the IADL-VLV testing protocol. We will also assess the IVI-VLV’s and IADL-VLV’s responsiveness to change in vision over time.

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