Project Details
Projekt
Developing patient-relevant outcome measures for clinical trials of sight-restoring interventions in very low and ultra-low vision
Applicant
Professor Robert Patrick Finger, Ph.D.
Subject Area
Ophthalmology
Epidemiology and Medical Biometry/Statistics
Epidemiology and Medical Biometry/Statistics
Term
from 2018 to 2023
Project identifier
Deutsche Forschungsgemeinschaft (DFG) - Project number 399435571
Promising new technologies and treatments for restoring vision in inherited retinal dystrophies (IRDs) are rapidly evolving, including gene therapy, stem cells and retinal visual prostheses. However, testing the efficacy of these innovations is challenging as conventionally used clinical endpoints such as letter chart visual acuity or visual field testing are inadequate to capture the very small functional benefits expected with these interventions. Real-world functioning and quality of life (QoL), however, can be improved by even the small improvements in visual function expected with these interventions and thus should be captured in clinical trials. In addition, they reflect the real benefit for patients gained through these interventions. Against this background we developed two measures of real-world functioning and QoL, the Instrumental Activities of Daily Living – Very Low Vision (IADL-VLV) and the Impact of Vision Impairment – Very Low Vision (IVI-VLVL). The first is a standardized measure of observed performance on several real-world tasks and the latter is a standardized measure of vision-related QoL. These were developed with persons with low or ultra-low vision due to IRDs. In this project we will further validate these two outcome measures as clinical endpoints by assessing measurement precision and responsiveness to change in function over time by following a group of patients with IRDs for two years. This will allow us to establish a minimal clinically relevant change which will be important for the design of future interventional trials. Following best practice will guarantee that the outcome measures are not only relevant to patients but also acceptable to regulatory agencies. At the end of this project we will have two ready to use clinical endpoints which will allow for a fast translation of novel sight restoration interventions into clinical research and ultimately practice.
DFG Programme
Priority Programmes
