Ophthalmology

Natural History Study of Developmental Retinal Disorders in Preparation for Clinical Trials

Supervisors: Dr Mervyn Thomas (mt350@leicester.ac.uk)  ,Dr Zhanhan Tu (zhanhan.tu@leicester.ac.uk) and Artur Boronat (artur.boronat@leicester.ac.uk)

This project proposes a comprehensive natural history study of developmental foveal disorders, aimed at understanding disease progression and identifying critical time points for intervention. By collecting longitudinal data from patients with these disorders, the study will establish a detailed baseline of disease trajectories, which is essential for designing future clinical trials. This data will inform the selection of endpoints, patient stratification criteria, and timing of interventions.

The project will also involve creating a biorepository of patient samples for future genetic and biomarker studies, using state-of-the-art high-resolution imaging techniques for novel biomarker characterization. The ACF will gain valuable experience in longitudinal research, data analysis, and clinical trial preparation, positioning them strongly for fellowship funding in ophthalmology and rare disease research.

Additionally, the ACF will have the opportunity to develop and validate objective core outcome sets (COS) for ophthalmology clinical trials, focusing on high-resolution imaging techniques and patient and public involvement and engagement (PPIE) approaches. Standardising outcome measures across studies will enhance the quality of evidence and ensure patient perspectives are central to trial design and implementation, ultimately improving the relevance and impact of future clinical trials.