Decentralized Dry Eye Study, Planning & Feasibility

PROJECT SUMMARY Dry eye disease (DED) is one of the most common chronic eye diseases. Traditional randomized clinical trials for DED are challenged with sample sizes too small to demonstrate effect, lack of diversity among participants, and insufficient correlation between objective markers and patient-reported symptoms. This proposal outlines a novel approach to studying evaporative loss DED through a decentralized clinical trial design. This non- interventional planning and feasibility proposal shifts the focus of DED study away from doctors’ offices and into participants’ home environments. Subjective evaporative loss DED symptoms are collected remotely, electronically, and sequentially. Self-collected ocular surface samples are collected in two ways: with a self- collected Schirmer strip and with a self-collected conjunctival swab. All study material is mailed to participants’ homes. Self-collected ocular surface samples are placed in study vials and return mailed to a central location, UCSF Proctor Foundation laboratory, for RNA-deep sequence exploratory analysis of the transcriptome as a biomarker for DED. To mimic repeat ocular surface collection after a future DED intervention, Schirmer strips and conjunctiva self-swabbing will be repeated after 4 weeks. This decentralized approach to DED study promotes patient engagement, recruitment, communication, and participant diversity and also seeks to identify new objective markers of DED efficacy that can be collected remotely. Specific aims of this R34 are (1) To develop a remote trial design allowing for decentralized recruitment and study of DED, (2) To prepare an operations manual and statistical analysis plan allowing for execution and scalability of a decentralized DED study design, and (3) To determine feasibility and utility of obtaining transcriptomic analysis from remotely collected Schirmer strips and conjunctiva self-swab collection. This planning grant leverages the power of decentralized trials to efficiently, economically, and objectively study one of the most encountered eye diseases, evaporative loss dry eye. Ultimately, this decentralized study platform will provide a foundation for future DED trials, facilitating the comparative re-evaluation of existing therapeutics and a standardized platform for assessing the efficacy of new dry eye treatments.