Designing the next generation of clinical trials in intermediate AMD-a consensus driven, pragmatic, proof of concept early intervention study.

A greater understanding of the pathophysiology of AMD has yielded potential therapeutic strategies for slowing vision loss. Structural imaging biomarkers and novel functional outcomes, both under intensive study as surrogate endpoints in AMD clinical trials, are new granular tools needed to assess disease progression and follow response to therapy. These advances have created, for the first time, the possibility to intervene early in AMD, and to measure success in a time frame feasible for clinical trials. At the 2024 Ryan Initiative for Macular Research (RIMR) meeting, a consensus was reached for design of a hypothetical, pragmatic, proof of concept, 24-month, early intervention study to determine the efficacy of an intervention in subjects with intermediate AMD (iAMD). Although further research is needed, using natural history cohorts and intervention trials to validate the proposed trial design and endpoints, this paper presents an AMD expert consensus framework for early AMD clinical trials, that we hope will also be useful for therapeutic developers and regulators. Viable clinical trial strategies are needed to develop treatments that slow the progression of AMD prior to the development of vision-threatening late AMD; this consensus document should aid in reaching this goal.

Duke Scholars

Author Glenn Jay Jaffe Ophthalmology, Vitreoretinal Diseases & Surgery