HONU: A multicenter, prospective, observational study of the progression of intermediate age-related macular degeneration

Abstract

Purpose : Treatments that effectively slow or prevent Age-related macular degeneration (AMD) progression to its advanced stages are urgently needed to prevent irreversible vision loss. However, large and lengthy clinical trials are necessary due to our inability to accurately identify high-risk individuals for such trials and lack of earlier endpoints. The purpose of this observational study is to inform the design of feasible interventional clinical trials for intermediate AMD (iAMD).

Methods : HONU is a prospective, multicenter observational study of patients with iAMD. Key eligibility criteria (study eye more than one large drusen >125 mm and pigmentary abnormalities and fellow eye with OCT atrophic changes) were chosen to enrich the study population for those more likely to have rapid disease progression. The three-year study length was designed to maximize the opportunity to capture disease progression and conversion from iAMD to more advanced AMD stages. Patients will be assessed every 12 weeks throughout the study with standard ophthalmologic examinations and a range of visual functional testing and multimodal retinal imaging. Patient Reported Outcome and biomarker samples (whole blood, plasma, and optional aqueous humor) will also be collected.

Results : HONU will enroll 400 high-risk patients across approximately 90 sites globally. The objective of the study is to assess iAMD disease progression by evaluating the timeline and rates of conversion from baseline iAMD to nascent geographic atrophy (nGA) and subsequently to geographic atrophy (GA). Moreover, anatomic and structural changes in multimodal imaging will be evaluated longitudinally and their relationship with visual function parameters studied. Novel algorithms will also be developed and samples assessed to investigate potential new biomarkers of progression.

Conclusions : The HONU study will provide robust natural history data on disease progression in iAMD patients needed to inform the design and improve the feasibility of future interventional studies in patients with iAMD, by investigating potential earlier clinical endpoints and developing an enrichment strategy for faster progressing patients.

This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.

 

Figure 1. HONU study design.