Validating candidate endpoints for intermediate age-related macular degeneration trials in a multi-centre...

Area of Science:

• Ophthalmology
• Clinical Trials
• Biomarkers

Background:

• Regulatory acceptance of clinical study endpoints is lacking for intermediate age-related macular degeneration (iAMD).
• iAMD represents a critical stage with high risk for irreversible vision loss.
• Standardized endpoints are needed to evaluate treatments for iAMD.

Purpose of the Study:

• To develop and validate structural, functional, and patient-reported endpoints for intermediate age-related macular degeneration (iAMD) trials.
• To establish a framework for conducting multi-centre iAMD clinical studies.
• To provide insights into the natural history and sub-phenotypes of iAMD.

Main Methods:

• Prospective observational, multi-centre cohort study.
• Recruitment of over 700 participants with iAMD.
• Development and validation of candidate clinical outcome assessments.

Main Results:

• A framework was successfully established to address challenges in multi-centre iAMD study conduct.
• Insights into the natural history and sub-phenotypes of iAMD were gained.
• The study paves the way for regulatory acceptance of new iAMD endpoints.

Conclusions:

• The MACUSTAR consortium successfully developed and validated endpoints for intermediate age-related macular degeneration (iAMD).
• The established framework facilitates future iAMD clinical trials.
• This work will enable regulatory pathways for novel iAMD treatments.