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Review
. 2019;241(2):61-72.
doi: 10.1159/000491402. Epub 2018 Aug 28.

MACUSTAR: Development and Clinical Validation of Functional, Structural, and Patient-Reported Endpoints in Intermediate Age-Related Macular Degeneration

Robert P Finger  1 , Steffen Schmitz-Valckenberg  2 , Matthias Schmid  3 , Gary S Rubin  4 , Hannah Dunbar  4 , Adnan Tufail  5 , David P Crabb  6 , Alison Binns  6 , Clara I Sánchez  7 , Philippe Margaron  8 , Guillaume Normand  8 , Mary K Durbin  9 , Ulrich F O Luhmann  10 , Parisa Zamiri  8 , José Cunha-Vaz  11 , Friedrich Asmus  12 , Frank G Holz  2 ; on behalf of the MACUSTAR consortium
Affiliations
Review

MACUSTAR: Development and Clinical Validation of Functional, Structural, and Patient-Reported Endpoints in Intermediate Age-Related Macular Degeneration

Robert P Finger et al. Ophthalmologica. 2019.

Abstract

Purpose: Currently, no outcome measures are clinically validated and accepted as clinical endpoints by regulatory agencies for drug development in intermediate age-related macular degeneration (iAMD). The MACUSTAR Consortium, a public-private research group funded by the European Innovative Medicines Initiative intends to close this gap.

Procedures: Development of study protocol and statistical analysis plan including predictive modelling of multimodal endpoints based on a review of the literature and expert consensus.

Results: This observational study consists of a cross-sectional and a longitudinal part. Functional outcome measures assessed under low contrast and low luminance have the potential to detect progression of visual deficit within iAMD and to late AMD. Structural outcome measures will be multimodal and investigate topographical relationships with function. Current patient-reported outcome measures (PROMs) are not acceptable to regulators and may not capture the functional deficit specific to iAMD with needed precision, justifying development of novel PROMs for iAMD. The total sample size will be n = 750, consisting mainly of subjects with iAMD (n = 600).

Conclusions: As clinical endpoints currently accepted by regulators cannot detect functional loss or patient-relevant impact in iAMD, we will clinically validate novel candidate endpoints for iAMD.

Keywords: Clinical endpoint; Disease progression; Intermediate age-related macular degeneration; Patient-reported outcomes; Structure-function correlation.

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Figures

Fig. 1
Fig. 1
Overall structure of the MACUSTAR clinical study.
See this image and copyright information in PMC

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