MACUSTAR: Development and Clinical Validation of Functional, Structural, and Patient-Reported Endpoints in Intermediate Age-Related Macular Degeneration
| Autor: | Parisa Zamiri, Clara I. Sánchez, Frank G. Holz, Steffen Schmitz-Valckenberg, Matthias Schmid, Alison M. Binns, Mary Durbin, José Cunha-Vaz, Ulrich F O Luhmann, Adnan Tufail, Hannah Dunbar, Guillaume Normand, Friedrich Asmus, Robert P. Finger, Philippe Margaron, David P. Crabb, Gary S. Rubin |
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| Rok vydání: | 2018 |
| Předmět: |
medicine.medical_specialty
Fundus Oculi Visual Acuity 01 natural sciences Retina Sensory disorders Donders Center for Medical Neuroscience [Radboudumc 12] Macular Degeneration 03 medical and health sciences 0302 clinical medicine Statistical Analysis Plan Physical medicine and rehabilitation Clinical endpoint Humans Medicine Patient Reported Outcome Measures Fluorescein Angiography 0101 mathematics Protocol (science) business.industry 010102 general mathematics Disease Management General Medicine Macular degeneration medicine.disease Sensory Systems 3. Good health Ophthalmology Drug development Sample size determination 030221 ophthalmology & optometry Observational study RE business Tomography Optical Coherence Predictive modelling Research Article |
| Zdroj: | Ophthalmologica Ophthalmologica; International Journal of Ophthalmology, 241, 2, pp. 61-72 Ophthalmologica; International Journal of Ophthalmology, 241, 61-72 |
| ISSN: | 0030-3755 |
| DOI: | 10.1159/000491402 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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