MACUSTAR: Development and Clinical Validation of Functional, Structural, and Patient-Reported Endpoints in Intermediate Age-Related Macular Degeneration
| العنوان: | MACUSTAR: Development and Clinical Validation of Functional, Structural, and Patient-Reported Endpoints in Intermediate Age-Related Macular Degeneration |
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| المؤلفون: | 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 |
| المصدر: | Ophthalmologica Ophthalmologica; International Journal of Ophthalmology, 241, 2, pp. 61-72 Ophthalmologica; International Journal of Ophthalmology, 241, 61-72 |
| سنة النشر: | 2018 |
| مصطلحات موضوعية: | 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 |
| الوصف: | 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. |
| وصف الملف: | application/pdf |
| تدمد: | 0030-3755 |
| DOI: | 10.1159/000491402 |
| URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::99ede0633be028ac361c5f4d507afb09 |
| Rights: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....99ede0633be028ac361c5f4d507afb09 |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 00303755 |
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| DOI: | 10.1159/000491402 |