Validating candidate endpoints for intermediate age-related macular degeneration trials in a multi-centre setting—lessons from the MACUSTAR study
Terheyden, J. H., Dunbar, H. M. P., Schmitz-Valckenberg, S. , Behning, C., Martinho, C., Luhmann, U. F. O., Saßmannshausen, M., Lüning, A., Miliu, A., Aires, I. D., Basile, P. G., Batuca, J., Schmid, M., Moll, K-P., Zakaria, N., Tufail, A., Binns, A., Crabb, D. P. 
ORCID: 0000-0001-8754-3902, Leal, S., Finger, R. P., Holz, F. G., Agostini, H., Aires, I. D., Altay, L., Atia, R., Bandello, F., Basile, P. G., Batuca, J., Behning, C., Belmouhand, M., Berger, M., Binns, A. ORCID: 0000-0001-8621-498X, Boon, C. J. F., Böttger, M., Brazier, J. E., Carapezzi, C., Carlton, J., Carneiro, A., Charil, A., Coimbra, R., Cosette, D., Cozzi, M., Crabb, D. P., Cunha-Vaz, J., Dahlke, C., Dunbar, H., Finger, R. P., Fletcher, E., Gutfleisch, M., Hartgers, F., Higgins, B., Hildebrandt, J., Höck, E., Hogg, R., Holz, F. G., Hoyng, C. B., Kilani, A., Krätzschmar, J., Kühlewein, L., Larsen, M., Leal, S., Lechanteur, Y. T. E., Lu, D., Luhmann, U. F. O., Lüning, A., Manivannan, N., Marques, I., Martinho, C., Miliu, A., Moll, K. P., Mulyukov, Z., Paques, M., Parodi, B., Parravano, M., Penas, S., Peters, T., Peto, T., Priglinger, S., Ramamirtham, R., Ribeiro, R., Rowen, D., Rubin, G. S., Sahel, J., Sánchez, C., Sander, O., Saßmannshausen, M., Schmid, M., Schmitz-Valckenberg, S., Siedlecki, J., Silva, R., Souied, E., Staurenghi, G., Tavares, J., Taylor, D. J., Terheyden, J. H., Tufail, A., Valmaggia, P., Varano, M., Wolf, A. & Zakaria, N. (2025).
Validating candidate endpoints for intermediate age-related macular degeneration trials in a multi-centre setting—lessons from the MACUSTAR study.
Eye,
doi: 10.1038/s41433-024-03568-2
Abstract
For the conduct of future interventional age-related macular degeneration (AMD) trials, the availability of clinical study endpoints is key. However, no endpoints have been accepted by regulators for evaluation of treatment for intermediate (i) AMD, i.e. the AMD stage at highest risk of developing irreversible geographic atrophy or macular neovascularization. The European MACUSTAR consortium has recruited more than 700 individuals to develop and validate structural, functional and patient-reported endpoints, enabling future iAMD trials based on a prospective observational, multi-centre cohort study. Reliably assessing candidate endpoints in a setting that involves multiple clinical sites across countries comes with a plurality of challenges in the study set-up, quality of data, recruitment of participants and study conduct. Therefore, the MACUSTAR consortium has established a framework that successfully addresses these topics, provides relevant insights into the natural history of iAMD and its sub-phenotypes, and will open new regulatory pathways. The MACUSTAR study is registered on ClinicalTrials.gov under NCT03349801.
| Publication Type: | Article |
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| Additional Information: | This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. |
| Publisher Keywords: | Prognostic markers, Retinal diseases |
| Subjects: | R Medicine > RE Ophthalmology |
| Departments: | School of Health & Psychological Sciences School of Health & Psychological Sciences > Optometry & Visual Sciences |
| SWORD Depositor: |
Available under License Creative Commons: Attribution International Public License 4.0.
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