Terheyden, JH;
Holz, FG;
Schmitz-Valckenberg, S;
Luening, A;
Schmid, M;
Rubin, GS;
Dunbar, H;
... Finger, RP; + view all
(2020)
Clinical study protocol for a low-interventional study in intermediate age-related macular degeneration developing novel clinical endpoints for interventional clinical trials with a regulatory and patient access intention-MACUSTAR.
Trials
, 21
, Article 659. 10.1186/s13063-020-04595-6.
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Abstract
Background: There is an unmet need for treatment options in intermediate age-related macular degeneration (iAMD). However, for any new interventions to be tested in clinical trials, novel currently unavailable clinical endpoints need to be developed. Thus, the MACUSTAR study aims to develop and evaluate functional, structural, and patient-reported candidate endpoints for use in future iAMD trials. Methods: The protocol describes a low-interventional clinical multicenter study employing a novel two-part design. The cross-sectional part (total duration, 1 month) and the longitudinal part (total duration, 36 months) include participants with iAMD and control groups with early/late/no AMD. The cross-sectional part’s primary objective is a technical evaluation of functional, structural, and patient-reported candidate outcomes. The longitudinal part’s primary objective is to assess the prognostic power of changes in functional, structural, and patient-reported outcomes for progression from iAMD to late AMD. All data will be used to support a biomarker qualification procedure by regulatory authorities. Discussion: The MACUSTAR study characterizes and evaluates much needed novel functional, structural, and patient-reported endpoints for future clinical trials in iAMD and will improve our understanding of the natural history and prognostic markers of this condition. Trial registration: ClinicalTrials.gov NCT03349801. Registered on 22 November 2017
| Type: | Article |
|---|---|
| Title: | Clinical study protocol for a low-interventional study in intermediate age-related macular degeneration developing novel clinical endpoints for interventional clinical trials with a regulatory and patient access intention-MACUSTAR |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1186/s13063-020-04595-6 |
| Publisher version: | https://doi.org/10.1186/s13063-020-04595-6 |
| Language: | English |
| Additional information: | This work is licensed under a Creative Commons Attribution 4.0 International License. The images or other third party material in this article are included in the Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/ |
| Keywords: | Science & Technology, Life Sciences & Biomedicine, Medicine, Research & Experimental, Research & Experimental Medicine, Intermediate age-related macular degeneration, Clinical study protocol, Disease progression, Clinical endpoint, VISUAL-ACUITY LOSS, NATURAL-HISTORY, LOW LUMINANCE, GEOGRAPHIC ATROPHY, DARK-ADAPTATION, MICROPERIMETRY, PREVALENCE, DRUSEN, BIOMARKERS, MORPHOLOGY |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > Institute of Ophthalmology |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10109195 |
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