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Published in: Trials 1/2020

Open Access 01-12-2020 | Macular Degeneration | Study protocol

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

Authors: Jan H. Terheyden, Frank G. Holz, Steffen Schmitz-Valckenberg, Anna Lüning, Matthias Schmid, Gary S. Rubin, Hannah Dunbar, Adnan Tufail, David P. Crabb, Alison Binns, Clara I. Sánchez, Carel Hoyng, Philippe Margaron, Nadia Zakaria, Mary Durbin, Ulrich Luhmann, Parisa Zamiri, José Cunha-Vaz, Cecília Martinho, Sergio Leal, Robert P. Finger, on behalf of the MACUSTAR consortium

Published in: Trials | Issue 1/2020

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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
Appendix
Available only for authorised users
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Metadata
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
Authors
Jan H. Terheyden
Frank G. Holz
Steffen Schmitz-Valckenberg
Anna Lüning
Matthias Schmid
Gary S. Rubin
Hannah Dunbar
Adnan Tufail
David P. Crabb
Alison Binns
Clara I. Sánchez
Carel Hoyng
Philippe Margaron
Nadia Zakaria
Mary Durbin
Ulrich Luhmann
Parisa Zamiri
José Cunha-Vaz
Cecília Martinho
Sergio Leal
Robert P. Finger
on behalf of the MACUSTAR consortium
Publication date
01-12-2020
Publisher
BioMed Central
Published in
Trials / Issue 1/2020
Electronic ISSN: 1745-6215
DOI
https://doi.org/10.1186/s13063-020-04595-6

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