. 2020 Jul 18;21(1):659.

doi: 10.1186/s13063-020-04595-6.

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

Jan H Terheyden¹, Frank G Holz¹, Steffen Schmitz-Valckenberg¹, Anna Lüning¹, Matthias Schmid², Gary S Rubin³, Hannah Dunbar³, Adnan Tufail⁴, David P Crabb⁵, Alison Binns⁵, Clara I Sánchez⁶, Carel Hoyng⁶, Philippe Margaron⁷, Nadia Zakaria⁷, Mary Durbin⁸, Ulrich Luhmann⁹, Parisa Zamiri⁷, José Cunha-Vaz¹⁰, Cecília Martinho¹⁰, Sergio Leal¹¹, Robert P Finger¹²; MACUSTAR consortium

Collaborators, Affiliations

Collaborators

MACUSTAR consortium:
P Basile, C Behning, M Berger, A Binns, M Böttger, C Bouchet, J E Brazier, T Butt, C Carapezzi, J Carlton, A Charil, R Coimbra, S Nunes, D Crabb, J Cunha-Vaz, H Dunbar, M Durbin, R Finger, F Holz, C Hoyng, J Krätzschmar, S Leal, U Luhmann, A Lüning, Ph Margaron, C Martinho, B Melício, S Mohand-Saïd, D Rowen, G S Rubin, J Sahel, C I Sánchez, D Sanches Fernandes, M Schmid, S Schmitz-Valckenberg, A Skelly, L Stöhr, D Taylor, J Terheyden, A Tufail, L Vieweg, L Wintergerst, C Wojek, N Zakaria, P Zamiri

Affiliations

¹ Department of Ophthalmology, University Hospital Bonn, Ernst-Abbe-Str. 2, 53127, Bonn, Germany.
² Institute for Medical Biometry, Informatics and Epidemiology, Medical Faculty, University of Bonn, Bonn, Germany.
³ UCL Institute of Ophthalmology, University College London, London, UK.
⁴ Moorfields Eye Hospital, London, UK.
⁵ Division of Optometry and Visual Science, School of Health Sciences, City, University of London, London, UK.
⁶ Radboud University Medical Center, Nijmegen, Netherlands.
⁷ Novartis Pharma AG, Basel, Switzerland.
⁸ Carl Zeiss Meditec AG, Dublin, CA, USA.
⁹ Roche Pharmaceutical Research and Early Development, Translational Medicine Ophthalmology, Roche Pharma Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland.
¹⁰ Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal.
¹¹ Bayer AG, Berlin, Germany.
¹² Department of Ophthalmology, University Hospital Bonn, Ernst-Abbe-Str. 2, 53127, Bonn, Germany. robert.finger@ukbonn.de.

PMID: 32682441
PMCID: PMC7368769
DOI: 10.1186/s13063-020-04595-6

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

Jan H Terheyden et al. Trials. 2020.

. 2020 Jul 18;21(1):659.

doi: 10.1186/s13063-020-04595-6.

Authors

Collaborators

MACUSTAR consortium:
P Basile, C Behning, M Berger, A Binns, M Böttger, C Bouchet, J E Brazier, T Butt, C Carapezzi, J Carlton, A Charil, R Coimbra, S Nunes, D Crabb, J Cunha-Vaz, H Dunbar, M Durbin, R Finger, F Holz, C Hoyng, J Krätzschmar, S Leal, U Luhmann, A Lüning, Ph Margaron, C Martinho, B Melício, S Mohand-Saïd, D Rowen, G S Rubin, J Sahel, C I Sánchez, D Sanches Fernandes, M Schmid, S Schmitz-Valckenberg, A Skelly, L Stöhr, D Taylor, J Terheyden, A Tufail, L Vieweg, L Wintergerst, C Wojek, N Zakaria, P Zamiri

Affiliations

¹ Department of Ophthalmology, University Hospital Bonn, Ernst-Abbe-Str. 2, 53127, Bonn, Germany.
² Institute for Medical Biometry, Informatics and Epidemiology, Medical Faculty, University of Bonn, Bonn, Germany.
³ UCL Institute of Ophthalmology, University College London, London, UK.
⁴ Moorfields Eye Hospital, London, UK.
⁵ Division of Optometry and Visual Science, School of Health Sciences, City, University of London, London, UK.
⁶ Radboud University Medical Center, Nijmegen, Netherlands.
⁷ Novartis Pharma AG, Basel, Switzerland.
⁸ Carl Zeiss Meditec AG, Dublin, CA, USA.
⁹ Roche Pharmaceutical Research and Early Development, Translational Medicine Ophthalmology, Roche Pharma Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland.
¹⁰ Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal.
¹¹ Bayer AG, Berlin, Germany.
¹² Department of Ophthalmology, University Hospital Bonn, Ernst-Abbe-Str. 2, 53127, Bonn, Germany. robert.finger@ukbonn.de.

PMID: 32682441
PMCID: PMC7368769
DOI: 10.1186/s13063-020-04595-6

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.

Keywords: Clinical endpoint; Clinical study protocol; Disease progression; Intermediate age-related macular degeneration.

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Conflict of interest statement

J. H. Terheyden: Heidelberg Engineering, Optos, Carl Zeiss MedicTec, CenterVue

F.G. Holz: Acucela, Allergan, Apellis, Bayer, Boehringer-Ingelheim, Bioeq/Formycon, CenterVue, Ellex, Roche/Genentech, Geuder, Grayburg Vision, Heidelberg Engineering, Kanghong, LinBioscience, NightStarX, Novartis, Optos, Pixium Vision, Oxurion, Stealth BioTherapeutics, Zeiss

S. Schmitz-Valckenberg: Alcon/Novartis, Allergan, Bayer, Carl Zeiss MediTec, Bioeq/Formycon, Centervue, Heidelberg Engineering, Optos, Genentech/Roche

A. Lüning: F Heidelberg Engineering, Optos, Carl Zeiss MedicTec, CenterVue

M. Schmid: Pixum Vision

D. P. Crabb, Allergan, Roche, Santen, Centervue

C. Hoyng: Optos, Bayer

P. Margaron, N. Zakaria and Parisa Zamiri: employees of Novartis

M. Durbin: employee of Carl Zeiss Meditec AG

U. Luhmann: employee of F. Hoffmann-La Roche Ltd.

J. Cunha-Vaz: Alimera Sciences, Allergan, Bayer, Gene Signal, Novartis, Pfizer, Precision Ocular Ltd., Roche, Sanofi-Aventis, Vifor Pharma and Carl Zeiss Meditec.

S. Leal: Employee of Bayer Pharma AG, Berlin, Germany

R. P. Finger: C Novartis, Bayer, Santen, Opthea, Novelion, RetinaImplant, Oxford Innovation; F Novartis

References

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1. Colijn JM, Buitendijk GHS, Prokofyeva E, Alves D, Cachulo ML, Khawaja AP, et al. Prevalence of age-related macular degeneration in Europe: the past and the future. Ophthalmology. 2017;124:1753–1763. - PMC - PubMed
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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

Collaborators

Affiliations

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

Collaborators

Affiliations

Abstract

Conflict of interest statement

References

MeSH terms

Associated data

Grants and funding

LinkOut - more resources

Medical