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
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...
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| Format: | Article |
| Language: | English |
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BMC
2020-07-01
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| Series: | Trials |
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| Online Access: | http://link.springer.com/article/10.1186/s13063-020-04595-6 |
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| author | 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 |
| author_facet | 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 |
| author_sort | Jan H. Terheyden |
| collection | DOAJ |
| description | 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 |
| first_indexed | 2024-12-23T05:55:53Z |
| format | Article |
| id | doaj.art-2a193ab793284eaea4408db4a081b409 |
| institution | Directory Open Access Journal |
| issn | 1745-6215 |
| language | English |
| last_indexed | 2024-12-23T05:55:53Z |
| publishDate | 2020-07-01 |
| publisher | BMC |
| record_format | Article |
| series | Trials |
| spelling | doaj.art-2a193ab793284eaea4408db4a081b4092022-12-21T17:57:49ZengBMCTrials1745-62152020-07-0121111110.1186/s13063-020-04595-6Clinical 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—MACUSTARJan H. Terheyden0Frank G. Holz1Steffen Schmitz-Valckenberg2Anna Lüning3Matthias Schmid4Gary S. Rubin5Hannah Dunbar6Adnan Tufail7David P. Crabb8Alison Binns9Clara I. Sánchez10Carel Hoyng11Philippe Margaron12Nadia Zakaria13Mary Durbin14Ulrich Luhmann15Parisa Zamiri16José Cunha-Vaz17Cecília Martinho18Sergio Leal19Robert P. Finger20on behalf of the MACUSTAR consortiumDepartment of Ophthalmology, University Hospital BonnDepartment of Ophthalmology, University Hospital BonnDepartment of Ophthalmology, University Hospital BonnDepartment of Ophthalmology, University Hospital BonnInstitute for Medical Biometry, Informatics and Epidemiology, Medical Faculty, University of BonnUCL Institute of Ophthalmology, University College LondonUCL Institute of Ophthalmology, University College LondonMoorfields Eye HospitalDivision of Optometry and Visual Science, School of Health Sciences, City, University of LondonDivision of Optometry and Visual Science, School of Health Sciences, City, University of LondonRadboud University Medical CenterRadboud University Medical CenterNovartis Pharma AGNovartis Pharma AGCarl Zeiss Meditec AGRoche Pharmaceutical Research and Early Development, Translational Medicine Ophthalmology, Roche Pharma Research and Early Development, Roche Innovation Center BaselNovartis Pharma AGAssociation for Innovation and Biomedical Research on Light and ImageAssociation for Innovation and Biomedical Research on Light and ImageBayer AGDepartment of Ophthalmology, University Hospital BonnAbstract 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 2017http://link.springer.com/article/10.1186/s13063-020-04595-6Intermediate age-related macular degenerationClinical study protocolDisease progressionClinical endpoint |
| spellingShingle | 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 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 Intermediate age-related macular degeneration Clinical study protocol Disease progression Clinical endpoint |
| 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 |
| title_full | 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 |
| title_fullStr | 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 |
| title_full_unstemmed | 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 |
| title_short | 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 |
| title_sort | 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 |
| topic | Intermediate age-related macular degeneration Clinical study protocol Disease progression Clinical endpoint |
| url | http://link.springer.com/article/10.1186/s13063-020-04595-6 |
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