Published August 28, 2018 | Version v1
Journal article Open

MACUSTAR: Development and Clinical Validation of Functional, Structural, and Patient-Reported Endpoints in Intermediate Age-Related Macular Degeneration

Creators

  • 1. Department of Ophthalmology, University of Bonn, Bonn, Germany
  • 2. Institute for Medical Biometry, Epidemiology and Informatics, University Hospital 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. Diagnostic Image Analysis Group, Radboud University Medical Center, Nijmegen, The 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. Development Pharmaceuticals, Therapeutic Areas PAO, Bayer AG, Berlin, Germany

Description

Abstract
Purpose: Currently, no outcome measures are clinically validated and accepted as clinical endpoints by regulatory agencies for drug development in intermediate age-related macular degeneration (iAMD). The MACUSTAR Consortium, a public-private research group funded by the European Innovative Medicines Initiative intends to close this gap. Procedures: Development of study protocol and statistical analysis plan including predictive modelling of multimodal endpoints based on a review of the literature and expert consensus. Results: This observational study consists of a cross-sectional and a longitudinal part. Functional outcome measures assessed under low contrast and low luminance have the potential to detect progression of visual deficit within iAMD and to late AMD. Structural outcome measures will be multimodal and investigate topographical relationships with function. Current patient-reported outcome measures (PROMs) are not acceptable to regulators and may not capture the functional deficit specific to iAMD with needed precision, justifying development of novel PROMs for iAMD. The total sample size will be n = 750, consisting mainly of subjects with iAMD (n = 600). Conclusions: As clinical endpoints currently accepted by regulators cannot detect functional loss or patient-relevant impact in iAMD, we will clinically validate novel candidate endpoints for iAMD.

Notes

Name of action: Intermediate AMD: Development of novel clinical endpoints for clinical trials in patients with a regulatory and patient access intention - MACUSTAR This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 116076. This Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation programme and EFPIA.

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Is identical to
10.1159/000491402 (DOI)