Frank Holz MD, PhD
In order to prevent or delay the progression of intermediate age-related macular degeneration (iAMD) to late-stage AMD, the functional deficit in iAMD must first be characterised. The goal of the MACUSTAR Consortium is to seek novel clinical endpoints for clinical trials in iAMD so that interventional studies can be implemented, Frank Holz MD, from the co-ordinating centre in Bonn, Germany, said during a session of the 18th EURETINA Congress in Vienna.
MACUSTAR is funded by the European Innovative Medicines Initiative (IMI), the world’s largest public-private partnership in life sciences. IMI works as a partnership of the European Commission and EFPIA (European Federation of Pharmaceutical Industries and Associations). The MACUSTAR consortium includes researchers from the Netherlands, France, Portugal and the UK. It also includes industrial companies Bayer, Zeiss, Novartis and Roche. The total funding is €16 million for this study. EVICR.net is also involved in the study.
“AMD is a progressively degenerative disease than can lead to the advanced stages of neovascular AMD and geographic atrophy with subsequent visual loss. Despite a prevalence of approximately 15% in people over the age of 55 years, there are currently no available treatments to counteract the progression of iAMD,” noted Dr Holz, University of Bonn, Germany.
Currently, no outcome measures are clinically validated and accepted as clinical endpoints for regulatory agencies for drug development in iAMD. As clinical endpoints currently accepted by regulators cannot detect functional loss or patient-relevant impact in iAMD, the goal of MACUSTAR is to develop clinical endpoints in three main categories: function, structure (imaging) and patient-reported outcomes.
Robert Finger MD
Robert Finger MD, also of Bonn, brought delegates up to speed regarding the ongoing MACUSTAR project.
“Functional testing in MACUSTAR is used to identify and validate visual function outcomes that reliably capture the functional impairment in iAMD and to establish the discriminatory power of these variables in relation to differentiating between disease stages and thus allowing for the assessment of progression of AMD,” said Dr Finger.
Structural endpoints will be determined by standard retinal imaging, such as OCT, scanning laser ophthalmoscopy, fundus autofluorescence, fundus photography and fluorescein angiography, and innovative modalities such as quantitative autofluorescence, OCT-angiography and swept-source OCT.
Candidate endpoints include scotopic and mesopic microperimetry, dark adaptation, contrast sensitivity and the implementation of a specifically developed patient-reported outcome measure, the Vision Impairment in Low Luminance (VILL) questionnaire. This covers areas such as reading, mobility, safety and socio-emotional well-being, all with a focus on light conditions such as low contrast and low luminance that become challenging for people with iAMD.
Recruitment in 20 clinical sites across Europe will continue until February 2019, with the goal of including 750 patients: 600 patients with intermediate AMD; 50 with no abnormalities (or normal ageing changes); 50 with early AMD and 50 with late AMD. These patients will be followed up for three years.
Frank Holz: holz@ukb.uni-bonn.de
Robert Finger: Robert.Finger@ukb.uni-bonn.de
