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Develop and validate appropriate and acceptable outcome measures in intermediate age-related macular degeneration for future interventional clinical trials - Sofia ref.: 116076

Objective

Despite significant advances in the treatment and understanding of late stage age-related macular degeneration (AMD), it continues to be the main cause of irreversible severe visual loss in Europe and its prevalence and incidence will increase with current demographic trends. In order to reduce the significant burden of late stage AMD, novel interventions should aim at stopping or delaying progression from the preceding disease stage intermediate AMD (iAMD) to late stage AMD. As a prerequisite, validated clinical endpoints for iAMD are needed. These should be acceptable to regulatory agencies, health technology assessment (HTA) bodies, and payers. Currently such endpoints do not exist for iAMD clinical trials (CTs).

In addition, there is good evidence indicating that patients with iAMD experience some impairment of visual function yet it is unknown to what extend this impacts the patients’ life nor can it be reliably measured and quantified. It is also unknown whether there are specific risk factors in the population of iAMD patients which identify those with more rapid progression to late stages of the disease.

Therefore, to enable successful development of iAMD interventions validated functional, morphological and patient--reported
endpoints for CTs, which are clinically meaningful and accepted by regulatory agencies, are required. In addition, functional decline in iAMD, as well as, specific risk factors for iAMD progression to late stage AMD need to be better characterized to inform and improve conduct of future iAMD CTs.

Against this background, the major objective of MACUSTAR is to develop novel clinical endpoints for CTs with a regulatory and patient access intention in patients with iAMD. Additional objectives are to characterize visual impairment in iAMD and its progression, as well as, identify risk factors for progression.

For clinical endpoint development, functional, structural and patient-reported outcome measures will be assessed with regards to

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RIA - Research and Innovation action

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Call for proposal

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(opens in new window) H2020-JTI-IMI2-2015-07-two-stage

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Coordinator

UNIVERSITATSKLINIKUM BONN
Net EU contribution

Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.

€ 3 802 893,40
Address
VENUSBERG-CAMPUS 1
53127 Bonn
Germany

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Region
Nordrhein-Westfalen Köln Bonn, Kreisfreie Stadt
Activity type
Higher or Secondary Education Establishments
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Total cost

The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.

€ 4 548 971,88

Participants (18)

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