Development of Imaging Endpoints for Clinical Trials in AL & ATTR Amyloidosis: Amyloidosis Forum Proceedings (JIMG May 2025)-Article: Development of Imaging Endpoints for Clinical Trials in AL & ATTR Amyloidosis: Amyloidosis Forum Proceedings

Article: Development of Imaging Endpoints for Clinical Trials in AL & ATTR Amyloidosis: Amyloidosis Forum Proceedings

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The document summarizes the proceedings of the Amyloidosis Forum regarding the development of imaging endpoints for clinical trials in AL and ATTR amyloidosis. These rare protein misfolding disorders result in amyloid deposits in vital organs, primarily affecting cardiac function. The Forum, a joint initiative by the Amyloidosis Research Consortium and the FDA, focused on enhancing imaging biomarkers to assess organ dysfunction, disease stage, and treatment response in amyloidosis, with a particular focus on cardiac manifestations.<br /><br />The review discusses the application of various imaging techniques such as echocardiography, cardiac magnetic resonance imaging (CMR), single-photon emission computed tomography (SPECT), and positron emission tomography (PET) in diagnosing and evaluating treatment responses in AL and ATTR amyloidosis. Advanced imaging methods are highlighted for their potential to detect amyloid deposits early, correlate with clinical outcomes, and guide therapy. Despite these advancements, imaging standardization across multi-center trials remains a challenge, requiring consistency in protocols and the potential use of artificial intelligence for data interpretation.<br /><br />Key insights include a patient survey revealing a preference for noninvasive imaging over biopsy for diagnosing and monitoring amyloidosis. This is due to the ability of imaging to visualize the impact of treatments and understand disease status better, as highlighted by patient perspectives in the survey.<br /><br />Additionally, the Forum emphasized the need for developing validated imaging-based biomarkers as surrogate endpoints in clinical trials, addressing regulatory considerations and the importance of standardization. There is a push towards collaborative efforts and data sharing to strengthen drug development for systemic amyloidosis. Overall, the Forum aligns with the goal to advance early diagnosis and effectively measure treatment efficacy in this complex disease through improved imaging strategies.

Keywords

Amyloidosis

Imaging Endpoints

Clinical Trials

Cardiac Function

Biomarkers

Echocardiography

Magnetic Resonance Imaging

Artificial Intelligence

Noninvasive Imaging

Regulatory Considerations