Key Information
Source
Year
2025
summary/abstract
Abstract
Light chain amyloidosis and transthyretin amyloidosis are rare protein misfolding disorders characterized by amyloid deposition in organs, varied clinical manifestations, and poor outcomes. Amyloid fibrils trigger various signaling pathways that initiate cellular, metabolic, structural, and functional changes in the heart and other organs. Imaging modalities have advanced to enable detection of amyloid deposits in involved organs and to assess organ dysfunction, disease stage, prognosis, and treatment response. The Amyloidosis Forum hosted a hybrid meeting to focus on the use of imaging endpoints in clinical trials for systemic immunoglobulin light chain amyloidosis and transthyretin amyloidosis. Stakeholders from academia and industry, together with representatives from multiple regulatory agencies reviewed the use of imaging biomarkers with a focus on cardiac amyloidosis, described applications and limitations of imaging in clinical trials, and discussed qualification of imaging as a surrogate clinical outcome. Survey results provided important patient perspectives. This review summarizes the proceedings of the Amyloidosis Forum.
Key Words
amyloidosis
cardiac imaging
clinical trial endpoints
The Amyloidosis Forum was formed in 2019 as a public-private partnership between the nonprofit Amyloidosis Research Consortium1external link, opens in a new tab and the U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research to advance innovation in drug development for the treatment of systemic amyloidosis.2external link, opens in a new tab Initially, the Amyloidosis Forum focused on clinical trial endpoints and analysis methodologies to address complexities in the development of new therapies for systemic immunoglobulin light chain (AL) amyloidosis.3external link, opens in a new tab, 4external link, opens in a new tab, 5external link, opens in a new tab, 6external link, opens in a new tab A consistent theme discussed at previous forums was the role of imaging in clinical trials, clinical practice, and patient perspectives for both AL and transthyretin (ATTR) amyloidosis.2external link, opens in a new tab,5external link, opens in a new tab The Amyloidosis Forum has expanded its initiatives to assess the role of imaging in development of new therapies for both AL and ATTR amyloidosis.
This review summarizes the proceedings of the Amyloidosis Forum meeting held on the FDA campus, Potential Pathways for Development of Imaging Endpoints for Clinical Trials in AL and ATTR Amyloidosis.7external link, opens in a new tab Stakeholders from academia and industry, along with representatives from U.S. and multiple European health authorities reviewed the current use of imaging biomarkers in AL and ATTR amyloidosis and discussed the role of imaging in diagnosis, assessment of response to therapy, and applications/limitations of use in clinical trials. Survey results provided important patient perspectives on imaging.