Abstract
This manuscript summarizes the recommendations that emerged from the first Roundtable on Clinical Research in Immunoglobulin Light-chain Amyloidosis (AL), a meeting sponsored by the Amyloidosis Foundation (Clarkston, MI, USA) to develop a consensus of experts on a modern framework for clinical trial design and drug development in AL. Recent diagnostic and technical advances in AL, and updated consensus guidelines for assessing hematologic and organ responses, enable us to define study populations, appropriate end points, and other criteria for all phases of clinical research. This manuscript provides a framework for the design and conduct of systematic collaborative clinical research in AL to encourage more rapid testing of therapies and to expedite new drug development and approval.
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Acknowledgements
We thank the supporters of the Amyloidosis Foundation and the Foundation’s Board of Directors whose continued efforts and involvement made this first roundtable held in Boston in late 2010 a reality. We also thank S Frieda Pearce, PhD, for editing assistance.
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Comenzo, R., Reece, D., Palladini, G. et al. Consensus guidelines for the conduct and reporting of clinical trials in systemic light-chain amyloidosis. Leukemia 26, 2317–2325 (2012). https://doi.org/10.1038/leu.2012.100
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DOI: https://doi.org/10.1038/leu.2012.100
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