Accelerate Clinical Trials in Charcot-Marie-Tooth Disease (ACT-CMT): A Protocol to Address Clinical Trial Readiness in CMT1A.

With therapeutic trials on the horizon for Charcot-Marie-Tooth type 1A (CMT1A), reliable, valid, and responsive clinical outcome assessments and biomarkers are essential. Accelerate Clinical Trials in CMT (ACT-CMT) is an international study designed to address important gaps in CMT1A clinical trial readiness including the lack of a validated, responsive functional outcome measure for adults, and a lack of validated biomarkers for multicenter application in clinical trials in CMT1A. The primary aims of ACT-CMT include validation of the Charcot-Marie-Tooth Functional Outcome Measure, magnetic resonance imaging of intramuscular fat accumulation as a lower limb motor biomarker, and

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Copyright © 2022 Eichinger, Sowden, Burns, McDermott, Krischer, Thornton, Pareyson, Scherer, Shy, Reilly and Herrmann.

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KE reports grant support through the Charcot-Marie-Tooth Association and has served on advisory boards for Biogen, Roche, Dyne, and has received consulting fees from Fulcrum Therapeutics, Dyne, Acceleron Pharma, Avidity and Roche. JB reports work in the Burns Group is supported by the Australian Government Department of Health (NHMRC-MRFF #1152226), US National Institutes of Health (NINDS #1U01NS109403-01, NINDS #U54NS065712), US Muscular Dystrophy Association (Idea Grant #876246), Charcot-Marie Tooth Association of Australia, Charcot Marie Tooth Association (USA), Cerebral Palsy Alliance, Diabetes Australia, Humpty Dumpty Foundation, Kids Neuroscience Centre, The Children's Hospital at Westmead, and The University of Sydney. Consultancies over last 18 months: Acceleron Pharma, Pharnext, Passage Bio, Inc. He is a registered podiatrist working at the Children's Hospital at Westmead, Sydney, Australia. JT reports consultancies with Invicro, LLC and F. Hoffmann-La Roche Ltd. DP reports grant support from Telethon-UILDM, AFM-Telethon, National Institutes of Health, and the Charcot-Marie-Tooth Association, serves on clinical advisory boards for Inflectis, Alnylam, and Akcea, Arvinas, and Augustine, received travel grants from Kedrion Spa and Pfizer, and Istituto Neurologico Carlo Besta receives donations for research from Pfizer, LAM Therapeutics, Acceleron Pharma Inc. SS actively serves as a consultant to Disarm Therapeutics, Mitochondria in Motion, and Pfizer and has research support from the National Institutes of Health, Muscular Dystrophy Association, and the Charcot-Marie-Tooth Association. He reports grant support from U54 NS0657, the Muscular Dystrophy Association, and the Charcot-Marie-Tooth Association. He also serves as a consultant for Inflectis BioSci, Alnylam Pharma, Mitochondria in Motion, Passage Pharma, Applied Therapeutics, and DTx Pharm. MR reports grant support from U54 NS0657, Muscular Dystrophy Association, Charcot-Marie-Tooth Association, the Medical Research Council (MRC) and the Wellcome trust and consults for Inflectis, Alnylam, Akcea and served on a steering committee for Eidos. He reports grant support through U54 NS065712, NINDS. 5U01NS109403-04, 1R01DK115687-04, the CMT Association, Muscular Dystrophy Association Friedreich's Ataxia Research Alliance, Acceleron Pharma. He also reports consulting fees from Regenacy Pharmaceuticals, Acceleron Pharma, Alnylam, Neurogene, Applied Therapeutics, Sarepta, Passage Bio, Pfizer Guidepoint Global, GLG, Slingshot Insights, ClearView Health Partners, MedPace, DDB Health NY, Cydan, Trinity Partners, Schlesinger. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.