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Establishing a global certification standard for evaluating CMT
PI Burns and colleagues have established a suite of clinical outcome measures to evaluate the effectiveness of therapies for children and adults with Charcot-Marie-Tooth disease (CMT) and related neuropathies. These outcome measures have been well-validated and are ready to be used in clinical trials and cohort studies. However, a barrier to progressing to multicenter trials is the lack of well-trained clinical evaluators around the world to collect these measures. We were awarded a 2021 MDA Ideas Grant to develop an e-training and quality assurance system to promote accurate collection of these CMT-specific measurement tools, which are freely available online at www.ClinicalOutcomeMeasures.org. In this MDA Research Infrastructure Grant, we aim to establish a global certification standard to enable accurate measurement and avoid delay to testing rational therapies. A cadre of CMT Master Trainers in partnership with Patient Advocacy Groups in the USA, UK, Europe, Asia, South America and Australia will expand clinical evaluator training resources to sites and countries where trials are likely to occur. We will scale-up trial capacity by building a global certification standard and remote data monitoring system to activate a global network of reliable trial-ready clinical evaluators.
Digital Object Identifier (DOI)
Grantee: Joshua Burns MD
Grant type: Infrastructure Grant
Award total: $300,000.00
Institution: The University of Sydney
Country: Australia