Journal article
Are we prepared for clinical trials in Charcot-Marie-Tooth disease?
Brain research, Vol.1729, pp.146625-146625
02/15/2020
DOI: 10.1016/j.brainres.2019.146625
PMID: 31899213
Abstract
•Advances Charcot-Marie-Tooth (CMT) disease are leading to new therapies.•CMT is a challenging disease for designing clinical trials.•There are a range of clinical, functional and patient reported outcome measures in CMT.•Biomarkers of disease progression show high sensitivity in CMT.
There has been considerable progress in developing treatments for Charcot-Marie-Tooth disease with a number of therapies either completing or nearing clinical trials. In the case of CMT1A, the commonest subtype of CMT, there have been more than five randomised, double blind placebo-controlled trials. Although these trials were negative for the primary outcome measure, considerable lessons have been learnt leading to the collection of large prospective natural history data sets with which to inform future trial design as well as the development of new and sensitive outcome measures. In this review we summarise the difficulties of conducting clinical trials in a slowly progressive disease such as CMT1A and the requirement for sensitive, reproducible and clinically relevant outcome measures. We summarise the current array of CMT specific outcome measures subdivided into clinical outcome measures, functional outcome measures, patient reported outcome measures, biomarkers of disease burden and treatment specific biomarkers of target engagement. Although there is now an array of CMT specific outcome measures, which collectively incorporate clinically relevant, sensitive and reproducible outputs, a single outcome measure incorporating all three qualities remains elusive.
Details
- Title: Subtitle
- Are we prepared for clinical trials in Charcot-Marie-Tooth disease?
- Creators
- A.M Rossor - Department of Neuromuscular Diseases, University College London, Queen Square Institute of Neurology, London, United KingdomM.E Shy - Department of Neurology, University of Iowa, 200 Hawkins Drive, Iowa City, IA 52242, USAM.M Reilly - Department of Neuromuscular Diseases, University College London, Queen Square Institute of Neurology, London, United Kingdom
- Resource Type
- Journal article
- Publication Details
- Brain research, Vol.1729, pp.146625-146625
- DOI
- 10.1016/j.brainres.2019.146625
- PMID
- 31899213
- NLM abbreviation
- Brain Res
- ISSN
- 0006-8993
- eISSN
- 1872-6240
- Publisher
- Elsevier B.V
- Grant note
- DOI: 10.13039/501100000265, name: Medical Research Council; name: MRC Centre, award: G0601943; name: National Institutes of Neurological Diseases; name: Stroke and office of Rare Diseases, award: U54NS065712; DOI: 10.13039/501100012317, name: National Institute for Health Research University College London Hospitals Biomedical Research Centre
- Language
- English
- Date published
- 02/15/2020
- Academic Unit
- Neurology; Molecular Physiology and Biophysics; Stead Family Department of Pediatrics; Iowa Neuroscience Institute
- Record Identifier
- 9984070555702771
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