The use of physical therapy in CMT used to be a controversial matter in the recent past due to the report of fatigue and overwork weakness.12 However, a recent study examining the bilateral intrinsic hand and leg muscle strength in 271 CMT1A patients showed no difference between the dominant and the non-dominant side. The characteristic pes cavus formation in patients with Charcot-Marie-Tooth disease is due to a plantar flexion deformity of the first metatarsal bone. In an observational clinical trial with 20 CMT patients, the participants received resistance training either with or without additional creatine monohydrate supplementation. In a more recent pilot study, patients suffering from several types of CMT underwent a rigorous exercise regimen including treadmill, stretching, respiratory and proprioceptive exercises (TreSPE). Clinical approaches towards the improvement of the conceptual idea of ‘quality of life’ and mental health in CMT patients are scarce.
Despite its monogenetic cause, patients with CMT1A display a marked interindividual variability of disease severity. As no curative treatment is yet established for any type of Charcot-Marie-Tooth disease, rehabilitation and physical therapy remain the only positively disease-modifying measures to date. Piscosquito G, Reilly M, Schenone A, Fabrizi GM, Cavallaro T, Vita G, Quattrone A, Padua L, Gemignani F, Visioli F.
Fledrich R, Schlotter-Weigel B, Schnizer TJ, Wichert SP, Stassart RM, Meyer zu Horste G et al. Lewis RA, McDermott MP, Herrmann DN, Hoke A, Clawson LL, Siskind C, Feely SM, Miller LJ, Barohn RJ, Smith P, Luebbe E, Wu X, Shy ME.
Sinclair CD, Morrow JM, Miranda MA, Davagnanam I, Cowley PC, Mehta H, Hanna MG, Koltzenburg M, Yousry TA, Reilly MM, Thornton JS. Pareyson D, Schenone A, Fabrizi GM, Santoro L, Padua L, Quattrone A, Vita G, Gemignani F, Visioli F, Solari A. The Clinical Trials Regulations require the sponsor to notify the end of a trial to competent authorities (MHRA in the UK) and ethics committees of all member states concerned, within 90 days.
Ces informations sont fournies par Francois Houyez, Treatment Information and Access Director, Health Policy Advisor, EURORDIS, and the Drug Information, Transparency and Access task force. Pour rapprocher patients, familles et experts sur un forum modere multilingue, ou partager leurs experiences. Participez au plus grand evenement reunissant tous les acteurs des maladies rares en Europe lors de Conference europeenne bisannuelle sur les maladies rares et les medicaments orphelins (European Conference on Rare Diseases and Orphan Products, ou ECRD). I tracked clinical trials which fall in definition of cell therapy: administration of living cells in human with therapeutic purpose. The moderate-intensity aerobic exercises were performed twice per week for a duration of two months. A recently published meta-analysis of 20 clinical studies on the impairment in ‘quality of life’, emphasised the need for evidence-based approaches.31 Depression, anxiety and sleeping disorders for instance, are significantly more common in CMT patients than in the general population. The underlying reason for this variability is largely unknown and epigenetic factors have been discussed.26 At present, the assessment of the individual disease severity in patients with CMT1A is performed solely by clinical and electrophysiological examinations. However, much needed evidence-based data on rehabilitation is scarce and former concerns against rehabilitation measures on the grounds of fatigue and overwork weakness can be dismissed in favour of symptom alleviating, moderate aerobic exercises.
Estimation of the mutation frequencies in Charcot-Marie-Tooth disease type 1 and hereditary neuropathy with liability to pressure palsies: a European collaborative study. Clinical variability in two pairs of identical twins with the Charcot-Marie-Tooth disease type 1A duplication.

Factors that influence health-related quality of life in Australian adults with Charcot-Marie-Tooth disease. Charcot-Marie-Tooth Disease: An Overview of Genotypes, Phenotypes, and Clinical Management Strategies. Ascorbic acid in Charcot-Marie-Tooth disease type 1A (CMT-TRIAAL and CMT-TRAUK): a double-blind randomised trial. Outcome measures and rehabilitation treatment in patients affected by Charcot-Marie-Tooth neuropathy: a pilot study. Fatigue, reduced sleep quality and restless legs syndrome in Charcot-Marie-Tooth disease: a web-based survey. Strength training in patients with myotonic dystrophy and hereditary motor and sensory neuropathy: a randomized clinical trial. Charcot-Marie-Tooth disease type 1A duplication: spectrum of clinical and magnetic resonance imaging features in leg and foot muscles. New insights into the pathophysiology of pes cavus in Charcot-Marie-Tooth disease type 1A duplication. Night splinting does not increase ankle range of motion in people with Charcot-Marie-Tooth disease: a randomised, cross-over trial. Self-reported follow-up post-intervention adherence to resistance exercise training in Charcot-Marie-Tooth disease patients. 168th ENMC International Workshop: outcome measures and clinical trials in Charcot-Marie-Tooth disease (CMT). A rat model of Charcot-Marie-Tooth disease 1A recapitulates disease variability and supplies biomarkers of axonal loss in patients. Charcot-Marie-Tooth disease, psychiatric indicators and quality of life: a systematic review. Skeletal muscle MRI magnetisation transfer ratio reflects clinical severity in peripheral neuropathies.
Evolution of Charcot-Marie-Tooth disease type 1A duplication: a 2-year clinico-electrophysiological and lower-limb muscle MRI longitudinal study.
Selected items from the Charcot-Marie-Tooth (CMT) Neuropathy Score and secondary clinical outcome measures serve as sensitive clinical markers of disease severity in CMT1A patients.
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Every year I give a snapshot of some tracked data, captured from international clinical trials databases. Do the numbers change materially if you run them just on the industry sponsored 30% of trials?
The exercises were performed with adjustable wrist and ankle weights, according to the individual baseline strength. A holistic approach towards rehabilitation in CMT could therefore include voluntary psychological guidance, coping strategies for sensory loss and neuropathic pain, vocational rehabilitation, as well as genetic counselling.
High-dosage ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A: results of a randomized, double-masked, controlled trial.
They also facilitate stretching and minimise the later development of a neuropathic Charcot joint.

Recently, we were able to show in a large Europe-wide, clinical prospective study that certain secondary clinical outcome measures, e.g. Due to the slowly progressive nature of the disease, recent studies stress the importance of continuing exercises at home, in order to maintain individual physical abilities. Section 2.5 of the EU Detailed guidance on the request to the competent authorities for authorisation of a clinical trial on a medicinal product for human use the notification of substantial amendments and the declaration of the end of the trial (CT-1) (PDF, 878 KB), recommends that in most cases this should be the date of the last visit of the last patient undergoing the trial. The intensity of training was systematically increased in terms of weight and number of repetitions.
In comparison to a healthy control group, no significant pulmonary differences were observed. Fatigue and overwork-weakness, that would prohibit aerobic exercises in CMT patients, did not occur.
These Biomarkers may not only serve as sensitive surrogate markers of clinical disease severity, but also identify responders to a putative therapy. It states:”…the end of the research should be defined in relation to the collection of all data required to answer the research questions in the protocol” Once the end of trial has been declared, no further substantial amendments are possible and a Clinical Trial Summary Report must be produced within set timeframes (see Clinical Trial Summary Report station).
The training sessions significantly improved the activity of daily life (ADL) and strength.
Nearly all tested parameters showed improvement after TreSPE, though mainly not statistically significant.
However, no differences in performance were observed when comparing patients with or without creatine monohydrate supplementation.16 A follow-up study 20-34 months after completion of resistance training showed that patients who continued as well as patients who discontinued their training, lost strength in comparison to their baseline assessment. The authors partially justify this circumstance with the small number of participants (n=8). In a proof of principle study we have demonstrated that the expression levels of selected genes in sciatic nerve and skin tissue can be utilised to measure and predict the disease severity in CMT rats.
The functional abilities on the other hand, were only lost in those who discontinued their training.
Furthermore, they do not recommend usage of the Charcot-Marie-Tooth Neuropathy Score as well as the MRC scale for post-rehabilitation controls, since subtle improvements could not be detected with these measures.
Importantly, we validated these disease severity markers in skin biopsies of 46 patients with CMT1A.30 At the moment, these markers are examined with regard to disease progression within a large pan-European consortium. For multi-national trials, the end of trial form should only be submitted when the trial has ended in all countries. However, after 6 months of washout, most clinical measures began to deteriorate again without undercutting the baseline values. In the near future we hope to provide the clinical practice with applicable biomarkers which in turn may accelerate the development of a therapy for CMT1A.
Thus, a repetition of TreSPE-training within six months is generally recommended to merely maintain clinical abilities.

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