Introduction: Charcot-Marie-Tooth disease (CMT) is one of the most commonly inherited neuromuscular diseases – there is no effective treatment. Foot and ankle weakness is a major problem for children with CMT, thus interventions that focus on maintaining and increasing strength may provide a solution. Research Question: Is progressive resistance strength training an effective and safe intervention to improve strength, disability, gait and quality of life of children with CMT? Participants and Setting: Sixty children (6 to 17 years) with confirmed CMT who reside in Sydney, Australia will be recruited via referral from a paediatric neurologist, advertisements or the Australasian Paediatric CMT Registry. Intervention: Participants will be randomised to undergo a 24-week, thrice weekly, high-intensity progressive resistance foot and ankle exercise programme (HIGH) or low-intensity foot and ankle exercise control programme (LOW). Measurements: Outcome measures will be conducted at baseline, 6, 12 and 24 months. The primary outcome is isometric dorsiflexion strength measured by hand-held dynamometry. Secondary outcomes include disability, gait, quality of life, functional ankle instability and muscle volume and fatty infiltration of the anterior compartment of the lower leg (determined by MRI). Procedure: Randomisation and allocation will be by a computer-generated algorithm, maintained and assigned by an external phone-based system, concealed to the investigators. Participants, parents and the outcome assessors will be blinded to group assignment. Analysis: Treatment effect between groups is by intention-to-treat with a linear regression approach to analysis of covariance using 95% CI and p < 0.05. Discussion: This study is the first randomised controlled trial to evaluate the risks and benefits of strengthening the affected muscles in children with CMT
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