title: Randomised controlled trial protocol of foot and ankle exercise for children with Charcot-Marie-Tooth disease.
abstract: BACKGROUND
  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.
  
  
  OBJECTIVE
  Is progressive resistance strength training an effective and safe intervention to improve strength, disability, gait and quality of life of children with CMT?
  
  
  METHODS
  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.
  
  
  METHODS
  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).
  
  
  METHODS
  Out-come 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).
  
  
  METHODS
  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.
  
  
  METHODS
  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.
  
  
  CONCLUSIONS
  This study is the first randomised controlled trial to evaluate the risks and benefits of strengthening the affected muscles in children with CMT.
  
  
  BACKGROUND
  Australian New Zealand Clinical Trials Registry.
  
  
  BACKGROUND
  ACTRN12613000552785.
authors.affiliation: Institute for Neuroscience and Muscle Research, The Children's Hospital at Westmead - The University of Sydney; Faculty of Health Sciences, The University of Sydney.
authors.forename: Amy D
authors.initials: AD
authors.lastname: Sman
authors.affiliation: Faculty of Health Sciences, The University of Sydney.
authors.forename: Jacqueline
authors.initials: J
authors.lastname: Raymond
authors.affiliation: Faculty of Health Sciences, The University of Sydney.
authors.forename: Kathryn M
authors.initials: KM
authors.lastname: Refshauge
authors.affiliation: Institute for Neuroscience and Muscle Research, The Children's Hospital at Westmead - The University of Sydney.
authors.forename: Manoj P
authors.initials: MP
authors.lastname: Menezes
authors.affiliation: Medical Imaging, The Children's Hospital at Westmead, Sydney, Australia.
authors.forename: Terri
authors.initials: T
authors.lastname: Walker
authors.affiliation: Institute for Neuroscience and Muscle Research, The Children's Hospital at Westmead - The University of Sydney.
authors.forename: Robert A
authors.initials: RA
authors.lastname: Ouvrier
authors.affiliation: Institute for Neuroscience and Muscle Research, The Children's Hospital at Westmead - The University of Sydney; Faculty of Health Sciences, The University of Sydney.
authors.forename: Joshua
authors.initials: J
authors.lastname: Burns
mesh_terms: D000293:Adolescent; D000843:Ankle Joint / Q000503:physiopathology*; D002607:Charcot-Marie-Tooth Disease / Q000503:physiopathology / Q000628:therapy*; D002648:Child; D002985:Clinical Protocols; D005081:Exercise Therapy / Q000379:methods*; D033023:Foot Joints / Q000503:physiopathology*; D005684:Gait / Q000502:physiology; D006801:Humans; D053580:Muscle Strength / Q000502:physiology; D017063:Outcome Assessment, Health Care; D011788:Quality of Life; D055070:Resistance Training / Q000379:methods*; D016896:Treatment Outcome