Clinical Trial: BALTiC Study: A Feasibility Analysis of Home Based BALance Training in People With Charcot-Marie-Tooth Disease

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: BALTiC Study: A Feasibility Analysis of Home Based BALance Training in People With Charcot-Marie-Tooth Disease

Brief Summary: Charcot Marie Tooth (CMT) is the most common inherited neuropathy. It affects the nerves of the hands and feet first, slowly progressing towards the centre of the body. It causes varying degrees of weakness and reduced feeling, usually affecting the feet and ankles first. High incidence of falls and knock on effects in quality of life are common. This is a lifelong condition which, though not life limiting, does not have a cure. Research into the effect of balance rehabilitation in people with CMT has been limited to traditional exercises, which do not address the complex nature of balance impairments in this condition. Multi-sensory rehabilitation has proved beneficial in improving balance in people with sensory neuropathy. Research shows that strengthening trunk muscles can improve balance in older people. Trunk and muscles close to it are largely unaffected in people with CMT, therefore these muscles could be strengthened in these patients. This study proposes to assess the feasibility of multi-sensory balance training, strength training with a focus on the trunk and muscles close to the trunk, and falls management education. This comprehensive approach is reflective of a clinical physiotherapy programme. Though a life-long condition, many people with CMT lead full lives. A home based programme is proposed to so that patients can fit it into their lives without having to travel for treatment. Therapists will use self-management principles within treatment. Measurements will be taken prior to and following treatment including physical measures, questionnaires and interviews. The physiotherapy interventions will be taught home and data collection will be at the National Hospital for Neurology and Neurosurgery, University College London Hospitals (UCLH).

Detailed Summary:

A recent, unpublished focus group study of people with CMT found that they reported more falls than healthy people. They reported problems walking on uneven surfaces, difficulty getting off the floor and a necessity to consciously think about every step to avoid falling while walking (GM Ramdharry et al., 2015). A retrospective survey of people with CMT found that 89% of the 94 respondents reported falls to the ground. Half of them reported falling at least once a month with 5% falling every day (Ramdharry et al., 2011). We have just completed an exploratory study of factors predicting falls risk. Preliminary analysis of prospective falls data for 32 people over 6 months showed that they fell a median of 4 times (range 0 - 54 falls) and functional balance performance may be predictive (Ramdharry et al., 2015). A study of people with CMT1a found that sensory impairment increased visual dependence for a static balance task (van der Linden et al., 2010). There has only been one small study of balance training in people with CMT, comparing a novel rehabilitation device with more traditional exercises (Matjacić and Zupan, 2006). Both interventions showed improvement in functional balance scores. Studies of proximal strength training in CMT have shown improvements in walking parameters (Chetlin et al., 2004; Lindeman et al., 1995), but the effect on balance has not been explored. Because of the mixed sensori-motor presentation of people with CMT, a combined approach of multi-sensory balance training and proximal strengthening will be delivered. A home-based model has been proposed as CMT is a life-long condition that needs to be managed by the individual outside of the medical or therapy environment.

STUDY DESIGN & JUSTIFICATION A single blinded randomised controlled design has been chosen in this feasibility study. The intention is to see if there is an effect of
Sponsor: University College London Hospitals

Current Primary Outcome: Balance performance [ Time Frame: 12 weeks ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Recruitment rate [ Time Frame: 1 year ]
  For this feasibility study, recruitment rate data will be gathered
• Falls monitoring [ Time Frame: 20 weeks ]
  Falls monitoring will be done one month before, during the 12 week invention/control period and one month afterwards using postcards or a web based app, depending on participant preference.
• Bruininks Oseretsky Test (BOT) [ Time Frame: 12 weeks ]
  Clinician rated measure of functional balance performance.
• Berg Balance test [ Time Frame: 12 weeks ]
  Clinician rated measure of functional balance performance.
• BESTest [ Time Frame: 12 weeks ]
  Clinician rated measure of functional balance performance.
• 10 minute timed walk [ Time Frame: 12 weeks ]
  Walking speed test
• Quantitative lower limb muscle strength testing [ Time Frame: 12 weeks ]
  Hand-held myometry protocol performed by rater for the major lower limb muscle groups.
• CMTES: Charcot Marie Tooth Examination Score [ Time Frame: 12 weeks ]
  Clinician rated disease severity scale
• Falls Self Efficacy Scale [ Time Frame: 12 weeks ]
  Patient reported outcome measure of balance confidence. Questionnaire.
• Walk-12 [ Time Frame: 12 weeks ]
  Patient reported outcome measure of walking function. Questionnaire.
• IPAQ: International Physical Activity Questionnaire [ Time Frame: 12 weeks ]
  Patient reported outcome measure of physical activity. Questionnaire.
• SF36: Short Form 36 [ Time Frame: 12 weeks ]
  Patient reported outcome measure of health related quality of life. Questionnaire.
• Hospital Anxiety and Depression Score [ Time Frame: 12 weeks ]
  Patient reported outcome measure of depression and anxiety symptoms. Questionnaire.

Original Secondary Outcome: Same as current

Information By: University College London Hospitals

Dates:
Date Received: November 28, 2016
Date Started: December 2016
Date Completion: November 2017
Last Updated: November 30, 2016
Last Verified: November 2016