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

NCT02982343 | Completed

• 1 other identifier: 16/LO/0720
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

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).

Conditions

Charcot-Marie-Tooth Disease, Type IA

Outcome Measures

Primary Outcomes (1)

• Balance performance

  Balance performance will be measured using laboratory measures of postural stability during static and dynamic tasks. Centre of pressure excursion and velocity will be the main variables

  12 weeks

Secondary Outcomes (13)

• Recruitment rate

  1 year

• Falls monitoring

  20 weeks

• Bruininks Oseretsky Test (BOT)

  12 weeks

• Berg Balance test

  12 weeks

• BESTest

  12 weeks

Study Arms (2)

Falls management plus exercise training

EXPERIMENTAL

Falls management and education session. Home proximal lower limb strength training and multi-sensory balance training.

Behavioral: Falls management plus exercise training

Falls management only

ACTIVE COMPARATOR

Falls management and education session only.

Behavioral: Falls management only

Interventions

Falls management plus exercise training BEHAVIORAL

Participants will undergo a session of falls management and education followed by 12 weeks of strength and balance training. Strength training will be performed 4 times per week. Resistance training of the proximal muscles will be prescribed progressing to loads of 60% of 1 repetition maximum, 8-12 repetitions. Weaker muscles will be trained using 2 sets of 8-12 repetitions against gravity. They will also give a selection of exercises targeting balance to challenge stability and sensory feedback using equipment such as foam cushions, though will be individual based on participants' limitations and abilities. Participants will be asked to perform balance exercises daily.

Falls management plus exercise training

Falls management only BEHAVIORAL

Participants will undergo a session of falls management and education.

Falls management only

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Genetically confirmed diagnosis of CMT1a
• History of falls
• Mixed sensory and motor presentation
• Over 18 years
• Able to stand unsupported for five minutes
• Able to walk for 50m with or without a walking stick or orthotic devices

You may not qualify if:

• Presence of other significant neurological disorders (such as multiple sclerosis, cerebrovascular diseases, epilepsy, movement disorders), or major comorbidities (e.g. vestibular dysfunction, use of medication which may affect balance, cognitive impairment, presence of medical conditions in which exercise training may be detrimental).
• Presence of peripheral neuropathy caused by a condition other then CMT.
• Limb surgery during the six months prior to screening (or planned before final assessment).
• Participation in any other concurrent intervention trial, or less than six months after cessation of an intervention trial.
• Women of child-bearing age if they are pregnant, planning a pregnancy during their time in the study or in the 12 weeks following giving birth. This is due to the effects of pregnancy on balance.

Sponsors & Collaborators

• University College London Hospitals: lead
• Charcot Marie Tooth United Kingdom: collaborator
• Kingston University: collaborator

Study Sites (1)

National Hospital for Neurology and Neurosurgery, UCLH

London, WC1N 3BG, United Kingdom

Location

Related Publications (6)

• Ramdharry, G., M. Dudziec, D. Tropman, E. Dewar, A. Wallace, M. Laura, R. Grant, and M. Reilly. 2015. 'Exploring the Causes of Falls and Balance Impairments in People with Charcot-Marie Tooth Disease'. Physiotherapy 101 (May): e1255. doi:10.1016/j.physio.2015.03.1159.

  BACKGROUND

• Ramdharry GM, Pollard A, Anderson C, Laura M, Murphy SM, Dudziec M, Dewar EL, Hutton E, Grant R, Reilly MM. A pilot study of proximal strength training in Charcot-Marie-Tooth disease. J Peripher Nerv Syst. 2014 Dec;19(4):328-32. doi: 10.1111/jns.12100.

  PMID: 25582960 BACKGROUND

• van der Linden MH, van der Linden SC, Hendricks HT, van Engelen BG, Geurts AC. Postural instability in Charcot-Marie-Tooth type 1A patients is strongly associated with reduced somatosensation. Gait Posture. 2010 Apr;31(4):483-8. doi: 10.1016/j.gaitpost.2010.02.005. Epub 2010 Mar 11.

  PMID: 20226674 BACKGROUND

• Matjacic Z, Zupan A. Effects of dynamic balance training during standing and stepping in patients with hereditary sensory motor neuropathy. Disabil Rehabil. 2006 Dec 15;28(23):1455-9. doi: 10.1080/09638280600646169.

  PMID: 17166808 BACKGROUND

• Chetlin RD, Gutmann L, Tarnopolsky M, Ullrich IH, Yeater RA. Resistance training effectiveness in patients with Charcot-Marie-Tooth disease: recommendations for exercise prescription. Arch Phys Med Rehabil. 2004 Aug;85(8):1217-23. doi: 10.1016/j.apmr.2003.12.025.

  PMID: 15295743 BACKGROUND

• Lindeman E, Leffers P, Spaans F, Drukker J, Reulen J, Kerckhoffs M, Koke A. Strength training in patients with myotonic dystrophy and hereditary motor and sensory neuropathy: a randomized clinical trial. Arch Phys Med Rehabil. 1995 Jul;76(7):612-20. doi: 10.1016/s0003-9993(95)80629-6.

  PMID: 7605179 BACKGROUND

MeSH Terms

Conditions

Charcot-Marie-Tooth Disease

Interventions

Exercise

Condition Hierarchy (Ancestors)

Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Motor Activity; Movement; Musculoskeletal Physiological Phenomena; Musculoskeletal and Neural Physiological Phenomena

Study Officials

• Gita M Ramdharry, PhD

  University College London Hosptials NHS Trust

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principle Investigator

Study Record Dates

• First Submitted: November 28, 2016
• First Posted: December 5, 2016
• Study Start: December 1, 2016
• Primary Completion: March 1, 2017
• Study Completion: March 1, 2018
• Last Updated: May 3, 2018

Record last verified: 2018-05

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)