None listed
Conditions
Brief summary
Congenital myopathies refers to a group of muscle disorders that appears in infancy. It results in muscle weakness due to abnormalities within the muscle cell. Congenital myopathies as a group are one of the most common neuromuscular disorders. There are no effective medicines or exercise regimes that currently exist to improve the muscle weakness for these children. Their muscle weakness leads to difficulty in doing normal physical activities, like getting dressed, walking, climbing stairs or opening jars. We think that a home exercise program could increase their muscle size and strength, to help them perform physical activities better. We think the home exercise programme could also improve the strength of their bones, reducing the chance of a fracture, and improve their quality of life. The home exercise programme will use Whole body vibration training (WBVT). To use WBVT, children stand on a specialised vibration platform that moves like a see-saw, a few millimetres at a time, exercising the muscles, for a few minutes a day. WBVT is an emerging, simple and safe training method, which has been shown to improve muscle function and fitness in adults and children, and we think could help children with congenital myopathies also. Children in the study will undergo comprehensive clinical assessments at baseline and after the 3 month WBVT treatment period. This will then be compared with an observation period of 3 months. Children will then repeat the WBVT treatment for another 3 months, followed by a further observation period of 3 months with assessments after each stage. This novel therapy is non-invasive, simple, and potentially could help affected children maintain their ability to walk, perform physical activities, and improve their quality of life.
Interventions
Treatment
Whole Body Vibration Training (WBVT) will be performed on the Galileo Basic vibration plate (Novotec Medical, Pforzheim, Germany). Each session will consist of three 3-minute bouts of training, interspersed with a 3-minute rest. Sessions will be performed four times a week, over two 3 month periods. This study will have a single intervention group, comparing baseline (T0), a control period of 3 months (T1), with 3 months treatment, followed by post-treatment outcomes (T2). To establish
Treatment Whole Body Vibration Training (WBVT) will be performed on the Galileo Basic vibration plate (Novotec Medical, Pforzheim, Germany). Each session will consist of three 3-minute bouts of training, interspersed with a 3-minute rest. Sessions will be performed four times a week, over two 3 month periods. This study will have a single intervention group, comparing baseline (T0), a control period of 3 months (T1), with 3 months treatment, followed by post-treatment outcomes (T2). To establish efficacy of the first intervention period, and determine its duration of effect, a further 3 month control period (T3) and 3 month treatment phase (T4) will be repeated. A final 3 month control period (T5) will complete the project. Participants will act as their own controls. Participants will start with sessions of three 1-minute bouts at 12 Hz, and both intensity and duration will be gradually increased according to the response of each individual. However, by the end of week 4, all participants should be training at the prescribed protocol of 3 sets of 3 minutes at 20 Hz and 1 mm amplitude, 4 times a week. Training intensity will be maintained at 20 Hz for the remainder of the intervention. This training protocol is a well validated clinical paradigm used in previous published literature. Participants will stand barefoot on the plate. An adjustable metal frame will initially be used with all participants until they build up confidence and are able to safely support themselves during the vibration training. For participants with poor balance, the frame will be maintained at all times to safely regain balance when necessary. Training sessions will be performed at home. An experienced exercise physiologist and/or a specialist physiotherapist from the research team will supervise the participants performing training at home or school once a week, in order to monitor progress and provide feedback/support. It is the participant's personal preference whether the training will be at home or school. Parents/caregivers will provide ongoing supervision of home sessions. Participants will be asked to maintain a training diary to monitor compliance. Data recorded will include the date, intensity and duration of training, as well as any comments regarding adverse events, tiredness, or pain.
Sponsors
Liggins Institute at the University of Auckland
Study design
Allocation
Non-randomised trial
Primary purpose
Prevention
Eligibility
Sex/Gender
All
Age
4 Years to 16 Years
Healthy volunteers
No
Inclusion criteria
Participants will be aged 4- 16 years and have a confirmed diagnosis of Congenital Myopathy via muscle biopsy and/or genetic testing Participants are able to: i) stand on the vibration platform for the duration of treatment sessions, and ii) undergo some of the clinical and functional assessments necessary to evaluate treatment outcomes.
Exclusion criteria
1. Recent lower limb fracture and not cleared by an orthopaedic surgeon to participate in the study 2. Cognitive impairment which might affect the ability of participants to comply with testing procedures 3) History of another illness or findings on physical examination, which in the opinion of the investigators would prevent the patient from completing the study; 4) Use for greater than or equal to 30 days of medication that might interfere with study results and assessments within 3 months of enrolment (e.g. anabolic agents);
Outcome results
None listed