None listed
Conditions
Brief summary
In the majority of individuals with mitochondrial respiratory chain disorders there is a steady decline with time of muscle mass and strength, and with reducing mobility there is a high risk of reduced bone mineral density and fracture. There are currently no therapies that have been shown to be consistently effective in this cohort of patients. Rett syndrome is a severe neurodevelopmental disorder. Amongst other clinical problems there is a high risk of reduced bone mass, primarily related to reduced mobility and nutritional intake, and poor perfusion of lower limb extremities. Aims: 1. To investigate if whole body vibration training is effective and well tolerated over the medium-term in patients with mitochondrial respiratory chain disorders or Rett syndrome. 2. A medium-term home based training program in patients with mitochondrial respiratory chain disorders will evaluate if whole body vibration training influences: a. Muscle power and force b. Muscle mass c. Bone mass 3. A medium-term home based training program in patients with Rett syndrome will evaluate if whole body vibration training influences: a. Muscle mass b. Bone mass c. Lower limb skin blood flow
Interventions
Whole body vibration training (WBVT) using a vibration platform.
WBVT using a vibration platform is a new, simple and safe training method that has been shown to improve muscle mass and bone density. WBVT has been used in a number of clinical settings including healthy athletes, untrained individuals and the elderly and in individuals with cerebral palsy and cystic fibrosis and after stroke. It requires the participant to stand on a vibration plate that moves rapidly (10-20Hz) up and down over
Whole body vibration training (WBVT) using a vibration platform. WBVT using a vibration platform is a new, simple and safe training method that has been shown to improve muscle mass and bone density. WBVT has been used in a number of clinical settings including healthy athletes, untrained individuals and the elderly and in individuals with cerebral palsy and cystic fibrosis and after stroke. It requires the participant to stand on a vibration plate that moves rapidly (10-20Hz) up and down over a small distance (10mm) generating a vibration stimulus. The vibration causes the muscles in the legs and body to contract resulting in increased muscle mass. WBVT is believed to stimulate bone formation due to the increased forces applied to the bone by the increased muscle mass seen after WBVT. The bone responds to these increased forces by altering its biomechanical properties (mineral content and geometry) to increase its density and strength. Participants will perform WBVT daily for six months on a vibration plate that will be placed in the participant’s home. Each daily session consists of three 3 minute WBVT sessions with short breaks in between each session. The total daily training time will take about 15 minutes.
Sponsors
Children's Hospital at Westmead
Study design
Allocation
Non-randomised trial
Intervention model
Single group
Primary purpose
Treatment
Masking
Open (masking not used)
Eligibility
Sex/Gender
All
Age
8 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
For the mitochondrial patients - Children and adolescents aged between 8 – 18 years with a proven mitochondrial respiratory chain disorder, based on positive enzymology in muscle biopsies for the Rett syndrome patients - Children with Rett syndrome, with known pathogenic Methyl CgP binding protein 2 (MECP2) mutations, up to the age of 18 years
Exclusion criteria
for mitochondrial patients - anything more than mild cognitive impairment which would impede the ability of test subjects to comply with testing; severe myopathy preventing subjects from being able to stand on the vibration platform for at least 10 minutes; 25-hydroxyvitamin D level < 50 nmol/L; lower limb fracture within the preceding 6 months; use of a medication known to increase bone mass, eg bisphosphonates for Rett syndrome patients - inability to stand unaided or with the support of a frame on the vibration platform for at least 10 minutes; 25-hydroxyvitamin D level < 50 nmol/L; limb fracture within the preceding 6 months; use of a medication known to increase bone mass, eg bisphosphonates
Outcome results
None listed