RecruitingPhase 2Phase 3ACTRN12613000759796

Whole Body Vibration Training for Nemaline Myopathy

The effect of whole body vibration training on muscle, bone and motor function in children and adolescents with Nemaline Myopathy.

Sponsor

The Children's Hospital at Westmead

Enrollment

10 participants

Start Date

Mar 22, 2012

Study Type

Interventional

Conditions

Nemaline Myopathy

Summary

Nemaline myopathy (NM) is a form of genetic muscle disease characterised by the presence of numerous abnormal dense protein inclusions (nemaline bodies) on muscle biopsy. Children NM have varying degrees of muscle weakness with reduced functional capacity and exercise tolerance. Due to the strong association between muscle and bone (the muscle-bone unit) children with NM are also a risk of disuse osteopenia and minimal trauma fracture. To date, the optimal exercise regimen to increase muscle strength (force and power) is uncertain in NM. A training method that maintains muscle function throughout childhood and into adult life has the potential to improve the quality of life of children with NM. Whole body vibration training (WBVT) using a vibration platform is an emerging, simple and safe training method, which has been shown to improve muscle function and fitness in adults and children. In addition, WBVT has been shown to improve osteopenia in various populations. This project aims to evaluate the effect of 24 weeks WBVT on muscle and bone parameters in children with NM. Home-based Exercise Program for Children with Nemaline Myopathy: Ten children with nemaline myopathy, 4 – 18 years old will be enrolled into this study. The children will undertake a home-based WBVT program using the Galileo (trademark) vibration platform7 days a week for a 24 week (six-month) period. The WBVT period will be preceded by a 6-month observation period to establish the natural history of muscle function and bone density without intervention in each child. A series of investigations at baseline, the end of 6 months of observation and after 6 months of WBVT will evaluate at the effects of WBVT on muscle strength (force, power and efficiency), muscle size, functional mobility, bone strength and quality of life.

Eligibility

Sex: Both males and femalesMin Age: 4 YearssMax Age: 18 Yearss

Plain Language Summary

Simplified for easier understanding

This study is testing whether whole body vibration training (WBVT) — standing on a vibrating platform for short periods each day — can strengthen muscles and improve bone health in children and teenagers with nemaline myopathy, a rare genetic muscle disease. Children with this condition are often weaker than their peers and at risk of weak bones. The program runs for 6 months at home, preceded by a 6-month observation period to track what happens without treatment. You may be eligible if: - You are between 4 and 18 years old - You have a confirmed diagnosis of nemaline myopathy You may NOT be eligible if: - You have a cognitive impairment that would prevent you from completing the tests - Your vitamin D level is below 50 nmol/L (25-hydroxy vitamin D) - You had a lower limb fracture in the past 6 months - You are currently taking medication known to increase bone mass (e.g., bisphosphonates) Talk to your doctor about whether this trial might be right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

Home-based Exercise Program for Children with Nemaline Myopathy: Ten children with nemaline myopathy, 4 – 18 years old will be enrolled into this study. The children will undertake a home-based whole body vibration training (WBVT) program using the Galileo Trademark vibration platform 7 days a week for a 24 week (six-month) period. The WBVT period will be preceded by a 6-month observation period to establish the natural history of muscle function and bone density without intervention in each child. The Galileo vibration platform will be used for a period of 6 months. During the 6 month period, the Galileo vibration platform will be used 7 days per week for 9 minutes per day. The participant stands on the platform and may hold onto something for balance only but should not put their weight through their hands. During the operation of the platform, the participant should be supervised by a parent/caregiver. The vibration frequency will be increased over 3 days to the maximum of 24 Hz. The vibration amplitude will be 10mm. The WBVT will be suspended at any time during a session if the parent/caregiver feels the child is becoming distressed. Day 1: Vibration for 1 minute at 10Hz, 3 minutes rest, vibration for 1 minute at 10Hz, 3 minute rest, vibration for 2 minutes at 15Hz, 3 minute rest Day 2: Vibration for 2 minutes at 15Hz, 3 minutes rest, vibration for 2 minutes at 15Hz with 3 minutes rest, vibration for 3 minutes at 20Hz, 3 minute rest. Day 3: Vibration for 2 minutes at 20Hz, 3 minutes rest, vibration for 3 minutes at 20Hz with 3 minutes rest, Vibration for 3 minutes at 20Hz with 3 minutes rest. Day 4 onwards: Vibration for 3 minutes at 24Hz, 3 minutes rest, vibration for 3 minutes at 24Hz with 3 minute rest, Vibration for 3 minutes at 24Hz with 3 minute rest. It is important to keep in mind that the vibration training will be carried out as part of a therapy program. While the above protocol has been based on previous research in similar patient populations it may not be appropriate for all patients with nemaline myopathy. Some slight modofications to the above protocol may therefore be required if it is in the best interest of the study participant. A series of investigations at baseline, the end of 6 months of observation and after 6 months of WBVT will evaluate at the effects of WBVT on muscle strength (force, power and efficiency), muscle size, functional mobility, bone strength and quality of life.