Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12613000759796
Ethics application status
Approved
Date submitted
5/07/2013
Date registered
8/07/2013
Date last updated
8/07/2013
Type of registration
Retrospectively registered

Titles & IDs
Public title
Whole Body Vibration Training for Nemaline Myopathy
Scientific title
The effect of whole body vibration training on muscle, bone and motor function in children and adolescents with Nemaline Myopathy.
Secondary ID [1] 282788 0
None
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Nemaline Myopathy 289556 0
Condition category
Condition code
Neurological 289884 289884 0 0
Other neurological disorders
Musculoskeletal 289885 289885 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 289886 289886 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
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.
Intervention code [1] 287472 0
Treatment: Devices
Comparator / control treatment
None
Control group
Uncontrolled

Outcomes
Primary outcome [1] 289945 0
Muscle function will be assessed through Leonardo Jumping Platform, Isokinetic Cybex dynamometer and the Modified Bruce Protocol Treadmill Test.
Timepoint [1] 289945 0
Baseline, 6 months after standard therapy and after 3 and 6 months of WBVT
Primary outcome [2] 289946 0
Muscle size will be assessed through Peripheral Quantitative Computer Tomography (pQCT) of non-dominant Tibia and Magnetic Resonance Imaging (non-dominant thigh muscle cross sectional area).
Timepoint [2] 289946 0
Baseline, 6 months observation and after 6 months WBVT
Primary outcome [3] 289947 0
Bone Measurers assessed through Peripheral Quantitative Computer Tomography (pQCT) of non-dominant Tibia and total body and lumbar spine DXA.
Timepoint [3] 289947 0
Baseline, 6 months observation and after 6 months WBVT
Secondary outcome [1] 303630 0
Lower limb motor function assessed via activities performed on the Leonardo Jumping Platform (jumping and chair raise test)
Timepoint [1] 303630 0
Baseline, 6 months observation and after 6 months WBVT)
Secondary outcome [2] 303631 0
Overall motor function assessed via the 20 item Motor Function Measure and 6 minute walk test.
Timepoint [2] 303631 0
Baseline, 6 months observation and after 6 months WBVT
Secondary outcome [3] 303632 0
Motor Function Measure
Timepoint [3] 303632 0
Baseline, 6 months observation and after 6 months WBVT
Secondary outcome [4] 303633 0
Quality of life assessed using the PedsQL Neuromuscular module.
Timepoint [4] 303633 0
Baseline, 6 months observation and after 6 months WBVT
Secondary outcome [5] 303634 0
Dietary assessment assessed using a questionnaire (written specifically for the study) to determine calcium intake.
Timepoint [5] 303634 0
Baseline, 6 months observation and after 6 months WBVT
Secondary outcome [6] 303635 0
Biochemical analyses Blood will be collected for mineral homeostasis(calcium, phosphorus, alkaline phosphatase, vitamin D and parathyroid hormone) and bone turn over (osteocalcin, NTX, PINP)
Timepoint [6] 303635 0
Baseline, 6 months observation and after 6 months WBVT
Secondary outcome [7] 303636 0
Compliance with whole body vibration training assessed via diary entries completed by the family.
Timepoint [7] 303636 0
Compliance is measured during the 6 months WBVT intervention period by a training diary that is filled out every time the participant uses the plate. The clinical evaluator also rings the family once every month to check how things are going.

Eligibility
Key inclusion criteria
Children and adolescents aged between 4 – 18 years with nemaline myopathy.
Minimum age
4 Years
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Cognitive impairment which would impede the ability of test subjects to comply with testing; 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Phase 2 / Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
22/03/2012
Actual
22/03/2012
Date of last participant enrolment
Anticipated
31/12/2013
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
10
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 1211 0
The Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 7126 0
2145 - Westmead

Funding & Sponsors
Funding source category [1] 287563 0
Charities/Societies/Foundations
Name [1] 287563 0
A Foundation Building Strength for Nemaline Myopathy
Country [1] 287563 0
United States of America
Primary sponsor type
Hospital
Name
The Children's Hospital at Westmead
Address
Cnr of Hawkesbury Rd and Hainsworth St,
Westmead, NSW 2145
Country
Australia
Secondary sponsor category [1] 286312 0
None
Name [1] 286312 0
Address [1] 286312 0
Country [1] 286312 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 289538 0
Sydney Childrens Hospitals Network Human Research Ethics Committee
Ethics committee address [1] 289538 0
The Children's Hospital at Westmead,
Cnr of Hawkesbury Rd and Hainsworth St,
Westmead NSW 2145
Ethics committee country [1] 289538 0
Australia
Date submitted for ethics approval [1] 289538 0
Approval date [1] 289538 0
05/07/2011
Ethics approval number [1] 289538 0
11/CHW/79

Summary
Brief 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.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 41250 0
A/Prof Craig Munns
Address 41250 0
Institute of Endocrinology and Diabetes,
Cnr of Hawkesbury Rd and Hainsworth St,
Westmead NSW 2145
Country 41250 0
Australia
Phone 41250 0
+61298453200
Fax 41250 0
Email 41250 0
[email protected]
Contact person for public queries
Name 41251 0
A/Prof Craig Munns
Address 41251 0
Institute of Endocrinology and Diabetes,
Cnr of Hawkesbury Rd and Hainsworth St,
Westmead NSW 2145
Country 41251 0
Australia
Phone 41251 0
+61298453200
Fax 41251 0
Email 41251 0
[email protected]
Contact person for scientific queries
Name 41252 0
A/Prof Craig Munns
Address 41252 0
Institute of Endocrinology and Diabetes,
Cnr of Hawkesbury Rd and Hainsworth St,
Westmead NSW 2145
Country 41252 0
Australia
Phone 41252 0
+61298453200
Fax 41252 0
Email 41252 0
[email protected]

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

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