Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12616001471471
Ethics application status
Approved
Date submitted
19/10/2016
Date registered
21/10/2016
Date last updated
17/09/2020
Date data sharing statement initially provided
12/11/2018
Date results information initially provided
17/09/2020
Type of registration
Prospectively registered

Titles & IDs
Public title
Whole body vibration training in Congenital Myopathies
Scientific title
Whole-body vibration training as a therapy to improve mobility, muscle and bone health in children and adolescents with Congenital Myopathy
Secondary ID [1] 290355 0
nil known
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Congenital Myopathy 300644 0
Condition category
Condition code
Physical Medicine / Rehabilitation 300488 300488 0 0
Other physical medicine / rehabilitation
Musculoskeletal 300507 300507 0 0
Other muscular and skeletal disorders
Neurological 300508 300508 0 0
Neurodegenerative diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
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.
Intervention code [1] 296170 0
Prevention
Intervention code [2] 296178 0
Treatment: Devices
Comparator / control treatment
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.
Control group
Active

Outcomes
Primary outcome [1] 299917 0
Change in function and mobility as assessed by the 6 minute walk test
Timepoint [1] 299917 0
Baseline (T0), after a control period of 3 months (T1), after 3 months treatment (T2) after a further 3 month control period (T3), after a second 3 month treatment phase (T4), and after a final 3 month control period (T5)
Secondary outcome [1] 328515 0
Change in bone health/osteoporosis (composite) measured with comparison of bone mineral density through the DEXA and pQCT scans.
Timepoint [1] 328515 0
Assessed at each time point (T0, 1, 2, 3, 4, 5) ie;
baseline (T0), after a control period of 3 months (T1), after 3 months treatment (T2) after a further 3 month control period (T3), after a second 3 month treatment phase (T4), and after a final 3 month control period (T5)
Secondary outcome [2] 341009 0
Muscle function assessment as measured by:
Motor Function Measure dimensions 1 and 2
Timepoint [2] 341009 0
Baseline (T0), after 3 months control (T1), 3 months treatment (T2), further 3 months control (T3), further 3 months treatment (T4), after a final 3 month control period (T5)
Secondary outcome [3] 341010 0
General health measure:
Blood pressure using a sphygmomanometer
Timepoint [3] 341010 0
Baseline (T0), after 3 months control (T1), 3 months treatment (T2), further 3 months control (T3), further 3 months treatment (T4), after a final 3 month control period (T5)
Secondary outcome [4] 341011 0
Quality of life questionnaire using the PedsQl 3.0 neuromuscular module
Timepoint [4] 341011 0
At the start of the study (T0), after the first 3 months of treatment (T2), and at the end of the study, after the final 3 month control period (T5)
Secondary outcome [5] 341012 0
10 meter walk/run test
Timepoint [5] 341012 0
Baseline (T0), after 3 months control (T1), 3 months treatment (T2), further 3 months control (T3), further 3 months treatment (T4), after a final 3 month control period (T5)
Secondary outcome [6] 341013 0
Physical Activity Monitor (ActivPal) will quantify percentage time in sedentary, upright and ambulatory activities, The pattern (sedentary, standing, stepping) and intensity of a subject’s activities will be monitored.
Timepoint [6] 341013 0
Baseline (T0), after 3 months control (T1), 3 months treatment (T2), further 3 months control (T3), further 3 months treatment (T4), after a final 3 month control period (T5)
Secondary outcome [7] 341014 0
General health measure:
Height using a stadiometer
Timepoint [7] 341014 0
Baseline (T0), after 3 months control (T1), 3 months treatment (T2), further 3 months control (T3), further 3 months treatment (T4), after a final 3 month control period (T5)
Secondary outcome [8] 341015 0
Respiratory function using a portable spirometer (Micromed)
Timepoint [8] 341015 0
Baseline (T0), after 3 months control (T1), 3 months treatment (T2), further 3 months control (T3), further 3 months treatment (T4), after a final 3 month control period (T5)

Eligibility
Key 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.
Minimum age
4 Years
Maximum age
16 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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);

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



Intervention assignment
Other design features
Phase
Not Applicable
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Date of first participant enrolment
Anticipated
1/12/2017
Actual
5/03/2018
Date of last participant enrolment
Anticipated
1/04/2019
Actual
1/04/2019
Date of last data collection
Anticipated
1/11/2020
Actual
Sample size
Target
15
Accrual to date
Final
12
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 9477 0
The Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 18210 0
2145 - Westmead
Recruitment outside Australia
Country [1] 8329 0
New Zealand
State/province [1] 8329 0

Funding & Sponsors
Funding source category [1] 294737 0
Charities/Societies/Foundations
Name [1] 294737 0
Starship Foundation
Country [1] 294737 0
New Zealand
Funding source category [2] 294747 0
Charities/Societies/Foundations
Name [2] 294747 0
Neuromuscular Research Foundation Trust
Country [2] 294747 0
New Zealand
Funding source category [3] 294748 0
Charities/Societies/Foundations
Name [3] 294748 0
Neurological Foundation of New Zealand
Country [3] 294748 0
New Zealand
Primary sponsor type
University
Name
Liggins Institute at the University of Auckland
Address
85 Park Road, Grafton, Auckand, 1023
Private Bag 92019 Auckland Mail Centre
Auckland, 1142, New Zealand
Country
New Zealand
Secondary sponsor category [1] 293600 0
None
Name [1] 293600 0
Address [1] 293600 0
Country [1] 293600 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 296154 0
Central HDEC
Ethics committee address [1] 296154 0
Ministry of Health
Ethics Department
Freyberg Building
Reception – Ground Floor
20 Aitken Street
Wellington 6011
Ethics committee country [1] 296154 0
New Zealand
Date submitted for ethics approval [1] 296154 0
24/10/2016
Approval date [1] 296154 0
14/07/2017
Ethics approval number [1] 296154 0

Summary
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.
Trial website
Trial related presentations / publications
Public notes
Attachments [1] 1184 1184 0 0
/AnzctrAttachments/371683-WBVT STUDY PROTOCOL.docx (Protocol)
Attachments [2] 1187 1187 0 0
/AnzctrAttachments/371683-WBVT PIS and consent.docx (Participant information/consent)

Contacts
Principal investigator
Name 69790 0
Prof Paul Hofman
Address 69790 0
Liggins Institute
University of Auckland
Private Bag 92019
Grafton
Auckland 1142
Country 69790 0
New Zealand
Phone 69790 0
+64 9 923 6453
Fax 69790 0
Email 69790 0
[email protected]
Contact person for public queries
Name 69791 0
Dr Lisa Power
Address 69791 0
Liggins Institute
University of Auckland
Private Bag 92019
Grafton
Auckland 1142
Country 69791 0
New Zealand
Phone 69791 0
+64 9 3737599 ext 86098
Fax 69791 0
Email 69791 0
[email protected]
Contact person for scientific queries
Name 69792 0
Dr Lisa Power
Address 69792 0
Liggins Institute
University of Auckland
Private Bag 92019
Grafton
Auckland 1142
Country 69792 0
New Zealand
Phone 69792 0
+64 9 3737599 ext 86098
Fax 69792 0
Email 69792 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.