Neurofibromatosis Type 1NCT03888248
Whole-body Vibration in Children With Neurofibromatosis Type 1
A study testing Whole-body vibration and Muscle strengthening exercises in people with Neurofibromatosis Type 1 and Muscle Weakness, run by Manchester University NHS Foundation Trust at 1 site in United Kingdom.
Trial summary
It is known that children with NF1 can have weak muscles and suffer from tiredness. It is also known that in similar conditions affecting children's muscles, standing on a vibration device for a few minutes each day can strengthen muscles and improve their ability to perform day-to-day activities. Read more about this study
- Who is it for?
- Ages 6 to 16 years
- Conditions the study is for
- Neurofibromatosis Type 1, Muscle Weakness
- What is being tested?
- Whole-body vibration and Muscle strengthening exercises
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 22 Jul 2019to 30 Nov 2020
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Neurofibromatosis Type 1 (confirmed by NIH consensus criteria)
- Grip force less than -1 SDS according to age- and gender-matched normative data
Who cannot join
- Focal skeletal abnormalities (pseudoarthrosis, kyphoscoliosis likely to require surgery, known spondylolisthesis)
- Significant autistic spectrum disorder or learning difficulties (neurodevelopmental age less than 6 years)
- Inability to adequately weight-bear or balance on vibrating platform
- Lower limb plexiform causing limb length discrepancy
- Contraindications to use of whole-body vibration device (acute thrombosis, implants in activated regions of the body, acute inflammation of the locomotor system, herniae, propensity to fragility fractures or fracture within the past 3 months, history of renal calculi, epilepsy)
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Manchester University Hospitals NHS Foundation Trust
Manchester, M13 9WL
About this study
Official title: Whole-body Vibration Training Compared to Muscle-strengthening Exercises Alone in Improving Muscle Function in Children With Neurofibromatosis Type 1
It is known that children with NF1 can have weak muscles and suffer from tiredness. It is also known that in similar conditions affecting children's muscles, standing on a vibration device for a few minutes each day can strengthen muscles and improve their ability to perform day-to-day activities. The investigators believe this vibrating platform can be used to strengthen the muscles of children with NF1 also, beyond standard exercises, and therefore allow them to perform day-to-day activities better, do more activity and feel less tired.
If families are keen to take part in this study, the investigators will first need to check they are suitable for the trial. This will be based on the participant's age (6-16 years), their other medical problems that would affect use of the vibration device or tests to assess how effective it is, and their level of muscle weakness.
Read more
Once families have agreed to take part, participants will be randomised either to receive a muscle-strengthening exercise session delivered by a physiotherapist that children with weak muscles should continue to perform daily for the next 6 months; or to receive a vibration device to take home and use for a few minutes 5 days a week for 6 months as well as the exercises.
All participants will be invited to attend for a variety of activities and tests, just before starting the trial and 6 months later at the end of the trial to see if there is any benefit from the vibration device. The investigators will repeat these tests again 3 months later, to see if any benefits observed remain, even after the device is removed. They will involve jumping, hopping and balancing on a board, gripping a machine as hard and as long as possible, being fitted with a device that measures activity for 7 days, walking as far as possible for 6 minutes, scanning muscles using MRI, completing a tiredness and general well-being questionnaires, and parents completing a questionnaire of the participant's attention and intellect. The jumping will also be performed 3 months into the study, to see if there is any early improvement in this key test.
All participants will be given full details of what the trial involves before taking part. As with any other trial, participants and their families are free to stop taking part at any time. Although the investigators do not anticipate any safety issues, if any do arise, the families will be asked to contact the trial team.
Read the detailed description
Children with neurofibromatosis type 1 (NF1) have been shown in many studies to have reduced muscle mass, strength and function. This could potentially contribute to the fatigue and low quality of life reported in these children. However, to date interventions to improve muscle mass, strength and function in these children have not been established. Whole body vibration (WBV) has been demonstrated to improve walking speed and distance, muscle mass and strength, spasticity and balance in children with other neuromuscular conditions.
In this proposed preliminary matched-pairs interventional trial, the investigators aim to identify whether a home-based WBV therapy programme (9 minutes twice daily for 5 days per week) in addition to standard daily muscle-strengthening exercises improves muscle mass, strength and function over a six month period in children with NF1 aged 6-16 years, compared to daily muscle-strengthening exercises alone. Participants must have evidence of poor muscle strength, with grip force used to assess for this. Key exclusion criteria include significant learning difficulty or autistic spectrum disorder which would create difficulty in use of the WBV device, focal skeletal abnormalities and contraindications to WBV therapy.
The primary outcome measure will be dynamic muscle function using mechanography to measure peak power per kilogram on single two-legged jump.
Secondary/exploratory outcome measures include the 6-minute walk test (to assess lower limb muscle function and mobility), accelerometer data (as a surrogate marker of type and duration of physical activity), hand-held dynamometry (to assess upper limb grip force and endurance), measures on mechanography other than jumping power (to further assess dynamic muscle function), MRI for muscle cross-sectional area and signal intensity, fatigue and general well-being questionnaires and parental reports of attention and cognition.
These measures will be undertaken at baseline, on completion of intervention at 6 months, and again 3 months after to identify whether there is any sustained effect of the intervention following removal. Halfway through the intervention period (3 months), participants will also be invited just to complete the primary outcome of single two-legged jump on ground reaction force plate, as an interim measure.
The investigators hypothesise that WBV therapy will result in increased muscle mass and passive muscle strengthening of the lower limbs, which will translate into better muscle function in the lower limbs compared to baseline data and control group. As it is hypothesised that muscle weakness contributes to the fatigue demonstrated in children with NF1, the investigators would expect to see an improvement in their fatigue scores, which, combined with greater muscle strength, will translate into greater physical activity. The investigators hypothesise that WBV may also have an impact on muscle function in the upper limbs through central sensory stimulation and neuronal rewiring. Many children with NF1 have evidence of attention problems, and the investigators hypothesise that this may improve with WBV, again through central neuronal rewiring.
The trial will be conducted in compliance with this protocol, Good Clinical Practice (GCP) and applicable regulatory requirements.
- Conditions
- Neurofibromatosis Type 1, Muscle Weakness
- Being tested
- Whole-body vibration, Muscle strengthening exercises
- Keywords
- Neurofibromatosis Type 1, Whole-body vibration, Muscle weakness
- Medical subject headings
- Neurofibromatosis 1, Muscle Weakness
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Whole-body vibrationDevice
Home-based whole-body vibration therapy by standing on Galileo Med 15 platform for 9 mins twice a day for 5 days per week
Muscle strengthening exercisesOther
Daily muscle strengthening exercises to be performed at home following a group education session by paediatric physiotherapist
| Study group | Role | What they receive |
|---|---|---|
| Exercises | Active comparator | Daily muscle-strengthening exercises Receives: Other: Muscle strengthening exercises |
| Whole-body vibration + exercises | Experimental | Home-based whole-body vibration therapy plus daily muscle-strengthening exercises Receives: Device: Whole-body vibration; Other: Muscle strengthening exercises |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in peak power per kilogram on single two-legged jump using Leonardo Mechanography Ground Reaction Force Plate
Change from baseline to 6 months of peak power per kilogram of single two-legged jump using Leonardo Mechanography Ground Reaction Force Plate. Power will be measured in watts.
Measured: Baseline, 6 months
Secondary outcomes (4)
6 minute walk test
Distance in metres walked during 6 minutes
Measured: Baseline, 6 months, 9 months
Accelerometer (duration)
Duration of activity in minutes over 7 days using accelerometer
Measured: Baseline, 6 months, 9 months
Accelerometer (intensity)
Intensity of activity (accelerometer counts per minute) over 7 days using accelerometer
Measured: Baseline, 6 months, 9 months
PedsQL Multi-dimensional Fatigue Scale
Total score from age-appropriate validated questionnaire that asks for perceived fatigue within the domains of 'General', 'Sleep/Rest' and 'Cognitive'. Each domain consists of 6 questions, with participants/parents rating each question from 0 to 4 (therefore total score can range from 0 to 72). A higher total score indicates greater fatigue (i.e. worse outcome)
Measured: Baseline, 6 months, 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2019
- Study start22 Jul 2019
- Primary completion30 Nov 2020
- Study completion30 Nov 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Manchester University NHS Foundation Trust
- Sponsor type
- Other government
- Collaborators
- Manchester Metropolitan University
- Responsible party
- Sponsor
- Investigators
- Zulf Mughal, Principal investigator, Manchester University NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03888248
- Sponsor's protocol ID
- B00472
- Status last verified
- Mar 2021
- First submitted
- 20 Mar 2019
- Last updated
- 16 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Whole-body Vibration Training Compared to Muscle-strengthening Exercises Alone in Improving Muscle Function in Children With Neurofibromatosis Type 1
- ClinicalTrials.gov ID
- NCT03888248
- Acronym / sponsor ID
- B00472
- Conditions
- Neurofibromatosis Type 1, Muscle Weakness
- Keywords
- Neurofibromatosis Type 1, Whole-body vibration, Muscle weakness
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Manchester University NHS Foundation TrustOther government
- Collaborators
- Manchester Metropolitan University
- Study start
- 22 Jul 2019 (actual)
- Primary completion
- 30 Nov 2020 (actual)
- Study completion
- 30 Nov 2020 (actual)
- First posted
- 25 Mar 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
It is known that children with NF1 can have weak muscles and suffer from tiredness. It is also known that in similar conditions affecting children's muscles, standing on a vibration device for a few minutes each day can strengthen muscles and improve their ability to perform day-to-day activities. The investigators believe this vibrating platform can be used to strengthen the muscles of children with NF1 also, beyond standard exercises, and therefore allow them to perform day-to-day activities better, do more activity and feel less tired.
If families are keen to take part in this study, the investigators will first need to check they are suitable for the trial. This will be based on the participant's age (6-16 years), their other medical problems that would affect use of the vibration device or tests to assess how effective it is, and their level of muscle weakness.
Once families have agreed to take part, participants will be randomised either to receive a muscle-strengthening exercise session delivered by a physiotherapist that children with weak muscles should continue to perform daily for the next 6 months; or to receive a vibration device to take home and use for a few minutes 5 days a week for 6 months as well as the exercises.
All participants will be invited to attend for a variety of activities and tests, just before starting the trial and 6 months later at the end of the trial to see if there is any benefit from the vibration device. The investigators will repeat these tests again 3 months later, to see if any benefits observed remain, even after the device is removed. They will involve jumping, hopping and balancing on a board, gripping a machine as hard and as long as possible, being fitted with a device that measures activity for 7 days, walking as far as possible for 6 minutes, scanning muscles using MRI, completing a tiredness and general well-being questionnaires, and parents completing a questionnaire of the participant's attention and intellect. The jumping will also be performed 3 months into the study, to see if there is any early improvement in this key test.
All participants will be given full details of what the trial involves before taking part. As with any other trial, participants and their families are free to stop taking part at any time. Although the investigators do not anticipate any safety issues, if any do arise, the families will be asked to contact the trial team.
Detailed description
Children with neurofibromatosis type 1 (NF1) have been shown in many studies to have reduced muscle mass, strength and function. This could potentially contribute to the fatigue and low quality of life reported in these children. However, to date interventions to improve muscle mass, strength and function in these children have not been established. Whole body vibration (WBV) has been demonstrated to improve walking speed and distance, muscle mass and strength, spasticity and balance in children with other neuromuscular conditions.
In this proposed preliminary matched-pairs interventional trial, the investigators aim to identify whether a home-based WBV therapy programme (9 minutes twice daily for 5 days per week) in addition to standard daily muscle-strengthening exercises improves muscle mass, strength and function over a six month period in children with NF1 aged 6-16 years, compared to daily muscle-strengthening exercises alone. Participants must have evidence of poor muscle strength, with grip force used to assess for this. Key exclusion criteria include significant learning difficulty or autistic spectrum disorder which would create difficulty in use of the WBV device, focal skeletal abnormalities and contraindications to WBV therapy.
The primary outcome measure will be dynamic muscle function using mechanography to measure peak power per kilogram on single two-legged jump.
Secondary/exploratory outcome measures include the 6-minute walk test (to assess lower limb muscle function and mobility), accelerometer data (as a surrogate marker of type and duration of physical activity), hand-held dynamometry (to assess upper limb grip force and endurance), measures on mechanography other than jumping power (to further assess dynamic muscle function), MRI for muscle cross-sectional area and signal intensity, fatigue and general well-being questionnaires and parental reports of attention and cognition.
These measures will be undertaken at baseline, on completion of intervention at 6 months, and again 3 months after to identify whether there is any sustained effect of the intervention following removal. Halfway through the intervention period (3 months), participants will also be invited just to complete the primary outcome of single two-legged jump on ground reaction force plate, as an interim measure.
The investigators hypothesise that WBV therapy will result in increased muscle mass and passive muscle strengthening of the lower limbs, which will translate into better muscle function in the lower limbs compared to baseline data and control group. As it is hypothesised that muscle weakness contributes to the fatigue demonstrated in children with NF1, the investigators would expect to see an improvement in their fatigue scores, which, combined with greater muscle strength, will translate into greater physical activity. The investigators hypothesise that WBV may also have an impact on muscle function in the upper limbs through central sensory stimulation and neuronal rewiring. Many children with NF1 have evidence of attention problems, and the investigators hypothesise that this may improve with WBV, again through central neuronal rewiring.
The trial will be conducted in compliance with this protocol, Good Clinical Practice (GCP) and applicable regulatory requirements.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Exercises | Active comparator | Other: Muscle strengthening exercisesDaily muscle-strengthening exercises |
| Whole-body vibration + exercises | Experimental | Device: Whole-body vibration, Other: Muscle strengthening exercisesHome-based whole-body vibration therapy plus daily muscle-strengthening exercises |
Who may qualify
- Ages
- 6 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Neurofibromatosis Type 1 (confirmed by NIH consensus criteria)
- Grip force less than -1 SDS according to age- and gender-matched normative data
Key exclusion criteria (5 listed)
- Focal skeletal abnormalities (pseudoarthrosis, kyphoscoliosis likely to require surgery, known spondylolisthesis)
- Significant autistic spectrum disorder or learning difficulties (neurodevelopmental age less than 6 years)
- Inability to adequately weight-bear or balance on vibrating platform
- Lower limb plexiform causing limb length discrepancy
- Contraindications to use of whole-body vibration device (acute thrombosis, implants in activated regions of the body, acute inflammation of the locomotor system, herniae, propensity to fragility fractures or fracture within the past 3 months, history of renal calculi, epilepsy)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Whole-body vibrationHome-based whole-body vibration therapy by standing on Galileo Med 15 platform for 9 mins twice a day for 5 days per week | Device | Whole-body vibration + exercises |
| Muscle strengthening exercisesDaily muscle strengthening exercises to be performed at home following a group education session by paediatric physiotherapist | Other | Exercises, Whole-body vibration + exercises |
Primary outcome
Change in peak power per kilogram on single two-legged jump using Leonardo Mechanography Ground Reaction Force Plate
Time frame: Baseline, 6 months
Change from baseline to 6 months of peak power per kilogram of single two-legged jump using Leonardo Mechanography Ground Reaction Force Plate. Power will be measured in watts.
4 secondary outcomes
6 minute walk test
Time frame: Baseline, 6 months, 9 months
Accelerometer (duration)
Time frame: Baseline, 6 months, 9 months
Accelerometer (intensity)
Time frame: Baseline, 6 months, 9 months
PedsQL Multi-dimensional Fatigue Scale
Time frame: Baseline, 6 months, 9 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Mar 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Zulf Mughal principal investigator
Manchester University NHS Foundation Trust
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Whole-body vibration and Muscle strengthening exercises in people with Neurofibromatosis Type 1 and Muscle Weakness, run by Manchester University NHS Foundation Trust at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in peak power per kilogram on single two-legged jump using Leonardo Mechanography Ground Reaction Force Plate (time frame: Baseline, 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Mar 2021.