Whole-body Vibration in Children With Neurofibromatosis Type 1

NCT03888248 | Completed

• 1 other identifier: B00472
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Neurofibromatosis Type 1; Muscle Weakness

Keywords

Neurofibromatosis Type 1; Whole-body vibration; Muscle weakness

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline, 6 months

Secondary Outcomes (4)

• 6 minute walk test

  Baseline, 6 months, 9 months

• Accelerometer (duration)

  Baseline, 6 months, 9 months

• Accelerometer (intensity)

  Baseline, 6 months, 9 months

• PedsQL Multi-dimensional Fatigue Scale

  Baseline, 6 months, 9 months

Other Outcomes (14)

• Peak force (newtons) per kilogram on multiple one-legged hop using Leonardo Mechanography Ground Reaction Force Plate

  Baseline, 6 months, 9 months

• Efficiency of single two-legged jump using Leonardo Mechanography Ground Reaction Force Plate

  Baseline, 6 months, 9 months

• Balance tasks

  Baseline, 6 months, 9 months

Study Arms (2)

Exercises

ACTIVE COMPARATOR

Daily muscle-strengthening exercises

Other: Muscle strengthening exercises

Whole-body vibration + exercises

EXPERIMENTAL

Home-based whole-body vibration therapy plus daily muscle-strengthening exercises

Device: Whole-body vibration; Other: Muscle strengthening exercises

Interventions

Whole-body vibration DEVICE

Home-based whole-body vibration therapy by standing on Galileo Med 15 platform for 9 mins twice a day for 5 days per week

Whole-body vibration + exercises

Muscle strengthening exercises OTHER

Daily muscle strengthening exercises to be performed at home following a group education session by paediatric physiotherapist

Exercises; Whole-body vibration + exercises

Eligibility Criteria

• Age: 6 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Neurofibromatosis Type 1 (confirmed by NIH consensus criteria)
• Grip force less than -1 SDS according to age- and gender-matched normative data

You may not qualify if:

• 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)

Sponsors & Collaborators

• Manchester University NHS Foundation Trust: lead
• Manchester Metropolitan University: collaborator

Study Sites (1)

Manchester University Hospitals NHS Foundation Trust

Manchester, M13 9WL, United Kingdom

Location

MeSH Terms

Conditions

Neurofibromatosis 1; Muscle Weakness

Condition Hierarchy (Ancestors)

Neurofibromatoses; Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Manifestations; Neurologic Manifestations; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Signs and Symptoms

Study Officials

• Zulf Mughal

  Manchester University NHS Foundation Trust

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 20, 2019
• First Posted: March 25, 2019
• Study Start: July 22, 2019
• Primary Completion: November 30, 2020
• Study Completion: November 30, 2020
• Last Updated: March 16, 2021

Record last verified: 2021-03

Locations

GB (1)