The goal of this study is to investigate the effects of personalized exercise treatment on dynamic balance and physical function in comparison with regular follow-up in adults with rare-neuromuscular disorders: Charcot-Marie-Tooth (CMT), Facioscapulohumeral Muscular Dystrophy (FSHD), and Myotonic Dystrophy Type 1 (DM1). The key objectives are: 1. To investigate if the intervention group experiences improvements in dynamic balance that are superior to the control group 2. To investigate if the intervention group experiences long-term improvements in dynamic balance that are superior to the control group during the follow-up 3. To investigate if improvements in dynamic balance are associated with improvements in physical activity, body composition, estimated motor units, metabolomics, muscle echnogenecity and volume, and other indicators of health and quality of life. This is a national study and will involve 120 individuals with rare-neuromuscular disorders from Norway's four health regions.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
120
The intervention includes 12-day personalized exercise program set in a rehabilitation center, followed by a three-month digital intervention and then a second 12-day personalized exercise program in a rehabilitation center.
Usual care includes appointments at hospitals and in the community health system according to what has been recommended to that individual
Haukeland University Hospital
Bergen, Norway
NOT_YET_RECRUITINGHelse Møre and Romsdal HF (Molde Hospital)
Molde, Norway
NOT_YET_RECRUITINGNamsos Hospital
Namsos, Norway
NOT_YET_RECRUITINGOslo University Hospital
Oslo, Norway
RECRUITINGUniversity Hospital of North Norway
Tromsø, Norway
RECRUITINGDynamic balance assessed using Mini-Balance Evalulation Systems Test (Mini-BESTest)
The primary outcome measure will be change in dynamic balance from pre-intervention to post-intervention. In addition, there will be two follow-up assessments.
Time frame: Baseline, 4-month (primary), follow-up at 10-month and 16-month (only interventiongroup)
Fatigue severity scale
Measurements of fatigue, 9 questions on a scale from 1 to 7. And a visual analogue fatigue scale
Time frame: Baseline, 4-month, 10-month and 16-month (only intervention-group)
Clinical measures of strength and function
manual muscle testing, 30-s sit-to-stand, functional strength of neck and trunk, arm and hand function (9-HPT, Box and Block), walking function (10-mWT and 100-mTT), grip and pinch strength using dynamometers.
Time frame: Baseline 4-month, 10-month and 16-month (only intervention group)
Global Metabolomics
dried blood samples for extracting signal metabolites of known importance in characterizing the metabolic status (e.g. lactate, amino acids, and fatty acids)
Time frame: Baseline, 4-month, 10-month
Dual Energy X-ray Absorptiometry
body composition from different regions of the body
Time frame: 4-month, 10-month and 16-month
Neuromuscular ultrasound
Ultrasound imaging of selected muscles in the trunk, upper- and lower extremities scored using visual and/or quantitative grey-scale echogenicity analysis tools. Only collected in participants with FSHD and DM1.
Time frame: 4-month, 10-month and 16-month
Physical activity
tri-axial AX3 accelerometers, and monitoring pain, and activity and sleep diaries. Only collected in participants from Helse Sør-Øst.
Time frame: 4-month, 10-month and 16-month
Motor unit number estimation
Estimating the number of motor units using non-invasive peripheral nerve stimulation and surface electromyography recordings. Only for participants with CMT at Helse Sør-Øst.
Time frame: Baseline 4-month, 10-month and 16-month (only intervention-group)
ABC-scale
Activities- specific balance confidence scale.
Time frame: Basleine, 4 month, 10 month, 16 month (only intervention group)
PROMIS-29
Questionnaire with 7 domains and a numeric rating scale
Time frame: Baseline, 4 month, 10 month and 16 month (only intervention group)
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