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| Name | Class |
|---|---|
| Universitetssykehuset i Nord-Norge HF | UNKNOWN |
| Norwegian School of Sport Sciences | OTHER |
| Namsos Hospital | OTHER |
| Frambu Centre for Rare Disorders |
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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:
This is a national study and will involve 120 individuals with rare-neuromuscular disorders from Norway's four health regions.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Intervention | Experimental | The experimental group receives four-months of exercise-based intervention |
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| Control | Active Comparator | The control group follows usual care |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Exercise | Other | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Dynamic 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. | Baseline, 4-month (primary), follow-up at 10-month and 16-month (only interventiongroup) |
| Measure | Description | Time Frame |
|---|---|---|
| Fatigue severity scale | Measurements of fatigue, 9 questions on a scale from 1 to 7. And a visual analogue fatigue scale | Baseline, 4-month, 10-month and 16-month (only intervention-group) |
| Clinical measures of strength and function |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Kristin Ørstavik, MD, PhD | Contact | +4792422126 | krorstav@ous-hf.no | |
| Hanne L Fossmo, PhD stud | Contact | +4793630606 | halufo@ous-hf.no |
| Name | Affiliation | Role |
|---|---|---|
| Kristin Ørstavik, MD, PhD | Oslo University Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Haukeland University Hospital | Not yet recruiting | Bergen | 5021 | Norway |
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| ID | Term |
|---|---|
| D009468 | Neuromuscular Diseases |
| D002607 | Charcot-Marie-Tooth Disease |
| D020391 | Muscular Dystrophy, Facioscapulohumeral |
| D009223 | Myotonic Dystrophy |
| ID | Term |
|---|---|
| D009422 | Nervous System Diseases |
| D015417 | Hereditary Sensory and Motor Neuropathy |
| D009421 | Nervous System Malformations |
| D020271 | Heredodegenerative Disorders, Nervous System |
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| ID | Term |
|---|---|
| D015444 | Exercise |
| ID | Term |
|---|---|
| D009043 | Motor Activity |
| D009068 | Movement |
| D009142 | Musculoskeletal Physiological Phenomena |
| D055687 | Musculoskeletal and Neural Physiological Phenomena |
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| UNKNOWN |
| Vigør Rehabilitation Hospital | UNKNOWN |
| Kastvollen Rehabilitation Centre | UNKNOWN |
| Norwegian University of Science and Technology | OTHER |
| Haukeland University Hospital | OTHER |
| Molde Hospital | OTHER |
| Drammen sykehus | OTHER |
| Vikersund Bad Rehabiliteringssenter AS | UNKNOWN |
| Røde Kors Haugland rehabilitation center | OTHER |
This is a multi-site randomized control trial with two study arms - interventional and control group that will be investigated pre-and-post four-month intervention, and at 10-month and 16-month for follow-up. The participants will involve three different rare-NMDs (CMT, FSHD, DM1) from the four health regions of Norway - South-Eastern, Northern, Central, and Western. The participants will be randomly allocated to either the intervention or control group. The interventional group will undergo a personalized four month exercise training program at a rehabilitation centre followed by at-home digital exercises; whereas, the control group will receive usual care. The two groups will be stratified by study site, diagnosis and sex.
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The evaluators will be blinded to the treatment group of the participants. It is not possible to blind the participants themselves; however, they will be asked not to share their group allocation with the others, including the evaluators. A staff member may unblind the intervention assignment for any participant with a serious adverse event.
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| Usual Care | Other | Usual care includes appointments at hospitals and in the community health system according to what has been recommended to that individual |
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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.
| 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) | Baseline, 4-month, 10-month |
| Dual Energy X-ray Absorptiometry | body composition from different regions of the body | 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. | 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. | 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. | Baseline 4-month, 10-month and 16-month (only intervention-group) |
| ABC-scale | Activities- specific balance confidence scale. | Basleine, 4 month, 10 month, 16 month (only intervention group) |
| PROMIS-29 | Questionnaire with 7 domains and a numeric rating scale | Baseline, 4 month, 10 month and 16 month (only intervention group) |
| Helse Møre and Romsdal HF (Molde Hospital) | Not yet recruiting | Molde | Norway |
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| Namsos Hospital | Not yet recruiting | Namsos | 7803 | Norway |
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| Oslo University Hospital | Recruiting | Oslo | 0424 | Norway |
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| University Hospital of North Norway | Recruiting | Tromsø | 9038 | Norway |
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| D019636 | Neurodegenerative Diseases |
| D011115 | Polyneuropathies |
| D010523 | Peripheral Nervous System Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D020967 | Myotonic Disorders |