Personalized Training for People With Rare Neuromuscular Disorders (NCT06708468) | Clinical Trial Compass
RecruitingNot Applicable
Personalized Training for People With Rare Neuromuscular Disorders
Norway120 participantsStarted 2024-12-13
Plain-language summary
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.
Who can participate
Age range
18 Years – 70 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* A confirmed diagnosis of either FSHD, DM1 or CMT
* 18-70 years of age at the time of signing the informed consent.
* Any gender
* Ability to stand, rise from a chair and walk at least 10 meters with or without any need of assistive devices
* Indication for rehabilitation as confirmed by the treating neurologist or physiotherapist
* Ability to understand and follow instructions in Norwegian
* Capable of giving signed informed consent
Exclusion Criteria:
* Pregnancy or planning to become pregnant
* Any other neurological or non-neurological disorders affecting physical capacity, such as disabling arthritis, severe heart-failure/cardiomyopathy, on-going cancer treatment
* Alcohol or drug abuse as per their medical chart
* History of non-compliance to medical advice/follow-up
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Dynamic balance assessed using Mini-Balance Evalulation Systems Test (Mini-BESTest)
Timeframe: Baseline, 4-month (primary), follow-up at 10-month and 16-month (only interventiongroup)