Clinical trial NCT06708468: Personalized Training for People With Rare Neuromuscular Disorders
Official title: Personalized Exercise Training for People With Rare Neuromuscular Disorders: a Multi-center, Evaluator-blinded, Two Arm, Randomized Controlled Study to Assess the Effects on Physical Function From Personalized Strength and Balance Exercise in a Rehabilitation Setting.
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Neuromuscular Diseases (NMD)
Charcot Marie Tooth Disease (CMT)
Facioscapulohumeral Muscular Dystrophy
Myotonic Dystrophy Type 1 (DM1)
Keywords: personalized training
rehabilitation
rare-neuromuscular disorders
motor unit number estimation
neuromuscular ultrasound
dual-energy x-ray absorptiometry
activity tracking
metabolomics
dynamic balance
Lead sponsor: Oslo University Hospital
Sponsor class: OTHER
Collaborators: name: Universitetssykehuset i Nord-Norge HF
name: Norwegian School of Sport Sciences
name: Namsos Hospital
name: Frambu Centre for Rare Disorders
name: Vigør Rehabilitation Hospital
name: Kastvollen Rehabilitation Centre
name: Norwegian University of Science and Technology
name: Haukeland University Hospital
name: Molde Hospital
name: Drammen sykehus
name: Vikersund Bad Rehabiliteringssenter AS
name: Røde Kors Haugland rehabilitation center
First posted: 2024-11-27
Enrollment: 120
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: 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.
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: 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

Primary outcomes:
measure: 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)
Secondary outcomes:
measure: Fatigue severity scale
timeFrame: Baseline, 4-month, 10-month and 16-month (only intervention-group)
measure: Clinical measures of strength and function
timeFrame: Baseline 4-month, 10-month and 16-month (only intervention group)
measure: Global Metabolomics
timeFrame: Baseline, 4-month, 10-month
measure: Dual Energy X-ray Absorptiometry
timeFrame: 4-month, 10-month and 16-month
measure: Neuromuscular ultrasound
timeFrame: 4-month, 10-month and 16-month
measure: Physical activity
timeFrame: 4-month, 10-month and 16-month
measure: Motor unit number estimation
timeFrame: Baseline 4-month, 10-month and 16-month (only intervention-group)
measure: ABC-scale
timeFrame: Basleine, 4 month, 10 month, 16 month (only intervention group)
measure: PROMIS-29
timeFrame: Baseline, 4 month, 10 month and 16 month (only intervention group)
Other outcomes:
Locations:
city: Bergen
country: Norway
facility: Haukeland University Hospital
city: Molde
country: Norway
facility: Helse Møre and Romsdal HF (Molde Hospital)
city: Namsos
country: Norway
facility: Namsos Hospital
city: Oslo
country: Norway
facility: Oslo University Hospital
city: Tromsø
country: Norway
facility: University Hospital of North Norway
Study officials:
affiliation: Oslo University Hospital
name: Kristin Ørstavik, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: