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.

Oslo University Hospital5 sites in 1 country120 target enrollmentDecember 13, 2024

ConditionsNeuromuscular Diseases (NMD)Charcot Marie Tooth Disease (CMT)Facioscapulohumeral Muscular Dystrophy Myotonic Dystrophy Type 1 (DM1)

Overview

Phase: N/A
Intervention: Not specified
Conditions: Neuromuscular Diseases (NMD)
Sponsor: Oslo University Hospital
Enrollment: 120
Locations: 5
Primary Endpoint: Dynamic balance assessed using Mini-Balance Evalulation Systems Test (Mini-BESTest)
Status: Recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

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:

To investigate if the intervention group experiences improvements in dynamic balance that are superior to the control group
To investigate if the intervention group experiences long-term improvements in dynamic balance that are superior to the control group during the follow-up
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.

Registry

clinicaltrials.gov

Start Date

December 13, 2024

End Date

December 2028

Last Updated

last year

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Oslo University Hospital

Responsible Party

Principal Investigator

Kristin Ørstavik

head of Section, MD, PhD

Oslo University Hospital

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

Outcomes

Primary Outcomes

Dynamic balance assessed using Mini-Balance Evalulation Systems Test (Mini-BESTest)

Time Frame: Baseline, 4-month (primary), follow-up at 10-month and 16-month (only interventiongroup)

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.

Secondary Outcomes

Fatigue severity scale(Baseline, 4-month, 10-month and 16-month (only intervention-group))
Global Metabolomics(Baseline, 4-month, 10-month)
Dual Energy X-ray Absorptiometry(4-month, 10-month and 16-month)
Physical activity(4-month, 10-month and 16-month)
ABC-scale(Basleine, 4 month, 10 month, 16 month (only intervention group))
PROMIS-29(Baseline, 4 month, 10 month and 16 month (only intervention group))
Clinical measures of strength and function(Baseline 4-month, 10-month and 16-month (only intervention group))
Neuromuscular ultrasound(4-month, 10-month and 16-month)
Motor unit number estimation(Baseline 4-month, 10-month and 16-month (only intervention-group))