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Recruiting NCT06708468

Personalized Training for People With Rare Neuromuscular Disorders

No phase Interventional Neuromuscular Diseases (NMD) Charcot Marie Tooth Disease (CMT) Facioscapulohumeral Muscular Dystrophy Myotonic Dystrophy Type 1 (DM1)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Exercise, Usual Care.
Who it may be relevant to
Registry conditions: Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT), Facioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy Type 1 (DM1). Basic parameters: 18 years — 70 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Norway
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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.

Overview

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.

Interventions

  • Other Exercise
    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.
  • Other Usual Care
    Usual care includes appointments at hospitals and in the community health system according to what has been recommended to that individual

Primary outcome measures

  • 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)]
Secondary outcome measures (9)
  • Fatigue severity scale [Time frame: Baseline, 4-month, 10-month and 16-month (only intervention-group)]
  • Clinical measures of strength and function [Time frame: Baseline 4-month, 10-month and 16-month (only intervention group)]
  • Global Metabolomics [Time frame: Baseline, 4-month, 10-month]
  • Dual Energy X-ray Absorptiometry [Time frame: 4-month, 10-month and 16-month]
  • Neuromuscular ultrasound [Time frame: 4-month, 10-month and 16-month]
  • Physical activity [Time frame: 4-month, 10-month and 16-month]
  • Motor unit number estimation [Time frame: Baseline 4-month, 10-month and 16-month (only intervention-group)]
  • ABC-scale [Time frame: Basleine, 4 month, 10 month, 16 month (only intervention group)]
  • PROMIS-29 [Time frame: Baseline, 4 month, 10 month and 16 month (only intervention group)]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Single blind
Primary purpose
Treatment

Study locations

Norway · 5 centers
  • Haukeland University Hospital — Bergen
  • Helse Møre and Romsdal HF (Molde Hospital) — Molde
  • Namsos Hospital — Namsos
  • Oslo University Hospital — Oslo
  • University Hospital of North Norway — Tromsø

Identifiers

NCT: NCT06708468 · 760722

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗