Personalized Training for People With Rare Neuromuscular Disorders

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.

Conditions

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

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

Sponsors & Collaborators

• Universitetssykehuset i Nord-Norge HF

  collaborator UNKNOWN

• Norwegian School of Sport Sciences

  collaborator OTHER

• Namsos Hospital

  collaborator OTHER

• Frambu Centre for Rare Disorders

  collaborator UNKNOWN

• Vigør Rehabilitation Hospital

  collaborator UNKNOWN

• Kastvollen Rehabilitation Centre

  collaborator UNKNOWN

• Norwegian University of Science and Technology

  collaborator OTHER

• Haukeland University Hospital

  collaborator OTHER

• Molde Hospital

  collaborator OTHER

• Drammen sykehus

  collaborator OTHER

• Vikersund Bad Rehabiliteringssenter AS

  collaborator UNKNOWN

• Røde Kors Haugland rehabilitation center

  collaborator OTHER

• Oslo University Hospital

  lead OTHER

Principal Investigators

• Kristin Ørstavik, MD, PhD · Oslo University Hospital

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Model

PARALLEL

Eligibility

Min Age

18 Years

Max Age

70 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2024-12-13

Primary Completion

2026-12-31

Completion

2028-12-31

Countries

• Norway

Study Locations