Actively Recruiting

Phase Not Applicable
Age: 18Years - 70Years
All Genders
ID06708468

Personalized Exercise Training for People With Rare Neuromuscular Disorders A Multi-center, Evaluator-blinded, Two Arm, Randomized Controlled Study Assessing Effects on Physical Function From Personalized Strength and Balance Exercise in Rehabilitation

Led by Oslo University Hospital · Updated on 2025-04-27

120

Participants Needed

5

Research Sites

104 weeks

Total Duration

On this page

Sponsors

O

Oslo University Hospital

Lead Sponsor

U

Universitetssykehuset i Nord-Norge HF

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are evaluating the effects of personalized exercise treatment on dynamic balance and physical function in adults with rare neuromuscular disorders such as Charcot-Marie-Tooth disease, Facioscapulohumeral Muscular Dystrophy, and Myotonic Dystrophy Type 1. This study aims to determine if the exercise intervention improves dynamic balance more than regular follow-up care, and if these improvements last over time. The study also explores associations between balance improvements and other health indicators like physical activity, body composition, and quality of life in 120 participants from Norway's four health regions. Participants are randomly assigned to one of two groups: an experimental group receiving a personalized exercise program or a control group receiving usual care. The exercise intervention includes two 12-day personalized training sessions at a rehabilitation center separated by a three-month digital program. The control group continues with their regular hospital and community health appointments as recommended. The study is evaluator-blinded and follows participants over several months. During the study, participants undergo assessments at baseline, 4 months, 10 months, and 16 months. These include dynamic balance tests using the Mini-Balance Evaluation Systems Test, fatigue scales, clinical strength and function measurements, metabolomics, body composition scans, neuromuscular ultrasound, physical activity monitoring, motor unit estimation, and quality-of-life questionnaires. The intervention group has additional follow-up assessments at 10 and 16 months. This comprehensive monitoring helps researchers understand the effects and sustainability of personalized exercise on physical function and health in this population.

CONDITIONS

Brief Title

Personalized Training for People With Rare Neuromuscular Disorders

Who Can Participate

Age: 18Years - 70Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Confirmed diagnosis of Facioscapulohumeral Muscular Dystrophy (FSHD), Myotonic Dystrophy Type 1 (DM1), or Charcot-Marie-Tooth disease (CMT)
  • Age between 18 and 70 years at the time of consent
  • Any gender
  • Ability to stand, rise from a chair, and walk at least 10 meters with or without assistive devices
  • Indication for rehabilitation confirmed by a neurologist or physiotherapist
  • Ability to understand and follow instructions in Norwegian
  • Capable of giving signed informed consent
Not Eligible

You will not qualify if you...

  • Pregnancy or planning to become pregnant
  • Other neurological or non-neurological disorders affecting physical capacity, such as disabling arthritis, severe heart failure or cardiomyopathy, or ongoing cancer treatment
  • Alcohol or drug abuse as recorded in medical charts
  • History of not following medical advice or follow-up

AI-Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Approximately 4 months

Participants in the experimental group complete a 12-day personalized exercise program in a rehabilitation center, followed by a three-month digital exercise intervention, and then a second 12-day personalized exercise program in a rehabilitation center. Participants in the control group follow their usual care with hospital and community health appointments as recommended.

2 in-person rehabilitation visits of 12 days each and ongoing digital intervention over 3 months

Follow-up

Duration - Up to 12 months after treatment

Participants in the intervention group are assessed for longer-term outcomes at 10 and 16 months after baseline to monitor the effects of the personalized exercise training.

Follow-up visits at 10 months and 16 months for intervention group only

Trial Site Locations

Total: 5 locations

1

Haukeland University Hospital

Bergen, Norway, 5021

Not Yet Recruiting

2

Helse Møre and Romsdal HF (Molde Hospital)

Molde, Norway

Not Yet Recruiting

3

Namsos Hospital

Namsos, Norway, 7803

Not Yet Recruiting

4

Oslo University Hospital

Oslo, Norway, 0424

Actively Recruiting

5

University Hospital of North Norway

Tromsø, Norway, 9038

Actively Recruiting

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Research Team

K

Kristin Ørstavik, MD, PhD

H

Hanne L Fossmo, PhD stud

How is the study designed?

Study Type

INTERVENTIONAL

Masking

SINGLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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