Personalized Training for People With Rare Neuromuscular Disorders

NCT ID: NCT06708468

Last Updated: 2025-04-27

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

NA

Total Enrollment

120 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-12-13

Study Completion Date

2028-12-31

Brief Summary

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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.

Detailed Description

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Conditions

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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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 Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors
The evaluators will be blinded to the treatment group of the participants. It is not possible to blind the participants themselves; however, they will be asked not to share their group allocation with the others, including the evaluators. A staff member may unblind the intervention assignment for any participant with a serious adverse event.

Study Groups

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Intervention

The experimental group receives four-months of exercise-based intervention

Group Type EXPERIMENTAL

Exercise

Intervention Type OTHER

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.

Control

The control group follows usual care

Group Type ACTIVE_COMPARATOR

Usual Care

Intervention Type OTHER

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

Interventions

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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.

Intervention Type OTHER

Usual Care

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

Intervention Type OTHER

Other Intervention Names

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Personalized Exercise

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Universitetssykehuset i Nord-Norge HF

UNKNOWN

Sponsor Role collaborator

Norwegian School of Sport Sciences

OTHER

Sponsor Role collaborator

Namsos Hospital

OTHER

Sponsor Role collaborator

Frambu Centre for Rare Disorders

UNKNOWN

Sponsor Role collaborator

Vigør Rehabilitation Hospital

UNKNOWN

Sponsor Role collaborator

Kastvollen Rehabilitation Centre

UNKNOWN

Sponsor Role collaborator

Norwegian University of Science and Technology

OTHER

Sponsor Role collaborator

Haukeland University Hospital

OTHER

Sponsor Role collaborator

Molde Hospital

OTHER

Sponsor Role collaborator

Drammen sykehus

OTHER

Sponsor Role collaborator

Vikersund Bad Rehabiliteringssenter AS

UNKNOWN

Sponsor Role collaborator

Røde Kors Haugland rehabilitation center

OTHER

Sponsor Role collaborator

Oslo University Hospital

OTHER

Sponsor Role lead

Responsible Party

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Kristin Ørstavik

head of Section, MD, PhD

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Kristin Ørstavik, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Oslo University Hospital

Locations

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Haukeland University Hospital

Bergen, , Norway

Site Status NOT_YET_RECRUITING

Helse Møre and Romsdal HF (Molde Hospital)

Molde, , Norway

Site Status NOT_YET_RECRUITING

Namsos Hospital

Namsos, , Norway

Site Status NOT_YET_RECRUITING

Oslo University Hospital

Oslo, , Norway

Site Status RECRUITING

University Hospital of North Norway

Tromsø, , Norway

Site Status RECRUITING

Countries

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Norway

Central Contacts

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Kristin Ørstavik, MD, PhD

Role: CONTACT

+4792422126

Hanne L Fossmo, PhD stud

Role: CONTACT

+4793630606

Facility Contacts

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Kristin N Varhaug

Role: primary

Åse H Morsund

Role: primary

Kristina Devik

Role: primary

Kristin Ørstavik, MD PhD

Role: primary

+4792422126

Hanne L Fossmo, PhD stud

Role: backup

+4793630606

Kjell A Arntzen, MD PhD

Role: primary

Cathrine Ramberg, PhD

Role: backup

Other Identifiers

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760722

Identifier Type: -

Identifier Source: org_study_id

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