Neuromuscular Diseases (NMD)PETRA-NMDNCT06708468
Personalized Training for People With Rare Neuromuscular Disorders
A study testing Exercise in people with Neuromuscular Diseases (NMD) and Charcot Marie Tooth Disease (CMT), run by Oslo University Hospital at 5 sites in Norway.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT), Facioscapulohumeral Muscular Dystrophy
- What is being tested?
- Exercise
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 120 participants
- Where
- 5 sitesNorway
- Study dates
- 13 Dec 2024to Dec 2028
- Phase
- Interventional
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
Norway5 sites, 5 recruiting
- Not yet recruiting
Haukeland University Hospital
Bergen, 5021
Kristin N Varhaug, kristin.nielsen.varhaug@helse-bergen.no
- Not yet recruiting
Helse Møre and Romsdal HF (Molde Hospital)
Molde
Åse H Morsund, ase.hagen.morsund@helse-mr.no
- Not yet recruiting
Namsos Hospital
Namsos, 7803
Kristina Devik, kristina.devik@helse-nordtrondelag.no
- Recruiting
Oslo University Hospital
Oslo, 0424
Kristin Ørstavik, MD PhD, +4792422126, krorstav@ous-hf.no
Hanne L Fossmo, PhD stud, +4793630606, halufo@ous-hf.no
- Recruiting
University Hospital of North Norway
Tromsø, 9038
Kjell A Arntzen, MD PhD, Kjell.Arne.Arntzen@unn.no
Cathrine Ramberg, PhD, Cathrine.Ramberg@unn.no
About this study
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.
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:
Read more
- 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.
- Conditions
- Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT), Facioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy Type 1 (DM1)
- Being tested
- Exercise, Usual Care
- Keywords
- personalized training, rehabilitation, rare-neuromuscular disorders, motor unit number estimation, neuromuscular ultrasound, dual-energy x-ray absorptiometry, activity tracking, metabolomics, dynamic balance
- Medical subject headings
- Neuromuscular Diseases, Charcot-Marie-Tooth Disease, Muscular Dystrophy, Facioscapulohumeral, Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ExerciseOther
Also called Personalized 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.
Usual CareOther
Usual care includes appointments at hospitals and in the community health system according to what has been recommended to that individual
| Study group | Role | What they receive |
|---|---|---|
| Intervention | Experimental | The experimental group receives four-months of exercise-based intervention Receives: Other: Exercise |
| Control | Active comparator | The control group follows usual care Receives: Other: Usual Care |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dynamic balance assessed using Mini-Balance Evalulation Systems Test (Mini-BESTest)
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.
Measured: Baseline, 4-month (primary), follow-up at 10-month and 16-month (only interventiongroup)
Secondary outcomes (9)
Fatigue severity scale
Measurements of fatigue, 9 questions on a scale from 1 to 7. And a visual analogue fatigue scale
Measured: Baseline, 4-month, 10-month and 16-month (only intervention-group)
Clinical measures of strength and function
manual muscle testing, 30-s sit-to-stand, functional strength of neck and trunk, arm and hand function (9-HPT, Box and Block), walking function (10-mWT and 100-mTT), grip and pinch strength using dynamometers.
Measured: Baseline 4-month, 10-month and 16-month (only intervention group)
Global Metabolomics
dried blood samples for extracting signal metabolites of known importance in characterizing the metabolic status (e.g. lactate, amino acids, and fatty acids)
Measured: Baseline, 4-month, 10-month
Dual Energy X-ray Absorptiometry
body composition from different regions of the body
Measured: 4-month, 10-month and 16-month
Neuromuscular ultrasound
Ultrasound imaging of selected muscles in the trunk, upper- and lower extremities scored using visual and/or quantitative grey-scale echogenicity analysis tools. Only collected in participants with FSHD and DM1.
Measured: 4-month, 10-month and 16-month
Physical activity
tri-axial AX3 accelerometers, and monitoring pain, and activity and sleep diaries. Only collected in participants from Helse Sør-Øst.
Measured: 4-month, 10-month and 16-month
Motor unit number estimation
Estimating the number of motor units using non-invasive peripheral nerve stimulation and surface electromyography recordings. Only for participants with CMT at Helse Sør-Øst.
Measured: Baseline 4-month, 10-month and 16-month (only intervention-group)
ABC-scale
Activities- specific balance confidence scale.
Measured: Basleine, 4 month, 10 month, 16 month (only intervention group)
PROMIS-29
Questionnaire with 7 domains and a numeric rating scale
Measured: Baseline, 4 month, 10 month and 16 month (only intervention group)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Nov 2024
- Study start13 Dec 2024
- Primary completionDec 2027 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromuscular Diseases (NMD)Studies of the same condition, open and under way.
- More trials run by Oslo University HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 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.
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Oslo University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Universitetssykehuset i Nord-Norge HF, Norwegian School of Sport Sciences, Namsos Hospital, Frambu Centre for Rare Disorders, Vigør Rehabilitation Hospital, Kastvollen Rehabilitation Centre, Norwegian University of Science and Technology, Haukeland University Hospital, Molde Hospital, Drammen sykehus, Vikersund Bad Rehabiliteringssenter AS, Røde Kors Haugland rehabilitation center
- Responsible party
- Principal investigator, Kristin Ørstavik, Oslo University Hospital
- Investigators
- Kristin Ørstavik, MD, PhD, Principal investigator, Oslo University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06708468
- Sponsor's protocol ID
- 760722
- Acronym
- PETRA-NMD
- Status last verified
- Jun 2026
- First submitted
- 22 Nov 2024
- Last updated
- 18 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided