Ignite Creation Date:
2025-12-24 @ 5:43 PM
Ignite Modification Date:
2025-12-24 @ 5:43 PM
Study NCT ID:
NCT06708468
Status:
RECRUITING
Last Update Posted:
2025-04-27
First Post:
2024-11-22
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Personalized Training for People With Rare Neuromuscular Disorders
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D002607', 'term': 'Charcot-Marie-Tooth Disease'}, {'id': 'D020391', 'term': 'Muscular Dystrophy, Facioscapulohumeral'}, {'id': 'D009223', 'term': 'Myotonic Dystrophy'}], 'ancestors': [{'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D015417', 'term': 'Hereditary Sensory and Motor Neuropathy'}, {'id': 'D009421', 'term': 'Nervous System Malformations'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D011115', 'term': 'Polyneuropathies'}, {'id': 'D010523', 'term': 'Peripheral Nervous System Diseases'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D020967', 'term': 'Myotonic Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D015444', 'term': 'Exercise'}], 'ancestors': [{'id': 'D009043', 'term': 'Motor Activity'}, {'id': 'D009068', 'term': 'Movement'}, {'id': 'D009142', 'term': 'Musculoskeletal Physiological Phenomena'}, {'id': 'D055687', 'term': 'Musculoskeletal and Neural Physiological Phenomena'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'SINGLE', 'whoMasked': ['OUTCOMES_ASSESSOR'], 'maskingDescription': '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.'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': '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.'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 120}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-12-13', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-04', 'completionDateStruct': {'date': '2028-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-04-23', 'studyFirstSubmitDate': '2024-11-22', 'studyFirstSubmitQcDate': '2024-11-25', 'lastUpdatePostDateStruct': {'date': '2025-04-27', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-11-27', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Dynamic balance assessed using Mini-Balance Evalulation Systems Test (Mini-BESTest)', 'timeFrame': 'Baseline, 4-month (primary), follow-up at 10-month and 16-month (only interventiongroup)', 'description': '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.'}], 'secondaryOutcomes': [{'measure': 'Fatigue severity scale', 'timeFrame': 'Baseline, 4-month, 10-month and 16-month (only intervention-group)', 'description': 'Measurements of fatigue, 9 questions on a scale from 1 to 7. And a visual analogue fatigue scale'}, {'measure': 'Clinical measures of strength and function', 'timeFrame': 'Baseline 4-month, 10-month and 16-month (only intervention group)', 'description': '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.'}, {'measure': 'Global Metabolomics', 'timeFrame': 'Baseline, 4-month, 10-month', 'description': 'dried blood samples for extracting signal metabolites of known importance in characterizing the metabolic status (e.g. lactate, amino acids, and fatty acids)'}, {'measure': 'Dual Energy X-ray Absorptiometry', 'timeFrame': '4-month, 10-month and 16-month', 'description': 'body composition from different regions of the body'}, {'measure': 'Neuromuscular ultrasound', 'timeFrame': '4-month, 10-month and 16-month', 'description': '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.'}, {'measure': 'Physical activity', 'timeFrame': '4-month, 10-month and 16-month', 'description': 'tri-axial AX3 accelerometers, and monitoring pain, and activity and sleep diaries. Only collected in participants from Helse Sør-Øst.'}, {'measure': 'Motor unit number estimation', 'timeFrame': 'Baseline 4-month, 10-month and 16-month (only intervention-group)', 'description': '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.'}, {'measure': 'ABC-scale', 'timeFrame': 'Basleine, 4 month, 10 month, 16 month (only intervention group)', 'description': 'Activities- specific balance confidence scale.'}, {'measure': 'PROMIS-29', 'timeFrame': 'Baseline, 4 month, 10 month and 16 month (only intervention group)', 'description': 'Questionnaire with 7 domains and a numeric rating scale'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['personalized training', 'rehabilitation', 'rare-neuromuscular disorders', 'motor unit number estimation', 'neuromuscular ultrasound', 'dual-energy x-ray absorptiometry', 'activity tracking', 'metabolomics', 'dynamic balance'], 'conditions': ['Neuromuscular Diseases (NMD)', 'Charcot Marie Tooth Disease (CMT)', 'Facioscapulohumeral Muscular Dystrophy', 'Myotonic Dystrophy Type 1 (DM1)']}, 'descriptionModule': {'briefSummary': "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).\n\nThe key objectives are:\n\n1. To investigate if the intervention group experiences improvements in dynamic balance that are superior to the control group\n2. To investigate if the intervention group experiences long-term improvements in dynamic balance that are superior to the control group during the follow-up\n3. 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.\n\nThis is a national study and will involve 120 individuals with rare-neuromuscular disorders from Norway's four health regions."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '70 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* A confirmed diagnosis of either FSHD, DM1 or CMT\n* 18-70 years of age at the time of signing the informed consent.\n* Any gender\n* Ability to stand, rise from a chair and walk at least 10 meters with or without any need of assistive devices\n* Indication for rehabilitation as confirmed by the treating neurologist or physiotherapist\n* Ability to understand and follow instructions in Norwegian\n* Capable of giving signed informed consent\n\nExclusion Criteria:\n\n* Pregnancy or planning to become pregnant\n* Any other neurological or non-neurological disorders affecting physical capacity, such as disabling arthritis, severe heart-failure/cardiomyopathy, on-going cancer treatment\n* Alcohol or drug abuse as per their medical chart\n* History of non-compliance to medical advice/follow-up'}, 'identificationModule': {'nctId': 'NCT06708468', 'acronym': 'PETRA-NMD', 'briefTitle': 'Personalized Training for People With Rare Neuromuscular Disorders', 'organization': {'class': 'OTHER', 'fullName': 'Oslo University Hospital'}, 'officialTitle': '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.', 'orgStudyIdInfo': {'id': '760722'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Intervention', 'description': 'The experimental group receives four-months of exercise-based intervention', 'interventionNames': ['Other: Exercise']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'Control', 'description': 'The control group follows usual care', 'interventionNames': ['Other: Usual Care']}], 'interventions': [{'name': 'Exercise', 'type': 'OTHER', 'otherNames': ['Personalized Exercise'], 'description': '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.', 'armGroupLabels': ['Intervention']}, {'name': 'Usual Care', 'type': 'OTHER', 'description': 'Usual care includes appointments at hospitals and in the community health system according to what has been recommended to that individual', 'armGroupLabels': ['Control']}]}, 'contactsLocationsModule': {'locations': [{'zip': '5021', 'city': 'Bergen', 'status': 'NOT_YET_RECRUITING', 'country': 'Norway', 'contacts': [{'name': 'Kristin N Varhaug', 'role': 'CONTACT', 'email': 'kristin.nielsen.varhaug@helse-bergen.no'}], 'facility': 'Haukeland University Hospital', 'geoPoint': {'lat': 60.39299, 'lon': 5.32415}}, {'city': 'Molde', 'status': 'NOT_YET_RECRUITING', 'country': 'Norway', 'contacts': [{'name': 'Åse H Morsund', 'role': 'CONTACT', 'email': 'ase.hagen.morsund@helse-mr.no'}], 'facility': 'Helse Møre and Romsdal HF (Molde Hospital)', 'geoPoint': {'lat': 62.73752, 'lon': 7.15912}}, {'zip': '7803', 'city': 'Namsos', 'status': 'NOT_YET_RECRUITING', 'country': 'Norway', 'contacts': [{'name': 'Kristina Devik', 'role': 'CONTACT', 'email': 'kristina.devik@helse-nordtrondelag.no'}], 'facility': 'Namsos Hospital', 'geoPoint': {'lat': 64.46624, 'lon': 11.49572}}, {'zip': '0424', 'city': 'Oslo', 'status': 'RECRUITING', 'country': 'Norway', 'contacts': [{'name': 'Kristin Ørstavik, MD PhD', 'role': 'CONTACT', 'email': 'krorstav@ous-hf.no', 'phone': '+4792422126'}, {'name': 'Hanne L Fossmo, PhD stud', 'role': 'CONTACT', 'email': 'halufo@ous-hf.no', 'phone': '+4793630606'}, {'name': 'Tarun Arora, PhD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Kristian B Nilsen, MD PhD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Katja P Elgstøen, PhD', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'Oslo University Hospital', 'geoPoint': {'lat': 59.91273, 'lon': 10.74609}}, {'zip': '9038', 'city': 'Tromsø', 'status': 'RECRUITING', 'country': 'Norway', 'contacts': [{'name': 'Kjell A Arntzen, MD PhD', 'role': 'CONTACT', 'email': 'Kjell.Arne.Arntzen@unn.no'}, {'name': 'Cathrine Ramberg, PhD', 'role': 'CONTACT', 'email': 'Cathrine.Ramberg@unn.no'}], 'facility': 'University Hospital of North Norway', 'geoPoint': {'lat': 69.6489, 'lon': 18.95508}}], 'centralContacts': [{'name': 'Kristin Ørstavik, MD, PhD', 'role': 'CONTACT', 'email': 'krorstav@ous-hf.no', 'phone': '+4792422126'}, {'name': 'Hanne L Fossmo, PhD stud', 'role': 'CONTACT', 'email': 'halufo@ous-hf.no', 'phone': '+4793630606'}], 'overallOfficials': [{'name': 'Kristin Ørstavik, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Oslo University Hospital'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Oslo University Hospital', 'class': 'OTHER'}, 'collaborators': [{'name': 'Universitetssykehuset i Nord-Norge HF', 'class': 'UNKNOWN'}, {'name': 'Norwegian School of Sport Sciences', 'class': 'OTHER'}, {'name': 'Namsos Hospital', 'class': 'OTHER'}, {'name': 'Frambu Centre for Rare Disorders', 'class': 'UNKNOWN'}, {'name': 'Vigør Rehabilitation Hospital', 'class': 'UNKNOWN'}, {'name': 'Kastvollen Rehabilitation Centre', 'class': 'UNKNOWN'}, {'name': 'Norwegian University of Science and Technology', 'class': 'OTHER'}, {'name': 'Haukeland University Hospital', 'class': 'OTHER'}, {'name': 'Molde Hospital', 'class': 'OTHER'}, {'name': 'Drammen sykehus', 'class': 'OTHER'}, {'name': 'Vikersund Bad Rehabiliteringssenter AS', 'class': 'UNKNOWN'}, {'name': 'Røde Kors Haugland rehabilitation center', 'class': 'OTHER'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'head of Section, MD, PhD', 'investigatorFullName': 'Kristin Ørstavik', 'investigatorAffiliation': 'Oslo University Hospital'}}}}