[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"neuromuscular-diseases-nmd\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:neuromuscular-diseases-nmd":33},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,13,0,[8,69,111,145,166,219,231,258,313,354,387,417,440],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":42,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":58,"lastUpdatePostDateStruct":59,"startDateStruct":62,"completionDateStruct":64,"leadSponsor":66,"locationsCount":4},"100053565","phase-3-a-phase-3-study-to-evaluate-the-safety-and-efficacy-of-aoc-1044-also-referred-to-as-delpacibart-zotadirsen-in-participants-with-dmd-with-gene-mutations-amenable-to-exon-44-skipping-100053565",false,"NCT07587242","A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping","A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous AOC 1044 (Delpacibart Zotadirsen) for the Treatment of DMD With Gene Mutations Amenable to Exon 44 Skipping","SAFARI44","Key Inclusion Criteria:\n\n* Ambulatory males with clinical and genetic diagnosis of DMD\n* Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping\n* 7 to 16 years of age at time of consent\n* TTR and NSAA assessment completed within the protocol specified parameters at Screening\n* On a stable regimen of corticosteroids (including Vamolorone) for at least 6 months prior to Day 1. Steroid regimen must be anticipated to remain stable.\n\nKey Exclusion Criteria:\n\n* Previous treatment cell or gene therapy.\n* Treatment with another oligonucleotide within 6 months of informed consent (not including COVID-19 RNA vaccines).\n* Lab values outside of the protocol specified range at Screening\n* If on any of the following treatments (growth hormone, testosterone or givinostat), participants must be on a stable regimen and must plan to maintain it for the duration of the study. Participants will be excluded if regimen stability prior to informed consent is as follows:\n* Less than 1 month, for growth hormone and\u002For testosterone\n* Less than 6 months for givinostat","MALE","7 Years","16 Years",{"count":21,"type":22},70,"ESTIMATED","INTERVENTIONAL",[25],"PHASE3","A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping",[28,29,30,31,32,33,34,35,36,37,38,39,40,41],"Muscular Dystrophies","Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)","Muscular Disorders, Atrophic","Muscular Disease","Musculoskeletal Diseases","Neuromuscular Diseases (NMD)","Nervous System Diseases","Genetic Diseases","X-Linked","Hereditary","Neonatal Disease","Duchene Muscular Dystrophy","Congenital","DMD",[43,44,45,46,47,48,49,50,15,51,52,53,54,55,56,41],"AOC","AOC 1044","AOC 1044-CS3","AOC 1044-CS1","AOC 1044-CS2","EXPLORE44","EXPLORE44-OLE","SAFARI","SAFARI 44","Avidity","Avidity Biosciences","Exon Skipping Therapy","Avidity Biosciences Inc., A Novartis Company","del-zota","NOT_YET_RECRUITING","2026-07-10",{"date":60,"type":61},"2026-07-13","ACTUAL",{"date":63,"type":22},"2026-06",{"date":65,"type":22},"2030-07",{"name":67,"class":68},"Avidity Biosciences, Inc.","INDUSTRY",{"id":70,"slug":71,"hasResults":11,"nctId":72,"briefTitle":73,"officialTitle":74,"acronym":75,"eligibilityCriteria":76,"healthyVolunteers":11,"sex":77,"minAge":78,"maxAge":79,"enrollmentInfo":80,"targetDuration":4,"studyType":23,"phases":82,"briefSummary":84,"conditions":85,"keywords":89,"overallStatus":99,"whyStopped":4,"lastUpdateSubmitDate":100,"lastUpdatePostDateStruct":101,"startDateStruct":103,"completionDateStruct":105,"leadSponsor":107,"locationsCount":110},"100570532","personalized-training-for-people-with-rare-neuromuscular-disorders-100570532","NCT06708468","Personalized Training for People With Rare Neuromuscular Disorders","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.","PETRA-NMD","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\u002Fcardiomyopathy, on-going cancer treatment\n* Alcohol or drug abuse as per their medical chart\n* History of non-compliance to medical advice\u002Ffollow-up","ALL","18 Years","70 Years",{"count":81,"type":22},120,[83],"NA","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.",[33,86,87,88],"Charcot Marie Tooth Disease (CMT)","Facioscapulohumeral Muscular Dystrophy","Myotonic Dystrophy Type 1 (DM1)",[90,91,92,93,94,95,96,97,98],"personalized training","rehabilitation","rare-neuromuscular disorders","motor unit number estimation","neuromuscular ultrasound","dual-energy x-ray absorptiometry","activity tracking","metabolomics","dynamic balance","RECRUITING","2026-06-17",{"date":102,"type":61},"2026-06-18",{"date":104,"type":61},"2024-12-13",{"date":106,"type":22},"2028-12",{"name":108,"class":109},"Oslo University Hospital","OTHER",5,{"id":112,"slug":113,"hasResults":11,"nctId":114,"briefTitle":115,"officialTitle":116,"acronym":4,"eligibilityCriteria":117,"healthyVolunteers":11,"sex":77,"minAge":78,"maxAge":4,"enrollmentInfo":118,"targetDuration":4,"studyType":23,"phases":120,"briefSummary":121,"conditions":122,"keywords":126,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":136,"lastUpdatePostDateStruct":137,"startDateStruct":139,"completionDateStruct":140,"leadSponsor":142,"locationsCount":144},"100643406","3d-hand-orthosis-trial-100643406","NCT07633821","3D Hand Orthosis Trial","Cost-effectiveness of 3D-printed Hand Orthoses for Activities of Daily Living in Chronic Hand Conditions","Inclusion Criteria:\n\n* Diagnosed with a chronic, stable hand condition due to a neurological disorder, musculoskeletal disorder, neuromuscular disease or injury;\n* Minimum age of 18 years;\n* Indicated for a first or repeat prescription of a circular thumb, wrist or wrist-thumb orthosis for permanent use for improving ADL performance.\n\nExclusion Criteria:\n\n* Indicated for an orthosis for a non-functional hand;\n* Insufficient mastery of the Dutch language.",{"count":119,"type":22},100,[83],"In people with chronic hand conditions, hand orthoses are frequently prescribed to improve performance in activities of daily living (ADL). Conventional hand orthoses are custom-made on a plaster cast of the hand, a process that is time-consuming and labor-intensive. It has been demonstrated that the production time of manufacturing hand orthoses can be reduced by using 3-dimensional scanning and printing (i.e. 3D-printed hand orthosis), offering a promising cost-effective alternative to conventional hand orthoses. The current study builds on a previously conducted feasibility study, which demonstrated comparable effects of 3D-printed and conventional hand orthoses on ADL performance, hand function, and quality of life in people with chronic hand conditions. User satisfaction and production time favored the 3D-printed orthoses. However, to date only small and self-controlled studies have investigated the effects of 3D-printed versus conventional hand orthoses for permanent use on ADL performance and orthosis satisfaction in chronic hand conditions. Evidence from randomized controlled trials and data on the cost-effectiveness are lacking. The aims of this study are:\n\n1. To determine whether treatment with 3D-printed hand orthoses is non-inferior compared to treatment with conventional hand orthoses in terms of ADL performance, hand function, pain, quality of life and functional status in individuals with chronic hand conditions.\n2. To assess whether treatment with 3D-printed hand orthoses results in greater patient satisfaction compared to treatment with conventional hand orthoses.\n3. To assess the cost-effectiveness of treatment with 3D-printed hand orthoses compared to treatment with conventional hand orthoses.",[123,124,33,125],"Hand Injuries and Disorders","Neurological Conditions","Musculoskeletal Conditions (e.g., Tendinitis, Capsulitis)",[127,128,129,130,131,132,133,134,135],"Orthotic Devices","Cost-Effectiveness Analysis","Hand","Printing, Three-Dimensional","Activities of Daily Living","Rehabilitation","Quality of Life","Non-Inferiority Trial","Patient Satisfaction","2026-06-05",{"date":138,"type":61},"2026-06-08",{"date":63,"type":22},{"date":141,"type":22},"2030-09",{"name":143,"class":109},"Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)",1,{"id":146,"slug":147,"hasResults":11,"nctId":148,"briefTitle":149,"officialTitle":149,"acronym":4,"eligibilityCriteria":150,"healthyVolunteers":11,"sex":77,"minAge":78,"maxAge":4,"enrollmentInfo":151,"targetDuration":153,"studyType":154,"phases":4,"briefSummary":155,"conditions":156,"keywords":4,"overallStatus":99,"whyStopped":4,"lastUpdateSubmitDate":157,"lastUpdatePostDateStruct":158,"startDateStruct":160,"completionDateStruct":162,"leadSponsor":164,"locationsCount":144},"100629656","northwestern-neuromuscular-disease-biorepository-100629656","NCT07477509","Northwestern Neuromuscular Disease Biorepository","Inclusion Criteria:\n\n* Undergoing evaluation for or diagnosed with a neuromuscular disorder, including but not restricted to motor neuron diseases (MNDs), neuropathies, neuromuscular junction disorders, and myopathies as a patient under the care of a provider in the Northwestern Neuromuscular Division\n* Provision of signed and dated informed consent form personally or via legally authorized representative (LAR) \\[SOP: HRP-013\\] in accordance with Good Clinical Practice (GCP), International Conference on Harmonization (ICH), and local regulations.\n\nExclusion Criteria:\n\n* History of a bleeding disorder or current treatment with anticoagulants (e.g., Coumadin, heparin) in participating study participants that would preclude collection of CSF or skin biopsies for these sub-studies.\n* History of keloid formation for study participants who will undergo skin biopsy.",{"count":152,"type":22},2500,"5 Years","OBSERVATIONAL","The primary objective of this research is to collect clinical data and tissue samples from adult patients with neuromuscular disease cared for at the Northwestern Memorial Hospital neuromuscular clinic (Lavin Building, 19th floor).\n\nThis study has the following aims:\n\nAim 1: To consent a large cohort of adult study participants with neuromuscular disorders, including but not limited to motor neuron diseases, neuropathies, neuromuscular junction disorders, and myopathies for participation in the biorepository and to collect longitudinal data on their clinical disease phenotypes.\n\nAim 2: To obtain and store biological samples from biorepository study participants, including whole blood, plasma, serum, peripheral blood mononuclear cells \\[PBMCs\\], skin biopsies, and cerebrospinal fluid.\n\nAim 3: To develop a data-sharing process to provide de-identified biorepository participant clinical data and samples to partnered investigators to expedite discovery in neuromuscular disease diagnosis and treatment.",[33],"2026-05-21",{"date":159,"type":61},"2026-05-26",{"date":161,"type":61},"2026-05-01",{"date":163,"type":22},"2050-03",{"name":165,"class":109},"Northwestern University",{"id":167,"slug":168,"hasResults":11,"nctId":169,"briefTitle":170,"officialTitle":171,"acronym":172,"eligibilityCriteria":173,"healthyVolunteers":11,"sex":17,"minAge":174,"maxAge":78,"enrollmentInfo":175,"targetDuration":4,"studyType":23,"phases":177,"briefSummary":178,"conditions":179,"keywords":189,"overallStatus":99,"whyStopped":4,"lastUpdateSubmitDate":212,"lastUpdatePostDateStruct":213,"startDateStruct":215,"completionDateStruct":216,"leadSponsor":218,"locationsCount":144},"100638348","phase-3-efficacy-safety-and-tolerability-of-zeleciment-rostudirsen-dyne-251-administered-intravenously-every-4-weeks-in-ambulatory-participants-with-duchenne-muscular-dystrophy-forzetto-100638348","NCT07608432","Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)","A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping","FORZETTO","Inclusion Criteria:\n\n* Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping .\n* Rise From Floor (RFF) time must be \\\u003C 10 seconds for both screening assessments .\n* Receiving a stable daily or weekend dosage of glucocorticoids for at least 24 weeks prior to randomization with the expectation of maintaining a stable dose during the Placebo-Controlled Period of the study (unless dose adjustment is required by weight change)\n\nExclusion Criteria:\n\n* Receipt of ongoing immunosuppressive therapy (other than glucocorticoids) within 12 weeks prior to randomization\n* Use of any pharmacologic treatment (other than glucocorticoids) that may have an effect on muscle strength or function within 12 weeks prior to randomization\n* Any change in prophylaxis\u002Ftreatment for congestive heart failure (CHF) within 12 weeks prior to randomization\n* Receipt of eteplirsen within 1 week prior to randomization\n* Receipt of alternative exon-skipping or dystrophin-modifying therapy or zeleciment rostudirsen within 24 weeks prior to randomization\n* Receipt of givinostat within 12 weeks prior to randomization\n* Receipt of gene therapy at any time\n\nNote: Other inclusion or exclusion criteria may apply","4 Years",{"count":176,"type":22},90,[25],"The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping.",[180,181,182,41,28,183,184,185,29,186,187,188,33],"Duchenne Muscular Dystrophy (DMD)","Muscular Dystrophy, Duchenne","Muscular Dystrophy (DMD)","Muscular Dystrophy in Children","Muscular Dystrophy, Duchenne Type","Muscular Dystrophy, Duchenne and Becker Types","Genetic Disease, Inborn","Genetic Disease, X-Linked","Congenital, Hereditary, and Neonatal Diseases and Abnormalities",[190,41,191,192,193,194,195,196,197,198,172,199,200,201,202,203,204,205,206,207,208,209,210,211],"Ambulatory","Duchenne Muscular Dystrophy","Duchenne","Dyne","Dyne Therapeutics","DYNE-251","Dystrophy","Exon Skipping","Exon 51","Pediatric","PMO","Muscle Function","Muscular Dystropy, Duchenne","Rise From Floor","RFF","RFF Velocity","Rostudirsen","Time to rise","TTR","TTR Velocity","Zeleciment rostudirsen","Z-rostudirsen","2026-05-20",{"date":214,"type":61},"2026-05-27",{"date":63,"type":22},{"date":217,"type":22},"2032-10",{"name":194,"class":68},{"id":220,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":221,"targetDuration":4,"studyType":23,"phases":222,"briefSummary":26,"conditions":223,"keywords":224,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":225,"lastUpdatePostDateStruct":226,"startDateStruct":228,"completionDateStruct":229,"leadSponsor":230,"locationsCount":4},"100638788",{"count":21,"type":22},[25],[28,29,30,31,32,33,34,35,36,37,38,39,40,41],[43,44,45,46,47,48,49,50,15,51,52,53,54,55,56,41],"2026-05-08",{"date":227,"type":61},"2026-05-14",{"date":63,"type":22},{"date":65,"type":22},{"name":67,"class":68},{"id":232,"slug":233,"hasResults":11,"nctId":234,"briefTitle":235,"officialTitle":236,"acronym":237,"eligibilityCriteria":238,"healthyVolunteers":11,"sex":77,"minAge":4,"maxAge":4,"enrollmentInfo":239,"targetDuration":4,"studyType":154,"phases":4,"briefSummary":241,"conditions":242,"keywords":4,"overallStatus":99,"whyStopped":4,"lastUpdateSubmitDate":249,"lastUpdatePostDateStruct":250,"startDateStruct":252,"completionDateStruct":254,"leadSponsor":256,"locationsCount":144},"100628864","invasive-home-ventilation-in-denmark-100628864","NCT07467187","Invasive Home Ventilation in Denmark","The Evolution of Invasive Home Mechanical Ventilation in Denmark","HOMEVENT DK","Inclusion Criteria:\n\n* Patients are included if they have or have had a respiratory certified personale care assistent during the period 2016-2025.\n\nExclusion Criteria:\n\n* Not tracheostomized\n* Tracheostomized but on spontaneous breathing throughout 1 January 2016 - 31 December 2025\n* Not discharged to home, assisted living, nursing home, or rehabilitation during 1 January 2016 - 31 December 2025",{"count":240,"type":22},450,"The aim of this study is to describe national trends over the past 10 years in patients receiving invasive home mechanical ventilation (HMV) in Denmark. This includes indications for invasive HMV, diagnostic groups, and one-year mortality.",[33,243,244,180,245,246,247,248],"ALS (Amyotrophic Lateral Sclerosis)","Spinal Cord Injuries (SCI)","SMA - Spinal Muscular Atrophy","MSA - Multiple System Atrophy","Tracheostomized Patients","Tracheostomy","2026-04-15",{"date":251,"type":61},"2026-04-20",{"date":253,"type":61},"2026-04-13",{"date":255,"type":22},"2028-09-01",{"name":257,"class":109},"Rigshospitalet, Denmark",{"id":259,"slug":260,"hasResults":11,"nctId":261,"briefTitle":262,"officialTitle":263,"acronym":4,"eligibilityCriteria":264,"healthyVolunteers":11,"sex":77,"minAge":78,"maxAge":265,"enrollmentInfo":266,"targetDuration":4,"studyType":23,"phases":268,"briefSummary":269,"conditions":270,"keywords":288,"overallStatus":99,"whyStopped":4,"lastUpdateSubmitDate":253,"lastUpdatePostDateStruct":304,"startDateStruct":306,"completionDateStruct":308,"leadSponsor":310,"locationsCount":312},"100567749","phase-3-a-phase-3-study-of-ntla-2001-in-attrv-pn-100567749","NCT06672237","A Phase 3 Study of NTLA-2001 in ATTRv-PN","MAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)","Inclusion Criteria:\n\n* Diagnosis of ATTRv-PN\n* Karnofsky Performance Status (KPS) ≥ 60\n\nExclusion Criteria:\n\n* Other causes of amyloidosis (amyloidosis caused by non-TTR protein)\n* Other known causes of sensorimotor or autonomic neuropathy\n* Diabetes mellitus\n* New York Heart Association Class III or IV heart failure\n* Liver failure\n* Hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection\n* Prior receipt of a TTR silencer (Small interfering RNA (siRNA) or Antisense oligonucleotides (ASOs))\n* Estimated Glomerular Filtration Rate \\\u003C 30 mL\u002Fmin\u002F1.73 m2\n* Unable or unwilling to take vitamin A supplementation for the duration of the study\n* History of liver disease","85 Years",{"count":267,"type":22},60,[25],"This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.",[271,33,272,273,274,275,276,277,34,186,278,279,280,281,282,283,284,285,286,287],"Neuromuscular Disease","Neurodegenerative Disease","Neurodegenerative Disease, Hereditary","Neurodegenerative Diseases","Neuromuscular Diseases","Nerve Disorders","Nervous System Disease","Amyloidosis, Familial","Amyloidosis, Hereditary","Amyloidosis","Polyneuropathies","Amyloid Neuropathies","Amyloid Neuropathies, Familial","Peripheral Nervous System Disease","Peripheral Nervous System Diseases","Metabolism, Inborn Errors","Metabolic Diseases",[208,280,289,290,291,292,293,294,295,296,297,298,299,300,301,302,303],"Polyneuropathy","NTLA-2001","ATTR","ATTR-PN","ATTRv-PN","Transthyretin","TTR-mediated amyloidosis","Amyloidosis, hereditary","Amyloidosis, hereditary, transthyretin-related amyloidosis","Transthretin amyloid polyneuropathy","TTR PN","TTR polyneuropathy","nexiguran ziclumeran","nex-z","CRISPR",{"date":305,"type":61},"2026-04-16",{"date":307,"type":61},"2024-11-22",{"date":309,"type":22},"2028-08",{"name":311,"class":68},"Intellia Therapeutics",14,{"id":314,"slug":315,"hasResults":11,"nctId":316,"briefTitle":317,"officialTitle":318,"acronym":4,"eligibilityCriteria":319,"healthyVolunteers":11,"sex":77,"minAge":78,"maxAge":4,"enrollmentInfo":320,"targetDuration":4,"studyType":23,"phases":322,"briefSummary":323,"conditions":324,"keywords":341,"overallStatus":99,"whyStopped":4,"lastUpdateSubmitDate":345,"lastUpdatePostDateStruct":346,"startDateStruct":348,"completionDateStruct":350,"leadSponsor":352,"locationsCount":144},"100629707","effects-of-whole-body-electrical-muscle-stimulation-exercise-on-adults-with-neuromuscular-disease-100629707","NCT07478172","Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease","Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults withNeuromuscular Disease","Inclusion Criteria:\n\n* Age 18 or older\n* Diagnosed with one or more of the following neuromuscular conditions: Amyotrophic lateral sclerosis, primary lateral sclerosis, progressive muscle atrophy, spinal muscular atrophy, postpolio syndrome, inclusion body myositis, pompedisease, fascioscapulohumeral muscular dystrophy, charcot marie tooth disease, chronic inflammatory demyelinating polyneuropathy, hereditary spastic paraplegia, myasthenia gravis, lambert-eaton myasthenic syndrome, postural orthostatic tachycardia syndrome, mitochondrial myopathy, nemaline myopathy, centronuclear myopathy, lumbar radiculopathy, non-specific low back pain.\n* Ability to stand for approximately 15 minutes continuously with or without an assistive device (i.e. the length of time to stand to take a shower, complete meal preparation, wait in line at the bank, etc.)\n* At least some anti-gravity strength in major muscle groups as assessed by manual muscle testing (i.e. 2+\u002F5 strength or better)\n* Medical clearance to participate in an exercise program\n* Ability to provide informed consent\n* Ability to conform to the requirements of the study (i.e. attendance at assessment and intervention visits, maintain current level of non-study physical activity for the duration of the study, no intention to relocate mid-study)\n\nExclusion Criteria:\n\n* Diagnosed with one of the following neuromuscular conditions: Becker's muscular dystrophy, Duchenne muscular dystrophy, limb-girdle muscular dystrophy, myotonic dystrophy type 1 or 2, Freidrich's ataxia, any other NMD with known or suspected cardiac involvement or muscle fiber structural integrity defects.\n* Concurrent participation in another interventional research study\n* Unable to tolerate 15 minutes of continuous standing with or without an assistive device\n* Presence of a pacemaker, metal implants, or other implanted medical devices that could impact participant safety during WB-EMS intervention\n* Presence of cochlear implant, cortical stimulator, deep brain stimulator, ventriculoperitoneal shunt, recent skull defect, seizure in the past 12 months while taking anti-epilepsy medication, or previous serious adverse event with TMS, which could impact participant safety during TMS testing\n* Presence of unstable acute or chronic disease (i.e. renal failure, rheumatologic disease, cardia arrhythmia, neoplasm, uncontrolled hypertension)\n* Known pregnancy at time of screening; verbal screening will occur throughout the study.\n* Presence of a terminal disease (i.e. receiving hospice services)\n* Current or previous use of any drugs known to influence muscle mass or performance within 6 months; these may include but are not limited to anabolic steroids, IGF01, growth hormone, replacement androgen therapy, anti-androgen therapy\n* Presence of an additional neurologic conditions affecting somatosensory or motor function\u002Fcontrol (i.e. Parkinson's disease, Multiple Sclerosis, h\u002Fo stroke, TBI, SCI, ataxia, apraxia, hemiplegia, etc.)\n* Musculoskeletal condition or surgery in the past year that would confound results of exercise interventions (i.e. TKA, THA, RTC repair, spinal fusion)\n* Other medical conditions, signs, or symptoms that would interfere with study conductor interpretation of results as determined by an investigator",{"count":321,"type":22},50,[83],"This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adults with neuromuscular disease (NMD). Due to motor unit impairments, NMD patients often cannot tolerate traditional exercise. WB-EMS bypasses voluntary activation limits by directly stimulating muscle contractions. Up to 50 adults with conditions like ALS, SMA, and MG will undergo 20-minute supervised WB-EMS sessions (1-2 times weekly for 4-8 weeks) using the Katalyst system. Outcomes include neural excitability (TMS), motor unit behavior (EMG, NCS), functional tests (walk, balance, strength), and patient-reported fatigue, pain, and quality of life. Strict safety monitoring and exclusion criteria are in place. This study will provide preliminary data on WB-EMS as a potential exercise modality for NMD.",[33,325,326,327,328,329,86,330,331,332,333,334,335,336,337,338,339,340],"Amyotrophic Lateral Sclerosis","Myasthenia Gravis","Lambert-eaton Myasthenic Syndrome","Primary Lateral Sclerosis","Spinal Muscular Atrophy","Fascioscapulohumeral Muscular Dystrophy","Inclusion Body Myositis","Mitochondrial Myopathy","Nemaline Myopathy","Centronuclear Myopathy","Postpolio Syndrome","Pompe Disease (Late-onset)","Chronic Inflammatory Demyelinating Polyneuropathy","Hereditary Spastic Paraplegia","Postural Orthostatic Tachycardia Syndrome (POTS)","Progressive Muscular Atrophy",[271,342,343,344],"Electrical Stimulation","Whole Body stimulation","Exercise intervention","2026-03-12",{"date":347,"type":61},"2026-03-17",{"date":349,"type":61},"2026-03-10",{"date":351,"type":22},"2031-01-07",{"name":353,"class":109},"University of Missouri-Columbia",{"id":355,"slug":356,"hasResults":11,"nctId":357,"briefTitle":358,"officialTitle":358,"acronym":359,"eligibilityCriteria":360,"healthyVolunteers":11,"sex":77,"minAge":361,"maxAge":362,"enrollmentInfo":363,"targetDuration":153,"studyType":154,"phases":4,"briefSummary":365,"conditions":366,"keywords":371,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":378,"lastUpdatePostDateStruct":379,"startDateStruct":381,"completionDateStruct":383,"leadSponsor":385,"locationsCount":4},"100626161","walking-function-outcomes-following-surgical-correction-with-rehabilitation-versus-physical-therapy-alone-in-charcot-marie-tooth-disease-a-bidirectional-cohort-study-100626161","NCT07432035","Walking Function Outcomes Following Surgical Correction With Rehabilitation Versus Physical Therapy Alone in Charcot-Marie-Tooth Disease: A Bidirectional Cohort Study","CMT-WALK","Inclusion Criteria:\n\n* Participants aged 12 years or older at the time of enrollment\n* Genetically or clinically confirmed CMT, based on established diagnostic criteria\n* Presence of foot deformity and\u002For gait impairment attributable to CMT, as determined by a treating clinician\n* Ability to ambulate at least 10 meters, with or without assistive devices\n* Medically eligible for either functional surgical intervention or structured physical therapy, as determined by the treating team\n* Willingness and ability to participate in longitudinal follow-up assessments\n* Provision of written informed consent\n\nExclusion Criteria:\n\n* Previous major foot or ankle surgery that substantially altered lower-limb biomechanics\n* Presence of non-CMT-related neurological disorders affecting gait (eg, stroke, Parkinson disease, multiple sclerosis)\n* Severe musculoskeletal conditions unrelated to CMT that limit walking ability (eg, advanced hip or knee osteoarthritis)\n* Active lower-limb infection, ulceration, or acute injury at the time of enrollment\n* Severe cognitive impairment or psychiatric condition precluding reliable participation\n* Medical contraindications to surgery or exercise-based rehabilitation, when relevant\n* Inability to complete baseline functional assessments or anticipated inability to complete follow-up","12 Years","60 Years",{"count":364,"type":22},200,"The goal of this study is to compare changes in walking ability in people with Charcot-Marie-Tooth disease (CMT) who receive two different treatment approaches for foot deformities that affect walking.\n\nCMT is an inherited nerve condition that can cause muscle weakness, loss of sensation, and foot deformities. These changes often make walking difficult and can reduce independence and quality of life. Treatment options commonly include physical therapy alone or surgery to correct foot alignment followed by rehabilitation. However, it is not clear whether one approach leads to better long-term walking outcomes.\n\nThe main question this study aims to answer is whether individuals who undergo functional foot surgery followed by rehabilitation experience different changes in walking ability over time compared with those who receive structured physical therapy alone.\n\nResearchers will compare walking performance between these two treatment groups over a period of up to two years. Walking ability will be evaluated using standardized walking tests and patient questionnaires.\n\nParticipants included in this study are individuals with CMT-related foot deformities that affect walking and who received either surgery followed by rehabilitation or physical therapy alone. Researchers will analyze changes in walking ability over time and determine how many participants achieve meaningful improvement.\n\nThe findings from this study may help clinicians and individuals with CMT better understand how different treatment strategies influence walking function over time.",[86,367,368,369,33,370],"Pes Cavovarus","Surgery","Exercise Training","Gait Disorders",[372,373,374,375,376,377],"Charcot Marie Tooth disease","Walking performance","Functional surgery","Physical therapy","Gait impairment","Propensity score matching","2026-02-24",{"date":380,"type":61},"2026-02-25",{"date":382,"type":22},"2026-02-11",{"date":384,"type":22},"2028-02-10",{"name":386,"class":109},"Peking University Third Hospital",{"id":388,"slug":389,"hasResults":11,"nctId":390,"briefTitle":391,"officialTitle":391,"acronym":392,"eligibilityCriteria":393,"healthyVolunteers":11,"sex":77,"minAge":394,"maxAge":395,"enrollmentInfo":396,"targetDuration":4,"studyType":23,"phases":398,"briefSummary":399,"conditions":400,"keywords":4,"overallStatus":99,"whyStopped":4,"lastUpdateSubmitDate":407,"lastUpdatePostDateStruct":408,"startDateStruct":410,"completionDateStruct":412,"leadSponsor":414,"locationsCount":144},"100594606","emmvies--walking-training-at-home-in-virtual-reality-for-children-with-chronic-illnesses-impacting-physical-health-100594606","NCT07021638","EMMVIES : Walking Training at Home in VIrtual Reality for Children With Chronic Illnesses Impacting Physical Health","EMMVIES","Inclusion Criteria:\n\n* Patient aged 6 to 17 years at date of inclusion;\n* Able to walk for 5 minutes on a treadmill with or without body weight support, with or without technical aids;\n* Be able to understand and play virtual reality games (immersive or non-immersive);\n* Be able to answer questionnaires;\n* Patient affiliated to or benefiting from a social security scheme;\n* Informed consent, dated and signed by parents or guardians (if minors), to participate in the study;\n* Included in a care program specific to their chronic condition for at least 6 months;\n* Meet the following criteria depending on the group:\n\nPC group:\n\n* Diagnosis of CP\n* Functional level classified II or III according to the Gross Motor Function Classification System (GMFCS)\n* Rehabilitation goal: improvement of gait and\u002For gross motor function\n\nOB group:\n\n* Overweight or obese, defined by a BMI above the IOTF-25 percentile\n* Aim to increase daily physical activity\n* No history of hip pathology\n\nGroup NM:\n\n* Diagnosis of one of the following neuromuscular pathologies: spinal muscular atrophy type 3, Duchenne disease, congenital myopathy or Charcot Marie Tooth.\n* Functional level of grade 2 to 6 on the Walton scale, or grade 3 to 7 on the Vignos scale\n* Rehabilitation goal: improvement of walking and\u002For gross motor skills\n\nExclusion Criteria:\n\n* Patients weighing over 100 kg (due to the technical constraints of using the AMY mat).\n* For children aged 6 to 11: in connection with the immersive virtual reality device: problems with stereoscopic vision (perception of images in 3 dimensions), unstabilized epilepsy, facial trauma \\\u003C 3 months, hearing or visual impairment, pain, dizziness or nausea caused by using immersive virtual reality.\n* Patients who have undergone surgery in the last 3 months;\n* Patients who have received intramuscular botulinum toxin injections in the lower limbs or intensive therapy in the 3 months prior to inclusion;\n* Patient with a non-routine therapeutic program planned within 5 months that could lead to confusion with the tested program;\n* Patient with insufficient understanding of the French language;\n* Patient opposition (child or adolescent);\n* Persons deprived of liberty by judicial or administrative decision;\n* Persons under compulsory psychiatric care;\n* Persons under legal protection;","6 Years","17 Years",{"count":397,"type":22},30,[83],"Over the past 30 years, the prevalence of congenital or acquired chronic diseases in children has risen, affecting between 10 and 30% of them, or at least 100,000 children in France. Pathologies such as cerebral palsy (CP), neuromuscular diseases (NMD), obesity or congenital heart disease impact physical health by causing musculoskeletal, respiratory or cardiovascular deficiencies. These limitations influence their ability to participate in daily activities, affecting their quality of life and that of their families.To minimize these impacts, motor rehabilitation programs focusing on physical activity are proposed, but their effectiveness requires prolonged practice. However, these specific programs, often delivered in remote specialized centers, are difficult to access. Home programs have been developed to overcome these constraints. They enable children, with the support of their parents, to carry out therapeutic activities at home. Although their feasibility has been demonstrated, their effectiveness is relative. A multitude of protocols and tools have been tested, with no harmonization of practices.To support the implementation of home-based programs for children with CP or obesity, virtual reality has already been used, mainly on the basis of commercial solutions. This solution is therefore feasible and has proved relatively effective.With this in mind, and based on the user experience of children, parents and professionals, the investigators have initially co-developed with the French company EzyGain a connected treadmill specifically adapted to pediatric needs and the requirements of home programs. The AMY treadmill is a compact treadmill with on-board sensors and a safety system, communicating with a tablet application and a virtual reality headset.Taking into account the opportunities offered by this new technology, as well as building on the effects and features already known from home programs, the investigators have developed a new modality for home programs focusing on walking for children with CP, MNM and obesity, the EMMVIES program. The crucial step now is to investigate the feasibility, tolerance and clinical effects of this EMMVIES program.",[401,402,403,404,33,405,406],"Home-based Programs","EzyGain","AMY Treadmill","Cerebral Palsy","Obesity","Virtual Reality","2025-12-26",{"date":409,"type":61},"2025-12-31",{"date":411,"type":61},"2025-12-24",{"date":413,"type":22},"2027-12-01",{"name":415,"class":416},"University Hospital, Angers","OTHER_GOV",{"id":418,"slug":419,"hasResults":11,"nctId":420,"briefTitle":421,"officialTitle":421,"acronym":4,"eligibilityCriteria":422,"healthyVolunteers":11,"sex":77,"minAge":423,"maxAge":4,"enrollmentInfo":424,"targetDuration":4,"studyType":23,"phases":425,"briefSummary":426,"conditions":427,"keywords":4,"overallStatus":99,"whyStopped":4,"lastUpdateSubmitDate":432,"lastUpdatePostDateStruct":433,"startDateStruct":435,"completionDateStruct":437,"leadSponsor":438,"locationsCount":144},"100544013","the-effect-of-a-muscle-mimicking-fabric-type-shoulder-orthosis-on-functional-movements-of-the-upper-limb-in-patients-with-neuromuscular-disorder-100544013","NCT06363357","The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder","Inclusion Criteria\n\n1. Patients with a confirmed diagnosis of a neuromuscular disease (NMD) by genetic testing, muscle biopsy, or electrodiagnostic studies, presenting with prominent upper limb muscle weakness. Examples include:\n\n   1. Muscular Dystrophies: Duchenne\u002FBecker Muscular Dystrophy (DMD\u002FBMD), Limb-Girdle Muscular Dystrophy (LGMD), Facioscapulohumeral Muscular Dystrophy (FSHD), etc.\n   2. Motor Neuron Diseases: Spinal Muscular Atrophy (SMA, Types 2 and 3), Amyotrophic Lateral Sclerosis (ALS, upper limb-dominant), etc.\n   3. Peripheral Neuropathies: Charcot-Marie-Tooth (CMT) disease, etc.\n   4. Other Neuromuscular Conditions: Including but not limited to cervical spinal cord injury.\n2. Aged over 10 years.\n3. A score of 2 to 5 on the Brooke Upper Extremity Functional Rating Scale.\n4. Manual Muscle Test (MMT) grade of less than 3 for shoulder abduction.\n5. Ability to provide written informed consent from the participant and\u002For their legal representative, indicating willingness to participate in the study.\n\nExclusion Criteria\n\n1. Unwillingness or inability to provide informed consent.\n2. A score of 1 or 6 on the Brooke Upper Extremity Functional Rating Scale.\n3. Cognitive impairment severe enough to interfere with the proper use of a shoulder orthosis.\n4. Any other condition which, in the opinion of the investigator, would make study participation inappropriate or unsafe for the patient.","10 Years",{"count":397,"type":22},[83],"The goal of this clinical trial is to investigate the effect of a muscle-mimicking, fabric-type shoulder orthosis on functional movements of the upper limb in patients with neuromuscular disorder.\n\nThe main questions it aims to answer are:\n\n* What is the impact of the muscle-mimicking, fabric-type shoulder orthosis on upper limb functional movements in patients with neuromuscular disorder?\n* Are there observable differences in upper limb function when the shoulder orthosis is worn versus when it is not?\n\nParticipants will:\n\n* Receive education on how to wear and use the shoulder orthosis.\n* Undergo evaluations, including assessment of upper limb performance, shoulder muscle strength testing, active range of motion measurements, assessment of functional workspace, goal attainment scale evaluation, surface electromyography, physiological measurements such as blood pressure and heart rate, fatigue assessment, and assessment for any musculoskeletal or skin-related issues.\n\nResearchers will compare neuromuscular disorder patients before and while wearing and operating the shoulder orthosis to see if there are any significant effects on variables such as upper limb function, range of motion, functional workspace, goal attainment scale, and surface electromyography.",[181,127,428,33,330,429,243,430,431],"Upper Extremity","Spinal Muscular Atrophy (SMA)","LGMD","SCI - Spinal Cord Injury","2025-11-25",{"date":434,"type":61},"2025-12-03",{"date":436,"type":61},"2024-04-20",{"date":409,"type":22},{"name":439,"class":109},"Seoul National University Hospital",{"id":441,"slug":442,"hasResults":11,"nctId":443,"briefTitle":444,"officialTitle":444,"acronym":445,"eligibilityCriteria":446,"healthyVolunteers":11,"sex":77,"minAge":4,"maxAge":4,"enrollmentInfo":447,"targetDuration":448,"studyType":154,"phases":4,"briefSummary":449,"conditions":450,"keywords":456,"overallStatus":99,"whyStopped":4,"lastUpdateSubmitDate":461,"lastUpdatePostDateStruct":462,"startDateStruct":464,"completionDateStruct":466,"leadSponsor":468,"locationsCount":470},"100605729","ultrasound-evaluation-of-hematoma-risk-after-needle-emg-in-patient-on-doac-therapy-100605729","NCT07166302","Ultrasound Evaluation of Hematoma Risk After Needle EMG in Patient on DOAC Therapy","U-HAND","Inclusion Criteria:\n\n* Subjects must understand the nature of the study and must provide signed and dated written informed consent prior to conducting any study-related procedures\n* Willing and able to comply with all protocol procedures\n* subjects confirmed the daily (and recent) intake od direct anticoagulants in strandard dosing.\n* no other antitrombotic drug therapy (e.g. acetylsalicyl acid, clopidogrel, ticagralol, low molecular weight heparin or second direct anticoagulant) is taken.\n\nExclusion Criteria:\n\n* Any clinically significant medical or psychiatric condition or medical history that, in the opinion of the investigator, would interfere with the subject's ability to participate in the study or increase the risk of participation for that subject\n* other antitrombotic drug therapy (e.g. acetylsalicyl acid, clopidogrel, ticagralol, low molecular weight heparin or second direct anticoagulant) is recently taken.",{"count":21,"type":22},"7 Days","Approximately 30 minutes after needle EMG, patients who are taking direct oral anticoagulants (DOACs) will undergo an ultrasound examination to evaluate for the presence of possible intramuscular hematomas at the muscles where the EMG needle was inserted. These hematomas are considered a potential adverse effect of needle EMG.\n\nThe aim of the study is to determine whether needle EMG can be considered a safe procedure in this group of patients, without posing a risk of intramuscular hematoma formation.",[451,452,453,454,455,281,33],"Direct Acting Anticoagulant Adverse Reaction","Needle Injury","EMG","Peripheral Neuropathies","Mononeuropathies",[457,458,459,460],"electromyography","hematoma","ultrasonography","anticoagulants","2025-09-03",{"date":463,"type":61},"2025-09-10",{"date":465,"type":22},"2025-09",{"date":467,"type":22},"2026-12-31",{"name":469,"class":109},"Masaryk University",2]