[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"fascioscapulohumeral-muscular-dystrophy\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:fascioscapulohumeral-muscular-dystrophy":32},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,69,116,143,171],{"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":14,"conditions":26,"keywords":44,"overallStatus":56,"whyStopped":4,"lastUpdateSubmitDate":57,"lastUpdatePostDateStruct":58,"startDateStruct":61,"completionDateStruct":63,"leadSponsor":65,"locationsCount":68},"100595880","phase-3-a-study-to-evaluate-del-brax-also-referred-to-as-aoc-1020-in-participants-with-fshd-100595880",false,"NCT07038200","A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD","A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)","FORTITUDE-3","Inclusion Criteria:\n\n* Clinical and genetic diagnosis of FSHD1 or FSHD2\n* Ability to walk independently at pre-specified walking speed (orthoses and ankle braces allowed) for at least 10 meters at screening\n* Adequate muscle strength based on QMT composite score\n\nExclusion Criteria:\n\n* Breastfeeding, pregnancy, or intent to become pregnant during the study\n* Unwilling or unable to comply with contraceptive requirements\n* Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study\n* Blood Pressure \\> 140\u002F90 mmHg at Screening\n* Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of the drug, whichever is longer\n* Treatment with an oligonucleotide within 9 months of Screening","ALL","16 Years","70 Years",{"count":21,"type":22},200,"ESTIMATED","INTERVENTIONAL",[25],"PHASE3",[27,28,29,30,31,32,33,34,35,36,37,38,39,40,41,42,43],"Facioscapulohumeral Muscular Dystrophy","FSHD","FSHD - Facioscapulohumeral Muscular Dystrophy","FSHD1","FSHD2","Fascioscapulohumeral Muscular Dystrophy","Fascioscapulohumeral Muscular Dystrophy Type 1","Fascioscapulohumeral Muscular Dystrophy Type 2","Facioscapulohumeral Muscular Dystrophy 1","Facioscapulohumeral Dystrophy","Facio-Scapulo-Humeral Dystrophy","Facioscapulohumeral Muscular Dystrophy 2","Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1)","FSH Muscular Dystrophy","Landouzy Dejerine Dystrophy","Landouzy-Dejerine Muscular Dystrophy","Landouzy-Dejerine Syndrome",[45,46,47,48,49,50,51,52,15,53,54,55],"Avidity","Avidity Biosciences","del-brax","del brax","delbrax","AOC1020","AOC 1020","delpacibart braxlosiran","FORTITUDE Phase 3","FORTITUDE","FORTITUDE 3","RECRUITING","2026-05-27",{"date":59,"type":60},"2026-05-29","ACTUAL",{"date":62,"type":60},"2025-06-10",{"date":64,"type":22},"2028-07",{"name":66,"class":67},"Avidity Biosciences, Inc.","INDUSTRY",46,{"id":70,"slug":71,"hasResults":11,"nctId":72,"briefTitle":73,"officialTitle":74,"acronym":4,"eligibilityCriteria":75,"healthyVolunteers":11,"sex":17,"minAge":76,"maxAge":4,"enrollmentInfo":77,"targetDuration":4,"studyType":23,"phases":79,"briefSummary":81,"conditions":82,"keywords":100,"overallStatus":56,"whyStopped":4,"lastUpdateSubmitDate":105,"lastUpdatePostDateStruct":106,"startDateStruct":108,"completionDateStruct":110,"leadSponsor":112,"locationsCount":115},"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","18 Years",{"count":78,"type":22},50,[80],"NA","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.",[83,84,85,86,87,88,89,32,90,91,92,93,94,95,96,97,98,99],"Neuromuscular Diseases (NMD)","Amyotrophic Lateral Sclerosis","Myasthenia Gravis","Lambert-eaton Myasthenic Syndrome","Primary Lateral Sclerosis","Spinal Muscular Atrophy","Charcot Marie Tooth Disease (CMT)","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",[101,102,103,104],"Neuromuscular Disease","Electrical Stimulation","Whole Body stimulation","Exercise intervention","2026-03-12",{"date":107,"type":60},"2026-03-17",{"date":109,"type":60},"2026-03-10",{"date":111,"type":22},"2031-01-07",{"name":113,"class":114},"University of Missouri-Columbia","OTHER",1,{"id":117,"slug":118,"hasResults":11,"nctId":119,"briefTitle":120,"officialTitle":120,"acronym":28,"eligibilityCriteria":121,"healthyVolunteers":11,"sex":17,"minAge":122,"maxAge":123,"enrollmentInfo":124,"targetDuration":4,"studyType":23,"phases":126,"briefSummary":127,"conditions":128,"keywords":129,"overallStatus":56,"whyStopped":4,"lastUpdateSubmitDate":133,"lastUpdatePostDateStruct":134,"startDateStruct":136,"completionDateStruct":138,"leadSponsor":140,"locationsCount":142},"100576448","dietary-protein-requirements-in-adults-with-facioscapulohumeral-muscular-dystrophy-100576448","NCT06785428","Dietary Protein Requirements in Adults With Facioscapulohumeral Muscular Dystrophy","Inclusion Criteria:\n\n* Adult female or male participants who are 26 to 60 years of age at screening (inclusive)\n* Genetically confirmed with FSHD\n* Ambulatory\n* Has maintained stable use of medication and supplements, stable dietary and lifestyle habits, and stable body weight, for the last 3 months prior to screening and agree to maintain them throughout the study\n* Willing and able to agree to the requirements and restrictions of this study, be willing to give voluntary consent, be able to understand and read the questionnaires, and carry out all study-related procedures\n\nExclusion Criteria:\n\n* Individuals who are lactating or pregnant\n* Usage of corticosteroids within 3 months of study entry or had ever taken steroids for a duration exceeding 1 year\n* On androgens or growth hormone within 6 months before screening and for duration of study; topical physiologic androgen replacement is permitted\n* On sympathomimetic agents, antidepressants, or β-receptor blockers\n* Have cardiovascular disease\n* Evidence of an alternative diagnosis other than FSHD or a coexisting myopathy or dystrophy\n* Current\u002Factive malignancy (e.g., remission less than 5 years' duration), with the exception of fully excised or treated basal cell carcinoma, cervical carcinoma in-situ, or ≤ 2 squamous cell carcinomas of the skin\n* Type 1 or type 2 diabetes mellitus\n* History of sensitivity to protein pharmaceuticals\n* Known active substance abuse, including alcohol\n* Renal impairment (serum creatinine ≥ 2 times the upper limit of normal,(ULN))\n* History of severe restrictive or obstructive lung disease, or evidence for interstitial lung disease on screening chest radiograph\n* Major surgery within 4 weeks prior to metabolic trial 1\n* Any other active or unstable medical\u002Fpsychological conditions or use of medications\u002Fsupplements\u002Ftherapies that, in the opinion of the investigator, may adversely affect the participant's ability to complete the study or its measures or pose a significant risk to the participant.","26 Years","60 Years",{"count":125,"type":22},10,[80],"Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common types of muscular dystrophy, affecting about 4 out of 100,000 individuals. The disease is characterized by progressive muscle loss (i.e., muscle atrophy) commonly affecting the face, shoulders, and upper arm muscles. The muscle loss ultimately results in reduced strength and impaired physical performance. At present there is no cure for FSHD, therefore, physicians have focused on therapeutic interventions to help alleviate these symptoms.\n\nDaily consumption of adequate amounts of dietary protein is essential to support muscle mass maintenance and overall health and function across the lifespan. However, previous research has reported inadequate protein intake in individuals with FSHD. The characteristic of progressive muscle loss in individuals with FSHD and other muscular dystrophies is ultimately due to an imbalance in the rate of muscle building (i.e., muscle protein synthesis) and muscle breakdown (i.e., muscle protein breakdown), where individuals with FSHD have been shown to have reduced rates of muscle building. As inadequate protein intake is known to result in a loss of muscle mass, strength and function, this loss may be amplified in individuals with FSHD.\n\nDietary recommendations traditionally have been determined through nitrogen balance techniques, where the current recommended dietary allowance (RDA) for daily protein intake for adults is 0.8 g\u002Fkg\u002Fd. However, recent research indicates how the nitrogen balance technique potentially underestimates protein requirements. Therefore, there is a need to reassess current dietary recommendations in adults with FSHD in order to help support the maintenance of muscle strength and function.\n\nRecent efforts to understand protein requirements in various populations have been completed using the indicator amino acid oxidation technique (IAAO). This non-invasive method is reported to provide a robust measure of protein requirements. Due to its non-invasive nature, the IAAO method allows researchers to use this technique in individuals with FSHD, where there is currently limited work in studying this population.\n\nThe purpose of this study is to measure the protein requirements in individuals with FSHD using the non-invasive IAAO technique.",[32],[130,131,132],"Protein requirements","Facioscapulohumeral muscular dystrophy","Indicator amino acid oxidation technique","2026-02-05",{"date":135,"type":60},"2026-02-06",{"date":137,"type":60},"2025-01-19",{"date":139,"type":22},"2026-03",{"name":141,"class":114},"McGill University",2,{"id":144,"slug":145,"hasResults":11,"nctId":146,"briefTitle":147,"officialTitle":147,"acronym":4,"eligibilityCriteria":148,"healthyVolunteers":11,"sex":17,"minAge":149,"maxAge":4,"enrollmentInfo":150,"targetDuration":4,"studyType":23,"phases":152,"briefSummary":153,"conditions":154,"keywords":4,"overallStatus":56,"whyStopped":4,"lastUpdateSubmitDate":162,"lastUpdatePostDateStruct":163,"startDateStruct":165,"completionDateStruct":167,"leadSponsor":169,"locationsCount":115},"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":151,"type":22},30,[80],"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.",[155,156,157,83,32,158,159,160,161],"Muscular Dystrophy, Duchenne","Orthotic Devices","Upper Extremity","Spinal Muscular Atrophy (SMA)","ALS (Amyotrophic Lateral Sclerosis)","LGMD","SCI - Spinal Cord Injury","2025-11-25",{"date":164,"type":60},"2025-12-03",{"date":166,"type":60},"2024-04-20",{"date":168,"type":22},"2025-12-31",{"name":170,"class":114},"Seoul National University Hospital",{"id":172,"slug":173,"hasResults":11,"nctId":174,"briefTitle":175,"officialTitle":176,"acronym":4,"eligibilityCriteria":177,"healthyVolunteers":178,"sex":17,"minAge":179,"maxAge":180,"enrollmentInfo":181,"targetDuration":4,"studyType":23,"phases":183,"briefSummary":184,"conditions":185,"keywords":4,"overallStatus":56,"whyStopped":4,"lastUpdateSubmitDate":190,"lastUpdatePostDateStruct":191,"startDateStruct":193,"completionDateStruct":195,"leadSponsor":196,"locationsCount":198},"100514716","assessments-in-patients-with-muscular-pathology-and-in-control-subjects--the-actilige-next-study-100514716","NCT05982119","Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study","Gait Analysis Parameter, Stair Climbing and Upper Limb Evaluation in Patients With Muscular Pathology and in Control Subjects: The ActiLiège Next Study","Inclusion criteria\n\n* For the patients:\n\n  * Genetically confirmed diagnosis of DMD, FSHD, DM1, CMT or FKRP mutations or confirmed CNM based on muscle biopsy.\n  * FSHD, DM1, CMT and CNM patients should be ambulant or in transition.\n  * DM1 and CMT patients should present sensori-motor signs on physical examination.\n  * Under the age of 20 years for patients with DMD, CNM or between the ages of 5 and 80 years for patients with FSHD, CMT and DM1.\n  * More than 2 years old for patients with FKRP mutations\n  * Non-ambulant DMD patients must be able to remain seated in an arm- or a wheelchair for at least one hour.\n  * Patients with DMD treated with corticosteroids for at least 6 months or initiated corticosteroid at V0 (except for patients under 4).\n  * Signed informed consent form by patient himself or, in case of minor patients, signed informed consent form by patient's parents or legal guardians.\n* For the control subjects:\n\n  * Ambulant boys and girls under 20 years old\n  * Signed informed consent form by patient him\u002Fherself or, in case of minor patients, signed informed consent form by patient's parents or legal guardians.\n\nExclusion Criteria:\n\n* For the patients:\n\n  * Patients with extreme cognitive disorders that limit their understanding of the exercises to be performed.\n  * Patients who have undergone a surgical procedure or who have experienced recent trauma (within fewer than 6 months) affecting the upper or lower limbs (for ambulant patients).\n  * A concomitant chronic or acute neurological, endocrine, infectious, allergic, or inflammatory pathology within the 3-week period immediately prior to inclusion.\n  * Patients who are participating in an interventional clinical trial.\n  * DMD patients in transition who are not on corticosteroids.\n* For the control subjects:\n\n  * Patients who have undergone a surgical procedure or who have experienced recent trauma (within fewer than 6 months) affecting the upper or lower limbs.\n  * Elite athletes (at the national level).\n  * A chronic or acute muscular, neurological, infectious, or inflammatory pathology within the 3-week period immediately prior to inclusion.\n  * An orthopedic, neuromuscular, or neurological pathology that affects the quality of the subject's walking gait.",true,"1 Year","80 Years",{"count":182,"type":22},300,[80],"The objective of the ActiLiège Next study is to collect longitudinal data from patients and control subjects using a wearable magneto-inertial device. By collecting natural history data in various neuromuscular disorders (Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy 1, Charcot-Marie-Tooth, Centronuclear Myopathy, Congenital Muscular Dystrophy), we aim to validate digital outcome measures to continuously assess motor function in real-life.",[186,32,187,188,93,189],"Duchenne Muscular Dystrophy","Myotonic Dystrophy 1","Charcot-Marie-Tooth","Congenital Muscular Dystrophy","2025-05-15",{"date":192,"type":60},"2025-05-20",{"date":194,"type":60},"2020-07-10",{"date":139,"type":22},{"name":197,"class":114},"Centre Hospitalier Universitaire de Liege",8]