[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"facioscapulohumeral-muscular-dystrophy\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:facioscapulohumeral-muscular-dystrophy":30},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,8,0,[8,55,108,136,158,188,212,243],{"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":32,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":43,"lastUpdatePostDateStruct":44,"startDateStruct":47,"completionDateStruct":49,"leadSponsor":51,"locationsCount":54},"100570532","personalized-training-for-people-with-rare-neuromuscular-disorders-100570532",false,"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":21,"type":22},120,"ESTIMATED","INTERVENTIONAL",[25],"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.",[28,29,30,31],"Neuromuscular Diseases (NMD)","Charcot Marie Tooth Disease (CMT)","Facioscapulohumeral Muscular Dystrophy","Myotonic Dystrophy Type 1 (DM1)",[33,34,35,36,37,38,39,40,41],"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":45,"type":46},"2026-06-18","ACTUAL",{"date":48,"type":46},"2024-12-13",{"date":50,"type":22},"2028-12",{"name":52,"class":53},"Oslo University Hospital","OTHER",5,{"id":56,"slug":57,"hasResults":11,"nctId":58,"briefTitle":59,"officialTitle":60,"acronym":61,"eligibilityCriteria":62,"healthyVolunteers":11,"sex":17,"minAge":63,"maxAge":19,"enrollmentInfo":64,"targetDuration":4,"studyType":23,"phases":66,"briefSummary":60,"conditions":68,"keywords":85,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":97,"lastUpdatePostDateStruct":98,"startDateStruct":100,"completionDateStruct":102,"leadSponsor":104,"locationsCount":107},"100595880","phase-3-a-study-to-evaluate-del-brax-also-referred-to-as-aoc-1020-in-participants-with-fshd-100595880","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","16 Years",{"count":65,"type":22},200,[67],"PHASE3",[30,69,70,71,72,73,74,75,76,77,78,79,80,81,82,83,84],"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",[86,87,88,89,90,91,92,93,61,94,95,96],"Avidity","Avidity Biosciences","del-brax","del brax","delbrax","AOC1020","AOC 1020","delpacibart braxlosiran","FORTITUDE Phase 3","FORTITUDE","FORTITUDE 3","2026-05-27",{"date":99,"type":46},"2026-05-29",{"date":101,"type":46},"2025-06-10",{"date":103,"type":22},"2028-07",{"name":105,"class":106},"Avidity Biosciences, Inc.","INDUSTRY",46,{"id":109,"slug":110,"hasResults":11,"nctId":111,"briefTitle":112,"officialTitle":113,"acronym":69,"eligibilityCriteria":114,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":115,"enrollmentInfo":116,"targetDuration":4,"studyType":23,"phases":118,"briefSummary":121,"conditions":122,"keywords":123,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":126,"lastUpdatePostDateStruct":127,"startDateStruct":129,"completionDateStruct":131,"leadSponsor":133,"locationsCount":135},"100585861","phase-1-a-first-in-human-study-of-epi-321-in-facioscapulohumeral-muscular-dystrophy-100585861","NCT06907875","A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy","A Phase 1\u002F2 Open-label Dose-escalation Study to Evaluate the Safety, Tolerability, and Biological Activity of EPI-321, an AAVrh74-delivered Epigenetic Editing Therapy in Adult FSHD Patients","Inclusion Criteria:\n\n* Able and willing to provide informed consent\n* Male or female 18 to 75 years of age\n* Clinical diagnosis of FSHD with genetic Type 1\n* FSHD Ricci clinical severity score 2 to 4 (on 5-point scale)\n* Has adequate liver function\n* Has adequate kidney function\n\nExclusion Criteria:\n\n* Has an anti-AAVrh74 total binding antibody titer \\> 1:400\n* Requires a walker or wheelchair for ambulation\n* Pregnant and\u002For breastfeeding at baseline or is planning to become pregnant during the first 12 months following EPI-321 administration\n* Has FSHD Type 2\n* Has a concurrent or past medical conditions could jeopardize the safety of the participant","75 Years",{"count":117,"type":22},12,[119,120],"PHASE1","PHASE2","The goal of this clinical trial is to learn how safe and tolerable EPI-321 is and whether there may be early signs it is working in male or female adult (18 to 75 years) participants with facioscapulohumeral muscular dystrophy (FSHD) Type 1 condition. The main questions it aims to answer are:\n\nHow safe is EPI-321 and how well can people handle it over time? How does EPI-321 interact with its target and does it show early signs of working?\n\nParticipants will receive a single dose of EPI-321 through a vein while being closely watched in a hospital and visit the clinic regularly for tests and checkups for about 5 years after getting EPI-321.",[30],[30,124,125],"EPI-321","Muscular Dystrophy","2026-04-20",{"date":128,"type":46},"2026-04-22",{"date":130,"type":46},"2025-05-08",{"date":132,"type":22},"2032-04-30",{"name":134,"class":106},"Epicrispr Biotechnologies, Inc.",7,{"id":137,"slug":138,"hasResults":11,"nctId":139,"briefTitle":140,"officialTitle":141,"acronym":4,"eligibilityCriteria":142,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":143,"enrollmentInfo":144,"targetDuration":4,"studyType":23,"phases":146,"briefSummary":147,"conditions":148,"keywords":4,"overallStatus":149,"whyStopped":4,"lastUpdateSubmitDate":150,"lastUpdatePostDateStruct":151,"startDateStruct":153,"completionDateStruct":155,"leadSponsor":156,"locationsCount":4},"100626399","phase-2-phase-2-study-evaluating-apitegromab-for-the-treatment-of-fshd-100626399","NCT07435129","Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD","A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter, 52-Week Study Evaluating the Efficacy and Safety of Apitegromab in Participants With Facioscapulohumeral Muscular Dystrophy (FORGE)","Inclusion Criteria:\n\n1. Male or female participants, 18 to 60 years of age at the time of informed consent.\n2. Genetic diagnosis of FSHD Type 1 or FSHD Type 2, confirmed with the appropriate documentation from an accredited laboratory\n3. Clinical severity score of 1.5 to 3.0 (Ricci score; range 0 to 5), inclusive, at screening\n4. Baseline 10-meter walk\u002Frun test time ≤5 seconds\n\nExclusion Criteria:\n\n1. Prior history of a hypersensitivity reaction to a mAb or recombinant protein bearing an Fc domain (eg, a soluble receptor-Fc fusion protein), apitegromab, or excipients of apitegromab\n2. Treatment with other investigational drugs in a clinical trial within 3 months or 5 half-lives, whichever is longer, before screening\n3. Previous treatment with apitegromab, or with other anti-myostatin therapies, including activin receptor antagonists\n4. Current or prior use of anabolic steroids, growth hormones, glucagon-like peptide-1 receptor agonist or other substances with known effects on muscle.\n5. Use of therapies with potentially significant muscle effects (eg, androgens, insulin-like growth factor, growth hormone, systemic beta-agonist, botulinum toxin, or muscle relaxants or muscle-enhancing supplements) or potentially significant neuromuscular effects (eg, acetylcholinesterase inhibitors) within 60 days before screening\n6. Use of systemic or corticosteroids within 60 days prior to screening. Inhaled or topical steroids are allowed.\n7. Pregnant or breastfeeding.\n8. Contraindications for MRI that may include, but are not limited to, certain implanted electronic devices, cochlear implants, metallic foreign bodies, vascular clips, and metallic implants; or claustrophobia, contrast agent allergies, inability to lie still, or external medical devices that may not be removed.\n\nHistory of alcoholism, or illicit drug use (drugs that are illegal and have not been prescribed).\n\nTaking medications that impede coagulation or platelet aggregation or has a history or active coagulopathy disorder.\n\nAny acute or comorbid condition interfering with the well-being of the participant within 7 days prior to screening, including active systemic infection, the need for acute treatment, or inpatient observation due to any reason","60 Years",{"count":145,"type":22},60,[120],"A randomized Phase 2 study to evaluate the efficacy and safety of apitegromab as a monotherapy in participant with FSHD",[30,69],"NOT_YET_RECRUITING","2026-02-24",{"date":152,"type":46},"2026-02-27",{"date":154,"type":22},"2026-08",{"date":50,"type":22},{"name":157,"class":106},"Scholar Rock, Inc.",{"id":159,"slug":160,"hasResults":11,"nctId":161,"briefTitle":162,"officialTitle":162,"acronym":4,"eligibilityCriteria":163,"healthyVolunteers":11,"sex":17,"minAge":164,"maxAge":4,"enrollmentInfo":165,"targetDuration":167,"studyType":168,"phases":4,"briefSummary":169,"conditions":170,"keywords":174,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":178,"lastUpdatePostDateStruct":179,"startDateStruct":181,"completionDateStruct":183,"leadSponsor":185,"locationsCount":187},"100624400","betterlife-fshd-a-patient-driven-health-and-research-platform-100624400","NCT07409142","BetterLife FSHD: A Patient-driven Health and Research Platform","Inclusion Criteria:\n\n* Individuals with a clinical or genetic diagnosis of FSHD, or individuals with a family history of FSHD who are showing symptoms\n* Age 1 year or older\n* Residing in the United States or its territories\n* If age 18 or older, individual must be able and willing to provide consent\n* If under age 18, individual must be able and willing to provide assent, when applicable, and have a parent or legal guardian register and provide consent\n\nExclusion Criteria:\n\n* Individuals residing outside the United States or its territories\n* Unable or unwilling to provide consent, or assent, when applicable","1 Year",{"count":166,"type":22},5000,"100 Years","OBSERVATIONAL","BetterLife FSHD is a registry platform built to support people living with FSHD. It connects patients with personalized resources, tools, and research opportunities that match their health experiences and needs. At the same time, BetterLife collects secure health and experience data from patients to support research. This data is shared with researchers to help better understand FSHD and work toward improved care, treatments, and outcomes for the community.\n\nLearn more and enroll at: www.BetterLifeFSHD.org",[171,81,70,71,69,72,30,76,79,172,173],"FSH","Facioscapulohumeral Muscular Dystrophy (FSHD)","Muscular Dystrophy, Facioscapulohumeral",[69,175,81,71,72,176,177],"Registry","Facioscapulohumeral dystrophy","facioscapulohumeral muscular dystrophy","2026-02-19",{"date":180,"type":46},"2026-02-20",{"date":182,"type":46},"2024-08-02",{"date":184,"type":22},"2060-01",{"name":186,"class":53},"FSHD Society",1,{"id":189,"slug":190,"hasResults":11,"nctId":191,"briefTitle":192,"officialTitle":192,"acronym":4,"eligibilityCriteria":193,"healthyVolunteers":194,"sex":17,"minAge":195,"maxAge":4,"enrollmentInfo":196,"targetDuration":4,"studyType":168,"phases":4,"briefSummary":198,"conditions":199,"keywords":4,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":203,"lastUpdatePostDateStruct":204,"startDateStruct":206,"completionDateStruct":208,"leadSponsor":210,"locationsCount":54},"100440770","biomarker-development-for-muscular-dystrophies-100440770","NCT05019625","Biomarker Development for Muscular Dystrophies","Inclusion Criteria:\n\n* Subjects with DM1 or DM2 based on genetic testing and\u002For clinical criteria (some subjects who have positive genetic testing may be asymptomatic, while other subjects who show characteristic clinical features may have declined to have genetic testing done). Control non-DM subjects are unknown to have DM or any other muscular dystrophy by history and may have had no genetic testing.\n* Able to provide informed consent or assent for participation in the study.\n* Demographic characteristics for single biofluid collection: Males and females age 5 years and older.\n* Demographic characteristics for serial biofluid and muscle function testing: Males and females age 14 years and older with DM1.\n* Demographic characteristics for biofluid and muscle biopsy: Males and females, ages 18-65 years.\n\nDemographic characteristics for single biofluid collection, ultrasound, and myography: Males and females age 14 years and older.\n\nExclusion Criteria:\n\n* Medical history of any of the following. State of immunosuppression; coagulopathy; pre-existing liver or kidney disease; documented HIV positive; documented hepatitis B and\u002For C positive.\n* Medications and other drugs. Use of anti-platelet drugs within 7 days prior to blood draw or biopsy; use of anticoagulants within 60 days prior to blood draw or biopsy; active drug or alcohol use or dependence that, in the opinion of the biopsy surgeon, would interfere with post-procedure wound care.\n* Other. Inability or unwillingness of the subject to give written informed consent.",true,"5 Years",{"count":197,"type":22},465,"Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. The investigators are interested in finding less invasive methods that reduce the need for muscle biopsies. The purpose of this research is to learn about the possibility of detecting and measuring the activity and severity of muscular dystrophies by examining a urine sample and a blood sample, and some muscles in the arms and legs using tests called ultrasound and electrical impedance myography; both tests are painless and non-invasive. The information that is gathered from this study may help to evaluate, prevent, diagnose, treat, and improve the understanding of human muscle diseases.",[200,201,202,30],"Myotonic Dystrophy","Duchenne Muscular Dystrophy","Becker Muscular Dystrophy","2025-11-19",{"date":205,"type":46},"2025-11-24",{"date":207,"type":46},"2015-02-20",{"date":209,"type":22},"2028-06",{"name":211,"class":53},"Massachusetts General Hospital",{"id":213,"slug":214,"hasResults":11,"nctId":215,"briefTitle":216,"officialTitle":217,"acronym":4,"eligibilityCriteria":218,"healthyVolunteers":194,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":219,"targetDuration":4,"studyType":168,"phases":4,"briefSummary":221,"conditions":222,"keywords":229,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":235,"lastUpdatePostDateStruct":236,"startDateStruct":238,"completionDateStruct":240,"leadSponsor":241,"locationsCount":187},"100063959","myotonic-dystrophy-and-facioscapulohumeral-muscular-dystrophy-registry-100063959","NCT00082108","Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry","National Registry of Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Patients and Family Members","Inclusion Criteria:\n\n* Diagnosed with DM, FSHD, or related diseases or are an unaffected family member of someone diagnosed with one of these diseases",{"count":220,"type":22},3000,"Myotonic dystrophy (DM) and facioscapulohumeral muscular dystrophy (FSHD) are inherited disorders characterized by progressive muscle weakness and loss of muscle tissue. The purpose of this registry is to connect people with DM or FSHD with researchers studying these diseases. The registry will offer individuals with DM and FSHD an opportunity to participate in research that focuses of their diseases. The registry will also help scientists to accomplish research on DM and FSHD and to distribute their findings to patients and care providers.",[200,30,125,223,224,225,226,227,228],"Myotonic Dystrophy Type 1","Myotonic Dystrophy Type 2","Congenital Myotonic Dystrophy","PROMM (Proximal Myotonic Myopathy)","Steinert's Disease","Myotonic Muscular Dystrophy",[175,69,230,125,231,200,232,233,171,234],"DM","Facioscapulohumeral Myotonic Dystrophy","DM1","DM2","MMD","2025-10-10",{"date":237,"type":46},"2025-10-15",{"date":239,"type":4},"2000-09",{"date":209,"type":22},{"name":242,"class":53},"University of Rochester",{"id":244,"slug":245,"hasResults":11,"nctId":246,"briefTitle":247,"officialTitle":248,"acronym":4,"eligibilityCriteria":249,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":250,"targetDuration":252,"studyType":168,"phases":4,"briefSummary":253,"conditions":254,"keywords":255,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":258,"lastUpdatePostDateStruct":259,"startDateStruct":261,"completionDateStruct":263,"leadSponsor":265,"locationsCount":187},"100362621","the-united-kingdom-facioscapulohumeral-muscular-dystrophy-patient-registry-100362621","NCT04001582","The United Kingdom Facioscapulohumeral Muscular Dystrophy Patient Registry","The UK Facioscapulohumeral Muscular Dystrophy Patient Registry","Inclusion Criteria:\n\n\\- All patients with a confirmed FSHD diagnosis (or pending diagnosis) who reside in the UK are eligible for inclusion.\n\nExclusion Criteria:\n\n* Any confirmed NMD other than FSHD\n* Living outside of the UK",{"count":251,"type":22},1018,"99 Years","Facioscapulohumeral Dystrophy (FSHD) is the third most common form of neuromuscular dystrophy worldwide with an estimated prevalence of one in 20,000. FSHD is an autosomal dominant genetic disease and is estimated to affect up to 3,000 people in the UK.\n\nThe patient registry facilitates a questionnaire based research study to better characterise and understand the disease in the UK, and helps to identify potential participants eligible for clinical trials.",[30],[69,30,256,257,125],"Facioscapulohumeral Muscular Dystrophy Type 1","Facioscapulohumeral Muscular Dystrophy Type 2","2024-05-07",{"date":260,"type":46},"2024-05-09",{"date":262,"type":46},"2013-05",{"date":264,"type":22},"2040-01",{"name":266,"class":53},"Newcastle University"]