[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"muscular-disease\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:muscular-disease":31},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,69,116,128],{"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":77,"sex":78,"minAge":79,"maxAge":80,"enrollmentInfo":81,"targetDuration":4,"studyType":23,"phases":83,"briefSummary":85,"conditions":86,"keywords":98,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":105,"lastUpdatePostDateStruct":106,"startDateStruct":108,"completionDateStruct":110,"leadSponsor":112,"locationsCount":115},"100600783","hyperthermia-in-patients-with-chronic-primary-pain---effects-on-thermoregulation-somatosensory-system-and-movement-evoked-pain-100600783","NCT07101978","Hyperthermia in Patients With Chronic Primary Pain - Effects on Thermoregulation, Somatosensory System and Movement Evoked Pain","Hyperthermia in Healthy and Patients With Chronic Primary Pain - Effects on Thermoregulation, Somatosensory System, Movement Evoked Pain (HYPPRI-1)","HYPPRI-1","Inclusion Criteria:\n\npatients:\n\n* Confirmed diagnosis of widespread pain (ICD-11 MG30.01)\n* Widespread Pain Index (WPI) ≥ 7 and Symptom Severity (SS) scale ≥ 5 or WPI) ≥ 3 and SS ≥ 9,\n* Pain \\>= 3 month and VAS \\>= 4,0\n* Body - infrared-A-Bulb Distance \\\u003C 38cm (overweight participants)\n* Signed declaration of consent\n\nhealthy:\n\n* No chronic illnesses\n* No acute infections\n* No regular medication: To avoid interactions\n* BMI ≤ 40kg\u002Fcm2\n* Mental health: No psychiatric diagnoses or psychotropic medication in your medical history\n\nExclusion Criteria:\n\n* Participation in other clinical studies\n* Contraindications for hyperthermia (severe cardiovascular diseases, tumour diseases, acute infections, pregnant and breastfeeding women)\n* Acute and \u002F or feverish microbial infections\n* Participants with severe somatic, rheumatic concomitant endocrine or neurological diseases, in particular neurological diseases associated with cognitive disorders, severe liver or kidney and cardiac diseases\n* participants who are permanently treated with opioids, cannabis, immunosuppressive drugs (e.g. corticoids, immunosuppressants) or alpha\u002Fbeta-A(nta)gonists due to a disease from the group described above\n* participants with pain due to a serious psychiatric illness (bipolar disorder, psychosis, personality disorder, severe depression, substance abuse) and serious systemic or neurological disorders\n* pregnancy or breastfeeding (for women)\n* Intake of medication within 6 weeks that inhibits the reuptake of the neurotransmitter serotonin or binds to receptors of this neurotransmitter group",true,"ALL","18 Years","70 Years",{"count":82,"type":22},60,[84],"NA","This study, in a quasi-experimental matched two-group pre-post design, investigates the effect of serial water-filtered whole-body hyperthermia on circadian core body temperature, the somatosensory system (nociception) and the movement evoked pain in healthy and patients with chronic primary pain (e.g., fibromyalgia). The intervention lasts 3 weeks with two treatment sessions per week.",[87,88,89,31,90,32,91,92,93,94,95,96,97],"Hyperthermia","Chronic Primary Pain","Widespread Pain","Rheumatic Diseases","Neuromuscular Disease","Fibromyalgia","Circadian Rhythm","Body Temperature Changes","Somatosensory Function","Quantitative Sensory Testing","Healthy",[99,100,101,102,103,104],"hyperthermia","widespread pain","chronic primary pain","circadian body temperature","quantitative sensory testing","healthy","2026-05-29",{"date":107,"type":61},"2026-06-02",{"date":109,"type":22},"2026-08-01",{"date":111,"type":22},"2028-12-31",{"name":113,"class":114},"Bern University of Applied Sciences","OTHER",1,{"id":117,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":118,"targetDuration":4,"studyType":23,"phases":119,"briefSummary":26,"conditions":120,"keywords":121,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":122,"lastUpdatePostDateStruct":123,"startDateStruct":125,"completionDateStruct":126,"leadSponsor":127,"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":124,"type":61},"2026-05-14",{"date":63,"type":22},{"date":65,"type":22},{"name":67,"class":68},{"id":129,"slug":130,"hasResults":11,"nctId":131,"briefTitle":132,"officialTitle":133,"acronym":4,"eligibilityCriteria":134,"healthyVolunteers":11,"sex":78,"minAge":135,"maxAge":136,"enrollmentInfo":137,"targetDuration":4,"studyType":139,"phases":4,"briefSummary":140,"conditions":141,"keywords":145,"overallStatus":147,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":149,"startDateStruct":151,"completionDateStruct":4,"leadSponsor":153,"locationsCount":156},"100057066","study-of-inherited-neurological-disorders-100057066","NCT00004568","Study of Inherited Neurological Disorders","Clinical and Molecular Manifestations of Inherited Neurological Disorders","* Participants include those with inherited neurological conditions based on the training and research needs of the Neurogenetics Branch program. There is no logical limit; however the total number of participants that can be enrolled in the protocol will be restricted. No more than 3,500 participants with either diagnosed or undiagnosed neurological conditions and their unaffected relatives will be enrolled in this evaluation and diagnostic protocol.\n\nINCLUSION CRITERIA:\n\nParticipants will be eligible if they:\n\n* Have either a known or suspected, inherited neurological disease, OR are an unaffected relative (first-, second-, third, or higher degree relative) of a participant with a genetic neurological disease.\n* Have the ability to understand and sign an informed consent or have a parent\u002Flegal guardian to do so if they are minor children or a legal guardian to provide consent for adults without consent capacity.\n* Aged 2 years and above.\n\nEXCLUSION CRITERIA:\n\nParticipants will not be eligible if they:\n\n-Have a systemic disease that compromises the ability to provide adequate neurologic examination or diagnosis.An example of this would be a contagious disease that would compromise our ability to do an adequate neurological exam.","2 Years","120 Years",{"count":138,"type":22},3500,"OBSERVATIONAL","This study is designed to learn more about the natural history of inherited neurological disorders and the role of heredity in their development. It will examine the genetics, symptoms, disease progression, treatment, and psychological and behavioral impact of diseases in the following categories: hereditary peripheral neuropathies; hereditary myopathies; muscular dystrophies; hereditary motor neuron disorders; mitochondrial myopathies; hereditary neurocognitive disorders; inherited neurological disorders without known diagnosis; and others. Many of these diseases, which affect the brain, spinal cord, muscles, and nerves, are rare and poorly understood.\n\nChildren and adults of all ages with various inherited neurological disorders may be eligible for this study. Participants will undergo a detailed medical and family history, and a family tree will be drawn. They will also have a physical and neurological examination that may include blood test and urine tests, an EEG (brain wave recordings), psychological tests, and speech and language and rehabilitation evaluations. A blood sample or skin biopsy may be taken for genetic testing. Depending on the individual patient s symptoms, imaging tests such as X-rays, CT or MRI scans and muscle and nerve testing may also be done.\n\nInformation from this study may provide a better understanding of the genetic underpinnings of these disorders, contributing to improved diagnosis, treatment, and genetic counseling, and perhaps leading to additional studies in these areas.",[142,31,143,144],"Motor Neuron Disease","Muscular Dystrophy","Peripheral Nervous System Disease",[146,143,142],"Myopathy","RECRUITING","2026-04-11",{"date":150,"type":61},"2026-04-14",{"date":152,"type":61},"2000-02-18",{"name":154,"class":155},"National Institute of Neurological Disorders and Stroke (NINDS)","NIH",2]