[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"dmd\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:dmd":41},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,10,0,[8,69,103,156,187,199,226,261,286,308],{"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":17,"minAge":18,"maxAge":77,"enrollmentInfo":78,"targetDuration":4,"studyType":23,"phases":80,"briefSummary":82,"conditions":83,"keywords":87,"overallStatus":92,"whyStopped":4,"lastUpdateSubmitDate":93,"lastUpdatePostDateStruct":94,"startDateStruct":96,"completionDateStruct":98,"leadSponsor":100,"locationsCount":102},"100615022","phase-2-phase-2-study-of-sat-3247-in-pediatric-ambulatory-patients-100615022","NCT07287189","Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients","A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Dose Comparison and Exploratory Efficacy Study of Orally Administered SAT-3247 in Ambulatory DMD Patients","BASECAMP","Key Inclusion Criteria:\n\n* Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing with a confirmed mutation in the DMD gene.\n* Male DMD patients who are ambulatory and aged ≥ 7 to \\\u003C 10 years at the time of screening.\n* Stable dose of systemic glucocorticoids (i.e., prednisolone, deflazacort, or vamorolone) according to the standard of care for ≥ 3 months prior to the Screening Visit and for the duration of the trial. Patients who are not receiving glucocorticosteroids are also eligible if stopped ≥ 3 months prior to the Screening Visit.\n* Stable doses of prescription medicines including ACE inhibitors, β-blockers, and diuretics (excluding glucocorticosteroids) and over-the-counter medicines and\u002For herbal supplements for supportive care ≥ 1 month prior to the Screening Visit and for the duration of the trial.\n* Participants that have previously received delandistrogene moxeparvovec (brand name Elevidys) either in a prior clinical trial or in the commercial setting \\> 18 months prior to screening whose muscle function tests have stabilized or demonstrated decline ≥ 3 months prior to Screening, as determined by investigator and documented in chart notes, will be eligible.\n* Participants that have previously received an exon skipper \\> 6 months prior to Screening whose muscle function tests have stabilized or demonstrated decline ≥ 3 months prior to Screening, as determined by investigator and documented in chart notes, will be eligible.\n* Participants receiving a stable dose of givinostat (brand name Duvyzat) for at least 18 months or longer prior to the Screening Visit will be eligible. Participants unable to tolerate givinostat who discontinued treatment before 18 months are eligible to enroll if date of last dose is ≥ 30 days from the Screening date. Givinostat should not be discontinued, if tolerated, to meet study entry criteria.\n* Participants that have received prior treatment with an investigational gene therapy product (other than delandistrogene moxeparvovec) ≥ 24 months prior to the Screening Visit.\n* If participating in a physical therapy\u002Fstrength training regimen, must be stable for ≥ 2 months prior to the Screening Visit and for the duration of the trial.\n\nKey Exclusion Criteria:\n\n* Ambulatory patients expected to experience loss of ambulation within ≤ 12 months.\n* Participants for whom MRI or open muscle biopsy are contraindicated.\n* Evidence of significant hepatic dysfunction, defined as GLDH \\> 2X upper limit of normal (ULN) at the Screening Visit.\n* Impaired cardiac function defined as a left ventricular ejection fraction of \\\u003C 50% on screening cardiac assessments (echocardiogram or MRI) or evidence of symptomatic cardiomyopathy.\n* A forced vital capacity \\\u003C 60% predicted at the Screening Visit.\n* Ongoing participation in any other therapeutic clinical trial or follow-up study for a therapeutic intervention\n* Consumption of grapefruit juice or grapefruit containing products\n* Severe behavioural or cognitive problems that preclude participation in the study, in the opinion of the investigator.\n\nAdditional entry criteria will be reviewed with the clinical site investigator.","9 Years",{"count":79,"type":22},51,[81],"PHASE2","Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and \\\u003C 10 years. The trial will study two doses of SAT-3247 in a randomized, double-blind, placebo-controlled weekday regimen for 12 weeks to determine the optimal dose, safety, tolerability, and preliminary efficacy.",[84,85,41,86,28],"Duchenne Muscular Dystrophy","Duchenne","Neuromuscular Diseases",[88,89,90,91],"muscle regeneration","satellite cell","asymmetric division","dystrophin","RECRUITING","2026-06-12",{"date":95,"type":61},"2026-06-16",{"date":97,"type":61},"2025-12-08",{"date":99,"type":22},"2027-06-30",{"name":101,"class":68},"Satellos Bioscience, Inc.",21,{"id":104,"slug":105,"hasResults":11,"nctId":106,"briefTitle":107,"officialTitle":108,"acronym":109,"eligibilityCriteria":110,"healthyVolunteers":11,"sex":17,"minAge":111,"maxAge":112,"enrollmentInfo":113,"targetDuration":4,"studyType":23,"phases":115,"briefSummary":116,"conditions":117,"keywords":127,"overallStatus":92,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":149,"startDateStruct":151,"completionDateStruct":152,"leadSponsor":154,"locationsCount":155},"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","18 Years",{"count":114,"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.",[118,119,120,41,28,121,122,123,29,124,125,126,33],"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",[128,41,84,85,129,130,131,132,133,134,109,135,136,137,138,139,140,141,142,143,144,145,146,147],"Ambulatory","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":150,"type":61},"2026-05-27",{"date":63,"type":22},{"date":153,"type":22},"2032-10",{"name":130,"class":68},1,{"id":157,"slug":158,"hasResults":11,"nctId":159,"briefTitle":160,"officialTitle":161,"acronym":4,"eligibilityCriteria":162,"healthyVolunteers":11,"sex":17,"minAge":163,"maxAge":4,"enrollmentInfo":164,"targetDuration":4,"studyType":23,"phases":166,"briefSummary":167,"conditions":168,"keywords":173,"overallStatus":92,"whyStopped":4,"lastUpdateSubmitDate":177,"lastUpdatePostDateStruct":178,"startDateStruct":180,"completionDateStruct":182,"leadSponsor":184,"locationsCount":155},"100538429","phase-2-vasodilator-and-exercise-study-for-dmd-vaso-rex-100538429","NCT06290713","Vasodilator and Exercise Study for DMD (VASO-REx)","Vasodilators and Exercise as Adjuvant Therapy for Duchenne Muscular Dystrophy (VASO-REx Study)","Inclusion Criteria:\n\n* Diagnosis of DMD confirmed by genetic report\n* Minimum entry age of 6.0 years old\n* Ambulatory\n* On stable glucocorticoid regimen (for \\> 3 months)\n\nExclusion Criteria:\n\n* Contraindication to a Magnetic resonance Imaging examination (e.g. severe claustrophobia, magnetic implants, unable\u002Funwilling to perform test)\n* Presence of unstable medical problems, including severe cardiomyopathy, left ventricular ejection fraction \\\u003C45%, cardiac conduction abnormalities as evidenced on ECG, uncontrolled seizure disorder, uncontrolled hypo or hypertension\n* Presence of a secondary condition that impacts muscle function or muscle metabolism (e.g., myasthenia gravis, endocrine disorder, mitochondrial disease)\n* Presence of a secondary condition leading to developmental delay or impaired motor control (e.g., cerebral palsy) or previous history of unprovoked rhabdomyolysis\n* Contraindications to phosphodiesterase 5 inhibitors (use of nitrates, alpha-adrenergic blockers, other phosphodiesterase 5 inhibitors) or other medications known to modulate blood flow or muscle metabolism\n* Participation in currently approved FDA trials or other investigational clinical trials during the period of the study","6 Years",{"count":165,"type":22},50,[81],"Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal muscle and improve cardiovascular health) and tadalafil, an FDA-approved vasodilator (to optimize blood flow and muscle perfusion which is impaired and often overlooked in DMD). Target: improved muscle function, vascular health, and DMD treatment.",[84,169,170,121,171,172,41],"Duchenne Disease","Muscular Dystrophy","Vasodilation","Exercise",[41,174,175,176],"Tadalafil","Drug and Exercise Intervention","Treatment Strategy","2026-05-12",{"date":179,"type":61},"2026-05-15",{"date":181,"type":61},"2024-06-05",{"date":183,"type":22},"2026-11",{"name":185,"class":186},"University of Florida","OTHER",{"id":188,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":189,"targetDuration":4,"studyType":23,"phases":190,"briefSummary":26,"conditions":191,"keywords":192,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":193,"lastUpdatePostDateStruct":194,"startDateStruct":196,"completionDateStruct":197,"leadSponsor":198,"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":195,"type":61},"2026-05-14",{"date":63,"type":22},{"date":65,"type":22},{"name":67,"class":68},{"id":200,"slug":201,"hasResults":11,"nctId":202,"briefTitle":203,"officialTitle":204,"acronym":4,"eligibilityCriteria":205,"healthyVolunteers":11,"sex":17,"minAge":111,"maxAge":206,"enrollmentInfo":207,"targetDuration":4,"studyType":23,"phases":209,"briefSummary":210,"conditions":211,"keywords":213,"overallStatus":92,"whyStopped":4,"lastUpdateSubmitDate":216,"lastUpdatePostDateStruct":217,"startDateStruct":219,"completionDateStruct":221,"leadSponsor":223,"locationsCount":225},"100515784","phase-2-ns-089ncnp-02-201-in-boys-with-duchenne-muscular-dystrophy-dmd-100515784","NCT05996003","NS-089\u002FNCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)","A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089\u002FNCNP-02 in Boys With Duchenne Muscular Dystrophy (DMD)","Inclusion Criteria:\n\n* Male ≥ 4 years and \\\u003C15 years of age\n* Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame\n* Able to walk independently without assistive devices\n* Ability to complete the TTSTAND without assistance in \\\u003C20 seconds\n* Stable dose of glucocorticoid for at least 3 months and the dose is expected to remain on a stable dose for the duration of the study.\n* Other inclusion criteria may apply.\n\nExclusion Criteria:\n\n* Has a body weight of \\\u003C20 kg at the time of informed consent (applies to participants screening for Part 1 only)\n* Evidence of symptomatic cardiomyopathy\n* Current or previous treatment with anabolic steroids (e.g., oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug\n* Current or previous treatment with any other investigational drug within 3 months prior to the first dose of study drug or within 5 times the half-life of a medication, whichever is longer\n* Surgery within the 3 months prior to the first dose of study drug or planned during the study duration\n* Previously treated in an interventional study of NS-089\u002FNCNP-02\n* Having received exon skipping oligonucleotide within 1 year prior to the first dose of IP\n* Other exclusion criteria may apply.","14 Years",{"count":208,"type":22},20,[81],"This is a Phase 2, open-label, multi-center, 2-part study of NS-089\u002FNCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to \\\u003C15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089\u002FNCNP-02 administered once weekly.\n\nThe study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.",[84,212,41],"Exon 44",[214,41,215],"Exon 44 Skipping","Brogidirsen","2026-03-04",{"date":218,"type":61},"2026-03-06",{"date":220,"type":61},"2024-02-22",{"date":222,"type":22},"2026-09-11",{"name":224,"class":68},"NS Pharma, Inc.",25,{"id":227,"slug":228,"hasResults":11,"nctId":229,"briefTitle":230,"officialTitle":231,"acronym":232,"eligibilityCriteria":233,"healthyVolunteers":11,"sex":234,"minAge":235,"maxAge":4,"enrollmentInfo":236,"targetDuration":238,"studyType":239,"phases":4,"briefSummary":240,"conditions":241,"keywords":246,"overallStatus":92,"whyStopped":4,"lastUpdateSubmitDate":251,"lastUpdatePostDateStruct":252,"startDateStruct":254,"completionDateStruct":256,"leadSponsor":258,"locationsCount":260},"100447167","swiss-registry-for-neuromuscular-disorders-100447167","NCT05102916","Swiss Registry for Neuromuscular Disorders","Swiss Registry for Neuromuscular Disorders (Swiss-Reg-NMD)","Swiss-Reg-NMD","Inclusion Criteria:\n\n* Children, adolescents and adults diagnosed with a NMD\n* Who are living or treated for a NMD in Switzerland, and\n* Who gave informed consent\n\nExclusion Criteria:\n\n* None if diagnosis is confirmed, whenever possible, by genetic testing, or at least by biopsy and\u002For electroneuromyography, according to international standards for the diagnosis of the given NMD.","ALL","0 Years",{"count":237,"type":22},2000,"80 Years","OBSERVATIONAL","The Swiss Patient Registry for DMD\u002FBMD and SMA was launched in 2008 in order to give Swiss patients access to new therapies. It was founded with the financial support of several patient organizations and research foundations. Since 2008, children, adolescents and adults with DMD, BMD and SMA are registered with the help of all major muscle centers in Switzerland. After nearly ten years of activity, the Swiss Patient Registry for DMD\u002FBMD and SMA implemented several adaptations in 2018 to meet current and future expectations of patient's organizations, health authorities and research organizations.",[242,41,243,244,245],"SMA","BMD","IMD","Congenital Muscular Dystrophy",[41,243,244,242,247,248,249,250],"LAMA2","COL-6","CMD","NMD","2026-01-13",{"date":253,"type":61},"2026-01-15",{"date":255,"type":61},"2018-06-20",{"date":257,"type":22},"2071-01-01",{"name":259,"class":186},"University of Bern",19,{"id":262,"slug":263,"hasResults":11,"nctId":264,"briefTitle":265,"officialTitle":266,"acronym":4,"eligibilityCriteria":267,"healthyVolunteers":11,"sex":17,"minAge":111,"maxAge":77,"enrollmentInfo":268,"targetDuration":4,"studyType":23,"phases":270,"briefSummary":272,"conditions":273,"keywords":274,"overallStatus":92,"whyStopped":4,"lastUpdateSubmitDate":277,"lastUpdatePostDateStruct":278,"startDateStruct":280,"completionDateStruct":282,"leadSponsor":284,"locationsCount":155},"100597451","phase-1-a-clinical-study-to-evaluate-the-safety-tolerability-and-efficacy-of-bbm-d101-in-the-treatment-of-duchenne-muscular-dystrophy-100597451","NCT07058662","A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.","A Phase 1\u002F2, Open-label Clinical Study to Evaluate the Safety, Tolerability and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.","Inclusion Criteria:\n\n1. The Participants and\u002For his legal guardian must fully understand the purpose, nature, methods, and potential risks of the study, and sign a written informed consent form.\n2. Ambulatory male subjects aged 4 years and above but under 9 years (4 years ≤ age \\\u003C 9 years).\n3. Any mutation in the DMD gene confirmed by genetic testing\n4. Serum creatine kinase (CK) during the screening period meets the study requirements.\n5. Receiving stable, standard-dose glucocorticoids before screening.\n6. The subject's AAV capsid antibodies meet the clinical trial requirements.\n7. Able to cooperate with motor function assessment, MRI, and muscle biopsy as required by the study.\n8. Laboratory test results during the screening period and at baseline meet the standards.\n9. The subject and\u002For his legal guardian must fully understand the study procedures, be willing to actively cooperate, commit to high compliance with the protocol, and ensure that the subject attends all scheduled visits.\n\nExclusion Criteria:\n\n1. Positive for hepatitis B surface antigen (HBsAg), hepatitis B virus deoxyribonucleic acid (HBV-DNA) ≥ 1000 U\u002FmL, hepatitis C virus ribonucleic acid (HCV-RNA) positive, human immunodeficiency virus (HIV) positive, or positive for Treponema pallidum antibodies.\n2. Currently receiving antiviral therapy for hepatitis B, hepatitis C, HIV, etc.\n3. The investigator deems the subject has severe behavioral or cognitive disorders that may hinder participation in this study.\n4. Poorly controlled asthma, or Duchenne Muscular Dystrophy (DMD) leading to significant decline in lung function, or recurrent infectious pneumonia that the investigator considers may affect respiratory function.\n5. Left ventricular ejection fraction (LVEF) \\\u003C 50% or New York Heart Association (NYHA) cardiac function class ≥ III.\n6. Severe or persistent arrhythmias (such as atrial fibrillation, frequent ventricular premature beats, ventricular bigeminy, ventricular trigeminy, severe bundle branch block, etc.), and congenital heart disease that is evaluated by the investigator as unsuitable for participation in this study.\n7. Any changes in preventive\u002Fcardiomyopathy treatment (initiation of treatment, drug changes, dosing regimen changes, treatment interruption, termination, or restart) within 1 month before the infusion of the study drug.\n8. History of liver diseases such as portal hypertension, splenomegaly, hepatic encephalopathy, liver fibrosis ≥ stage 3, or hepatic nodules\u002Fcysts found by ultrasound during screening, or elevated alpha-fetoprotein with clinical significance as determined by the investigator.\n9. Severe infection (such as pneumonia, pyelonephritis, or meningitis) within 4 weeks before the treatment visit (enrollment may be postponed).\n10. History of gene therapy or cell therapy (such as stem cell transplantation).\n11. History of or current presence of autoimmune diseases, severe renal, gastrointestinal, neurological, or coagulation disorders, malignant tumors, or other diseases.\n12. Other diseases that the investigator deems unsuitable for participation in this study.",{"count":269,"type":22},9,[271,81],"PHASE1","The purpose of the study is to evaluate the safety, tolerability, and efficacy of BBM-D101 to treat participants with Duchenne Muscular Dystrophy.",[41],[41,275,276],"gene therapy","Adeno-Associated Virus","2025-11-28",{"date":279,"type":61},"2025-12-04",{"date":281,"type":61},"2025-07-31",{"date":283,"type":22},"2031-06-30",{"name":285,"class":68},"Belief BioMed (Beijing) Co., Ltd",{"id":287,"slug":288,"hasResults":11,"nctId":289,"briefTitle":290,"officialTitle":291,"acronym":4,"eligibilityCriteria":292,"healthyVolunteers":11,"sex":234,"minAge":293,"maxAge":294,"enrollmentInfo":295,"targetDuration":4,"studyType":239,"phases":4,"briefSummary":297,"conditions":298,"keywords":4,"overallStatus":92,"whyStopped":4,"lastUpdateSubmitDate":299,"lastUpdatePostDateStruct":300,"startDateStruct":302,"completionDateStruct":304,"leadSponsor":306,"locationsCount":155},"100594145","ml-based-multi-sensor-fall-risk-screening-in-dmd-100594145","NCT07015632","ML-Based Multi-Sensor Fall Risk Screening in DMD","Quantitative Analysis of Upper Extremity Functional Movement of the Upper Extremity in Patients With Duchenne Muscular Dystrophy","Inclusion Criteria:\n\n1. Individuals with a confirmed genetic diagnosis of Duchenne Muscular Dystrophy (DMD)\n2. Age over 10 and under 30 years\n3. Brooke Scale score between 2 and 5\n4. Manual muscle test grade below 3 for shoulder abduction muscles\n\nExclusion Criteria:\n\n1. Individuals who are unable or unwilling to provide informed consent\n2. Brooke Scale score of 1 or 6\n3. Individuals with cognitive impairments that significantly limit their ability to participate in assessments","10 Years","30 Years",{"count":296,"type":22},30,"This prospective observational study aims to analyze changes in upper extremity functional movement over time in children with Duchenne Muscular Dystrophy (DMD). Thirty patients will be evaluated at three time points (baseline, 6 months, 12 months) using clinical assessments (PUL 2.0, Brooke Scale, grip strength), computer vision-based video analysis, and machine learning algorithms. The goal is to improve future upper limb evaluation methods for non-ambulatory DMD patients. The study includes safety monitoring and adheres to ethical standards, ensuring patient data confidentiality and providing compensation if adverse effects occur.",[41],"2025-06-10",{"date":301,"type":61},"2025-06-13",{"date":303,"type":61},"2024-07-18",{"date":305,"type":22},"2026-12-01",{"name":307,"class":186},"Seoul National University Hospital",{"id":309,"slug":310,"hasResults":11,"nctId":311,"briefTitle":312,"officialTitle":312,"acronym":313,"eligibilityCriteria":314,"healthyVolunteers":315,"sex":17,"minAge":18,"maxAge":316,"enrollmentInfo":317,"targetDuration":4,"studyType":239,"phases":4,"briefSummary":319,"conditions":320,"keywords":321,"overallStatus":92,"whyStopped":4,"lastUpdateSubmitDate":323,"lastUpdatePostDateStruct":324,"startDateStruct":326,"completionDateStruct":328,"leadSponsor":330,"locationsCount":155},"100560802","defining-outcome-measures-for-behavioural-and-emotional-problems-in-dystrophinopathies-100560802","NCT06581887","Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies","D-BRAIN","Inclusion Criteria:\n\n* DMD patients:\n\n  1. Male\n  2. Age range 7-17 years\n  3. A genetically proven diagnosis of DMD.\n  4. A genetic mutation that abrogates expression of Dp427 alone (assigned in DMD Group 1: Dp427-\u002FDp140+) or both Dp427 and Dp140 (assigned to DMD Group 2: Dp427-\u002FDp140-).\n  5. Ability to consent\u002Fassent\n\nBMD patients:\n\n1. Male\n2. Age range 7-17 years\n3. A genetically proven diagnosis of BMD.\n4. A genetic mutation that decreases expression of Dp427 alone (assigned to BMD Group 1), of both Dp427 and Dp140 (assigned to BMD Group 2).\n5. Ability to consent\u002Fassent\n\nControl participants:\n\n1. Male\n2. Age range 7-17 years.\n3. Ability to consent\u002Fassent\n\nExclusion Criteria:\n\n* DMD \\& BMD patients:\n\n  1. Significant visual or hearing impairment\n  2. Specific phobias or sensory sensitivities to stimuli similar to the ones used in this study\n  3. Current participation in a clinical trial investigating a new drug involved in dystrophin modulation.\n  4. Inability to consent (for parents\u002Fguardians or self-reporting participants aged 16 and 17) or assent. This will exclude the rare individuals with extremely severe learning disability, as the assent in these patients is impossible (or the consent in self-reporting participants aged 16 and 17).\n\nControl participants:\n\n1. Significant visual or hearing impairment\n2. Specific phobias or sensory sensitivities to stimuli similar to the ones used in this study\n3. Any diagnosis of neurological or psychiatric condition\n\nGeneral exclusion criteria for MRI:\n\n1. Claustrophobia\n2. Pacemakers and defibrillators\n3. Nerve stimulators\n4. Intracranial clips\n5. Intraorbital or intraocular metallic fragments\n6. Cochlear implants\n7. Ferromagnetic implants (e.g. thoracic implant for scoliosis)\n8. Inability to lie supine during less than 45 minutes\n9. Not having a general practitioner\n10. Severe learning disability which will require a general anaesthetic",true,"17 Years",{"count":318,"type":22},100,"Study aims to develop and to evaluate the neurophysiological and physiological response to a classical conditioning task.To better understand how Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) impacts mental health and how to assess it. Participants invited to complete questionnaires about behaviour, cognitive function and social interactions, complete computer tasks and have an optional MRI brain scan,",[41,243],[322],"Antisense oligonucleotide","2024-08-29",{"date":325,"type":61},"2024-09-03",{"date":327,"type":61},"2022-09-13",{"date":329,"type":22},"2024-12-31",{"name":331,"class":186},"University College, London"]