[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"lgmd\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:lgmd":33},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,56],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":37,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":44,"lastUpdatePostDateStruct":45,"startDateStruct":48,"completionDateStruct":50,"leadSponsor":52,"locationsCount":55},"100456966","phase-1-a-study-to-evaluate-the-safety-of-ab-1003-previously-lion-101-in-subjects-with-genetic-confirmation-of-lgmd2ir9-part1-100456966",false,"NCT05230459","A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I\u002FR9 (Part1)","A Two-part Multicenter Study: a Randomized, Double-blind, Placebo-controlled Dose-escalation Safety Phase (Part 1) Followed by Double-blind, Placebo-controlled, Adaptive Phase (Part 2) Study to Evaluate the Safety and Efficacy of AB-1003 in Adult Subjects With LGMD2I\u002FR9 Mutations in the Gene Encoding Fukutin Related Protein (FKRP)","Inclusion Criteria:\n\n1. Male and female subjects aged 18 and 65 years with clinical diagnosis of LGMD2I\u002FR9 and confirmation of FKRP gene mutation.\n2. Ability to walk\u002Frun 10 meters in \\\u003C30 seconds.\n3. Able to understand and comply with all study procedures.\n4. Sexually active females of childbearing potential and female and male partners of male subjects receiving study intervention must use a barrier method of contraception for the first 6 months after dosing.\n\nExclusion Criteria:\n\n1. Significant cardiomyopathy as defined by echocardiogram (left ventricular ejection fraction \\\u003C40%), evidence of conduction defect (increased PR and RR intervals, left bundle branch block and QTcF \\>480m\u002Fsec), NYHA Class 3 or 4 heart failure, or MRI gadolinium enhancement evidence of clinically important myocardial fibrosis.\n2. Contraindication to MRI or hypersensitivity to contrast dyes, shellfish or iodine.\n3. Implanted spinal rods, cardiac pacemaker or other implantation that would distort cardiac MRI images.\n4. History of active, ongoing chronic liver disease (e.g. hepatitis, HIV-related liver disease, hemochromatosis, steatosis, etc.) or abnormal liver function tests (abnormal GGT and\u002For abnormal total\u002Fdirect bilirubin \\>upper limit of normal \\[ULN\\] and\u002For elevated AST and ALT \\>2 ULN).\n5. Abnormal renal function (GFR \\\u003C60 ml\u002Fmin, using the Modification of Diet in Renal Disease equation).\n6. Any life-threatening disease, including malignant neoplasms and medical history or malignant neoplasms within the past 5 years prior to screening (except basal and squamous cell skin cancer).\n7. In the opinion of the investigator, a pre-existing medical condition that predisposes the subject to risks that outweighs the potential benefits.\n8. Requirement for daytime ventilatory support.\n9. Change in glucocorticosteroid treatment within 3 months prior to screening visit.\n10. Exposure to another investigational drug within 3 months prior to study treatment or any previous treatment with gene therapy.\n11. Ongoing participation in any other therapeutic clinical trial.\n12. Neutralizing antibody titer to AAV9 \\>1:5.\n13. Female subjects who are pregnant, plan to become pregnant in the next 12 months, or breastfeeding.","ALL","18 Years","65 Years",{"count":20,"type":21},10,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I\u002FR9 (LGMD2I\u002FR9). Participants will be treated in sequential, dose-level cohorts. (Part 1)",[28,29,30,31,32,33,34,35,36],"Limb Girdle Muscular Dystrophy","Limb-Girdle Muscular Dystrophy Type 2","LGMD2I","Muscular Dystrophy","LGMD2","LGMD","FKRP","FKRP Mutation","Fukutin Related Protein",[38,30,39,40,34,41,42],"gene therapy","LGMD2I\u002FR9","gene augmentation therapy","fukutin related protein","FKRP mutation","RECRUITING","2026-02-18",{"date":46,"type":47},"2026-02-20","ACTUAL",{"date":49,"type":47},"2023-05-15",{"date":51,"type":21},"2032-12",{"name":53,"class":54},"AskBio Inc","INDUSTRY",6,{"id":57,"slug":58,"hasResults":11,"nctId":59,"briefTitle":60,"officialTitle":60,"acronym":4,"eligibilityCriteria":61,"healthyVolunteers":11,"sex":16,"minAge":62,"maxAge":4,"enrollmentInfo":63,"targetDuration":4,"studyType":22,"phases":65,"briefSummary":67,"conditions":68,"keywords":4,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":77,"lastUpdatePostDateStruct":78,"startDateStruct":80,"completionDateStruct":82,"leadSponsor":84,"locationsCount":87},"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":64,"type":21},30,[66],"NA","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.",[69,70,71,72,73,74,75,33,76],"Muscular Dystrophy, Duchenne","Orthotic Devices","Upper Extremity","Neuromuscular Diseases (NMD)","Fascioscapulohumeral Muscular Dystrophy","Spinal Muscular Atrophy (SMA)","ALS (Amyotrophic Lateral Sclerosis)","SCI - Spinal Cord Injury","2025-11-25",{"date":79,"type":47},"2025-12-03",{"date":81,"type":47},"2024-04-20",{"date":83,"type":21},"2025-12-31",{"name":85,"class":86},"Seoul National University Hospital","OTHER",1]