[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"spinal-muscular-atrophy-type-3\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:spinal-muscular-atrophy-type-3":30},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,46,84,114],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":4,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":34,"lastUpdatePostDateStruct":35,"startDateStruct":38,"completionDateStruct":40,"leadSponsor":42,"locationsCount":45},"100596567","phase-2-a-study-to-evaluate-how-apitegromab-works-in-subjects-who-are-less-than-2-years-old-and-have-spinal-muscular-atrophy-100596567",false,"NCT07047144","A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy","A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects \u003C2 Years Old With Spinal Muscular Atrophy (SMA)","OPAL","Inclusion Criteria:\n\n1. Is \\\u003C2 years old at the time of the informed consent\n2. Had a gestational age of ≥35 weeks and gestational body weight ≥2.0 kg at birth\n3. Has confirmed diagnosis of 5q autosomal recessive SMA\n4. Has confirmed presence of SMN2 gene copy(ies)\n5. Must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved SMN2-targeted therapy (ie, nusinersen or risdiplam)\n6. Body weight for age is no less than 1st percentile based on the WHO Child Growth Standards at the Screening Visit\n7. Has delayed motor milestones for age attributed to SMA at the discretion of the Investigator or a CHOP-INTEND score \\\u003C55\n\nExclusion Criteria:\n\n1. Nutritional status that is not anticipated to be stable throughout the study or medical necessity for a gastric feeding tube, where most feeds are administered by this route\n2. Major orthopedic issues such as severe scoliosis or severe contractures or interventional procedure, including spine or hip surgery, which is considered to have the potential to substantially limit the ability of the subject to be evaluated on any motor function outcome measures, within 6 months before Screening or anticipated during the study\n3. Any other physical limitations (eg, the subject requires cast for contractures) that would prevent the subject from undergoing motor function outcome measures throughout the study.","ALL","2 Years",{"count":20,"type":21},52,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK\u002FPD, efficacy, safety, and tolerability of apitegromab in subjects \\\u003C2 years old with 5q autosomal recessive SMA who have delayed motor milestones for their age attributed to SMA at the discretion of the Investigator or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score \\\u003C55.",[27,28,29,30,31,32],"Spinal Muscular Atrophy","SMA","Spinal Muscular Atrophy Type 2","Spinal Muscular Atrophy Type 3","Neuromuscular Manifestations","Anti-myostatin","RECRUITING","2026-04-29",{"date":36,"type":37},"2026-05-01","ACTUAL",{"date":39,"type":37},"2025-09-15",{"date":41,"type":21},"2029-03",{"name":43,"class":44},"Scholar Rock, Inc.","INDUSTRY",25,{"id":47,"slug":48,"hasResults":11,"nctId":49,"briefTitle":50,"officialTitle":50,"acronym":4,"eligibilityCriteria":51,"healthyVolunteers":52,"sex":17,"minAge":53,"maxAge":4,"enrollmentInfo":54,"targetDuration":4,"studyType":56,"phases":4,"briefSummary":57,"conditions":58,"keywords":60,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":73,"lastUpdatePostDateStruct":74,"startDateStruct":76,"completionDateStruct":78,"leadSponsor":80,"locationsCount":83},"100580605","establishing-walking-related-digital-biomarkers-in-rare-childhood-onset-progressive-neuromuscular-disorders-100580605","NCT06839469","Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders","Inclusion Criteria:\n\n* Genetic confirmation of disease (DMD, SMA) or healthy control\n* Able to walk independently at least 25 meters\n* Ongoing corticosteroids therapy or initiation of corticosteroid therapy in the previous 3 months for DMD\n* Stable dose of FDA approved SMN up-regulator therapy or in an open-label extension phase of a study treatment for at least 6 months for SMA or gene replacement at enrollment for SMA or DMD participants.\n\nExclusion Criteria:\n\n* Use foot orthoses or assistive devices for community ambulation or a mobility device for community navigation\n* Use investigational medications intended for treatment of NMD within 30 days\n* Prior to study entry had an injury or surgery that would impact gait within the previous 3 months",true,"5 Years",{"count":55,"type":21},106,"OBSERVATIONAL","The purpose of this research is (1) to identify disease specific walking-related digital biomarkers of disease severity, and (2) monitor longitudinal changes in natural environments, for extended periods of time, in DMD and SMA.",[30,59],"Duchenne Muscular Dystrophy (DMD)",[28,61,62,63,64,65,66,67,68,69,70,71,72],"spinal muscular atrophy","duchenne muscular dystrophy","DMD","instrumented insole","neuromuscular disease","machine learning model","gait analysis","functional biomarker","wearable","six minute walk test","ambulatory","becker phenotype","2026-04-06",{"date":75,"type":37},"2026-04-09",{"date":77,"type":37},"2024-05-01",{"date":79,"type":21},"2028-04-01",{"name":81,"class":82},"Columbia University","OTHER",3,{"id":85,"slug":86,"hasResults":11,"nctId":87,"briefTitle":88,"officialTitle":89,"acronym":4,"eligibilityCriteria":90,"healthyVolunteers":11,"sex":17,"minAge":91,"maxAge":92,"enrollmentInfo":93,"targetDuration":4,"studyType":56,"phases":4,"briefSummary":95,"conditions":96,"keywords":98,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":104,"lastUpdatePostDateStruct":105,"startDateStruct":107,"completionDateStruct":109,"leadSponsor":111,"locationsCount":113},"100623712","gait-and-bone-health-in-sma-100623712","NCT07400198","Gait and Bone Health in SMA","Identification of Gait-related Digital Biomarkers of Bone Health in Spinal Muscular Atrophy","Inclusion Criteria:\n\n* Confirmed diagnosis of 5qSMA\n* Between 8 and 50 years of age\n* Able to walk 10 meters without support or an assistive device\n* All participants who meet the inclusion criteria regardless of treatment status will be included, including those on adjuvant therapies, whether investigational or approved\n\nExclusion Criteria:\n\n* Injury or surgery within previous 3 months that would impact ability to perform in-clinic gait assessments\n* Unwilling or unable to comply with all study procedures\n* Age less than 8 or over 50 years","8 Years","50 Years",{"count":94,"type":21},22,"The objective of this study is to understand how spatiotemporal and kinetic gait parameters are associated with bone health in pwSMA who are receiving DMT. We hypothesize that gait parameters are associated with BMD and can determine fracture risk in pwSMA. This is an observational study that involves one in-clinic visit (approximately 3 hours) with remote follow-up visits (approximately 15 minutes) every 3 months for 1 year to collect fracture and medical history. Each of the assessments included in this study are non-interventional and are not intended as a treatment nor are they at the level of standard of care.",[30,97],"Ambulatory Spinal Muscular Atrophy",[61,28,64,99,100,101,102,103],"bone health","fracture","bone mineral density","gait","low bone mass","2026-03-16",{"date":106,"type":37},"2026-03-18",{"date":108,"type":37},"2026-03-05",{"date":110,"type":21},"2027-06-30",{"name":112,"class":82},"Jacqueline Montes",1,{"id":115,"slug":116,"hasResults":11,"nctId":117,"briefTitle":118,"officialTitle":119,"acronym":4,"eligibilityCriteria":120,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":121,"targetDuration":4,"studyType":22,"phases":123,"briefSummary":125,"conditions":126,"keywords":127,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":129,"lastUpdatePostDateStruct":130,"startDateStruct":132,"completionDateStruct":134,"leadSponsor":136,"locationsCount":113},"100548502","phase-1-evaluation-of-safety-and-efficacy-of-gene-therapy-drug-in-the-treatment-of-spinal-muscular-atrophy-sma-type-3-patients-100548502","NCT06421831","Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients","A Multi-center, Open Label, Single-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Efficacy of Gene Therapy Drug GC101 in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients","Inclusion Criteria:\n\n* ≥2 years of age on the day of signing the informed consent form;\n* Genetic and clinical diagnosis of type 3 SMA with bi-allelic deletion of SMN1 of 5qSMA;\n* Hammersmith Functional Motor Scale - Expanded (HFMSE) score is between 10 and 54 at screening;\n* Female patients of childbearing age who are pregnant or lactating, as well as all enrolled patients （both male and female), should take effective contraceptive measures within 6 months after the treatment;\n* Patients or patient's legal guardian(s) must be able to understand the purpose and risks of the study and voluntarily provide signed and dated informed consent prior to any study-related procedures being performed.\n\nExclusion Criteria:\n\n* Patient who has participated in any previous gene therapy research trials;\n* Patient who has AAV9 neutralizing antibody titer ≥1:200;\n* Patient who has received Nusinersen within 120 days and Risdiplam within 15 days before treatment;\n* Patient who requires invasive or non-invasive ventilatory support averaging≥16 hours\u002Fday at screening;\n* SMN2 copy numbers \\>4；\n* Patient who needs nasal or gastric tube feeding for eating;\n* Patient who is positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody;\n* Known allergy or hypersensitivity to prednisolone or other glucocorticosteroids or their excipients\n* Severe contractures at screening that interfere with either the ability to attain\u002Fdemonstrate functional measures or with the ability to receive intrathecal (IT) dosing;\n* Patient who has other serious diseases, such as severe cardiovascular and cerebrovascular diseases, digestive system diseases, urinary system diseases, endocrine system diseases, hematological diseases, immune system diseases, nervous system diseases (including but not limited to epilepsy, meningitis, history of convulsions or seizures, cerebrospinal fluid circulation disorders), and mental illnesses, etc.;\n* Patient with previous injuries (such as upper or lower limb fractures) or surgical operations that have not fully recovered or reached a stable state;\n* Vaccination no longer than 2 weeks before treatment;\n* Patient who has any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in the study.",{"count":122,"type":21},21,[124,24],"PHASE1","The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.",[30],[128],"SMA type 3","2025-07-01",{"date":131,"type":37},"2025-07-03",{"date":133,"type":37},"2024-05-10",{"date":135,"type":21},"2028-12",{"name":137,"class":44},"GeneCradle Inc"]