[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"sma\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:sma":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,46,81,114],{"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":51,"acronym":52,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":17,"minAge":54,"maxAge":4,"enrollmentInfo":55,"targetDuration":57,"studyType":58,"phases":4,"briefSummary":59,"conditions":60,"keywords":65,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":70,"lastUpdatePostDateStruct":71,"startDateStruct":73,"completionDateStruct":75,"leadSponsor":77,"locationsCount":80},"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.","0 Years",{"count":56,"type":21},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.",[28,61,62,63,64],"DMD","BMD","IMD","Congenital Muscular Dystrophy",[61,62,63,28,66,67,68,69],"LAMA2","COL-6","CMD","NMD","2026-01-13",{"date":72,"type":37},"2026-01-15",{"date":74,"type":37},"2018-06-20",{"date":76,"type":21},"2071-01-01",{"name":78,"class":79},"University of Bern","OTHER",19,{"id":82,"slug":83,"hasResults":11,"nctId":84,"briefTitle":85,"officialTitle":86,"acronym":4,"eligibilityCriteria":87,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":88,"targetDuration":4,"studyType":58,"phases":4,"briefSummary":90,"conditions":91,"keywords":92,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":104,"lastUpdatePostDateStruct":105,"startDateStruct":107,"completionDateStruct":109,"leadSponsor":111,"locationsCount":113},"100384966","uk-sma-patient-registry-100384966","NCT04292574","UK SMA Patient Registry","Spinal Muscular Atrophy Patient Registry of the United Kingdom and Ireland","Inclusion Criteria:\n\n* All patients with a confirmed SMA diagnosis (or pending diagnosis) are eligible for inclusion. Diagnosis will be confirmed via genetic testing results\n\nExclusion Criteria:\n\n* There are no exclusion criteria for the registry",{"count":89,"type":21},800,"Spinal muscular atrophy (SMA) is a form of motor neuron disease, most commonly caused by a mutation in the survival motor neuron 1 gene (SMN1) which results in a wide disease spectrum affecting children and adults. It is an autosomal recessive disorder and is therefore caused by inheritance of a mutated gene from each parent. All forms of SMA have an estimated combined incidence of 1 in 6,000 to 1 in 10,000 live births, with a carrier frequency of 1\u002F40 to 1\u002F60.\n\nThe patient registry aims to facilitate a questionnaire-based research study in order to better characterise and understand the disease in the UK and in Ireland. Entry is via self-registration over a secure internet connection (https:\u002F\u002Fwww.sma-registry.org.uk\u002F). Online, patients are asked to read an information sheet about the research project and then indicate their consent to demonstrate willingness to participate. Following online consent, subjects will be entered into the registry. This is an on-going database and all participants are invited to update their information on a biannual basis.",[27,28],[27,28,93,94,95,96,97,98,99,100,101,102,103],"Neuromuscular Diseases","Motor Neuron Disease","Bulbo-Spinal Atrophy, X-Linked","Kennedy Disease","Spinal Muscular Atrophy with Respiratory Distress 1","Distal Spinal Muscular Atrophy","SMA type 1","SMA type 2","SMA type 3","SMA type 4","5q SMA","2024-07-22",{"date":106,"type":37},"2024-07-23",{"date":108,"type":37},"2008-07-13",{"date":110,"type":21},"2025-05-31",{"name":112,"class":79},"Newcastle University",1,{"id":115,"slug":116,"hasResults":11,"nctId":117,"briefTitle":118,"officialTitle":119,"acronym":120,"eligibilityCriteria":121,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":122,"enrollmentInfo":123,"targetDuration":4,"studyType":22,"phases":125,"briefSummary":127,"conditions":128,"keywords":129,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":133,"lastUpdatePostDateStruct":134,"startDateStruct":136,"completionDateStruct":138,"leadSponsor":140,"locationsCount":113},"100540885","a-head-to-head-study-comparing-the-functional-value-of-two-models-of-robotically-assisted-rehabilitation-in-sma-spinal-muscular-atrophy-patients-100540885","NCT06322654","A Head-to-head Study Comparing the Functional Value of Two Models of Robotically Assisted Rehabilitation in SMA (Spinal Muscular Atrophy) Patients","A Head-to-head Study Comparing the Functional Value of Two Models of Robotically Assisted Rehabilitation in SMA Patients: Single-center, Randomized, Single-blinded, Comparative Study of a Robotically Active Verticalization Model Versus Robotically Assisted Locomotion Model","SMArt","Inclusion Criteria:\n\n* Signing of informed consent to participate in the experiment by the participant or the participant's parent\u002Flegal guardian\n* Diagnosis of SMA (spinal muscular atrophy 1-4) confirmed by genetic testing, symptomatic or pre-symptomatic\n* Age between 0 and 21 years of age (will be determined on the day of starting participation in the project based on the date of birth)\n* Treatment under a drug program for spinal muscular atrophy, provided that the patient has a diagnosis of spinal muscular atrophy at the date of eligibility.\n\nExclusion Criteria:\n\n* Cardio-respiratory disorders requiring invasive ventilation\n* Advanced osteoporosis with multiple fractures prior to treatment\n* Functional deterioration during the rehabilitation process in the range of scales appropriately selected for the SMA type: Prechtl, HINE, CHOP-INTEND, HFMS, RULM\n* Lack of cooperation with the therapist\n* Other functional indications preventing exercise","21 Years",{"count":124,"type":21},200,[126],"NA","A head-to-head study comparing the functional value of two models of robotically assisted rehabilitation in patients with SMA. A single-center, randomized, single-blinded, comparative study of Robotically Assisted Verticalization versus Robotically Assisted Locomotion.\n\nThe objective of research:\n\nThe main goal of the project is to determine the optimal robotically assisted rehabilitation model for people with SMA depending on age and baseline functional status. The study consists of a head-to-head comparison of two rehabilitation models.\n\n1. Research period: 4 years\n2. Patients age: 0-21 y.o.\n3. Group size: 200 patients (100 patients in each group)\n4. Assignment of patients to study groups in a randomised manner",[28],[28,130,131,132],"Robotically-assisted therapy","Robotically Assisted Locomotion","Robotically Assisted Verticalization","2024-03-13",{"date":135,"type":37},"2024-03-21",{"date":137,"type":37},"2023-12-21",{"date":139,"type":21},"2027-07",{"name":141,"class":79},"Wiktor Dega University Orthopedic and Rehabilitation Hospital"]