[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100502585":3},{"organization":4,"armGroups":7,"interventions":18,"overallOfficials":24,"centralContacts":25,"locations":31,"responsibleParty":97,"collaborators":24,"id":99,"slug":24,"hasResults":100,"nctId":101,"briefTitle":102,"officialTitle":103,"acronym":24,"eligibilityCriteria":104,"healthyVolunteers":100,"sex":105,"minAge":106,"maxAge":107,"enrollmentInfo":108,"targetDuration":24,"studyType":111,"phases":112,"briefSummary":115,"conditions":116,"keywords":118,"overallStatus":34,"whyStopped":24,"lastUpdateSubmitDate":120,"lastUpdatePostDateStruct":121,"startDateStruct":124,"completionDateStruct":126,"leadSponsor":128,"locationsCount":129},{"fullName":5,"class":6},"GeneCradle Inc","INDUSTRY",[8,14],{"label":9,"type":10,"description":11,"interventionNames":12},"Low dosage group","EXPERIMENTAL","2.4x10\\^14 vg\u002Fperson of GC101 delivered one-time intrathecally (n=3)",[13],"Genetic: GC101",{"label":15,"type":10,"description":16,"interventionNames":17},"High dosage group","4.8x10\\^14 vg\u002Fperson of GC101 delivered one-time intrathecally (n=3)",[13],[19],{"type":20,"name":21,"description":22,"armGroupLabels":23,"otherNames":24},"GENETIC","GC101","Self-complementary AAV9 carrying a codon-optimized SMN coding sequence(coSMN1) driven by CMV enhancer and chicken β-actin promoter",[15,9],null,[26],{"name":27,"role":28,"phone":29,"phoneExt":24,"email":30},"GeneCradle, Inc. China","CONTACT","86-13501380583","ind@bj-genecradle.com",[32,52,65,81],{"facility":33,"status":34,"city":35,"state":24,"zip":36,"country":37,"cosmosGeoPoint":38,"geoPoint":43,"contacts":44},"Peking University， First Hospital, Department of Pediatrics","RECRUITING","Beijing","100034","China",{"type":39,"coordinates":40},"Point",[41,42],116.39723,39.9075,{"lat":42,"lon":41},[45,49],{"name":46,"role":28,"phone":47,"phoneExt":24,"email":48},"Yanbin Fan","86-13126582705","fanyanbin0323@163.com",{"name":50,"role":51,"phone":24,"phoneExt":24,"email":24},"Hui Xiong","PRINCIPAL_INVESTIGATOR",{"facility":53,"status":34,"city":35,"state":24,"zip":54,"country":37,"cosmosGeoPoint":55,"geoPoint":57,"contacts":58},"Bayi Children's Hospital, Seventh Medical Center, PLA general hospital","100700",{"type":39,"coordinates":56},[41,42],{"lat":42,"lon":41},[59,63],{"name":60,"role":28,"phone":61,"phoneExt":24,"email":62},"Xiuwei Ma","86-1361111080","pony007@vip.sina.com",{"name":64,"role":51,"phone":24,"phoneExt":24,"email":24},"Zhicun Feng",{"facility":66,"status":34,"city":67,"state":24,"zip":68,"country":37,"cosmosGeoPoint":69,"geoPoint":73,"contacts":74},"Shengjing Hospital of China Medical University","Shenyang","110004",{"type":39,"coordinates":70},[71,72],123.43278,41.79222,{"lat":72,"lon":71},[75,79],{"name":76,"role":28,"phone":77,"phoneExt":24,"email":78},"Ping Ma","86-15501095755","maping@joinn-lab.com",{"name":80,"role":51,"phone":24,"phoneExt":24,"email":24},"Hua Wang",{"facility":82,"status":34,"city":83,"state":24,"zip":84,"country":37,"cosmosGeoPoint":85,"geoPoint":89,"contacts":90},"Children's Hospital of Soochow University","Suzhou","215025",{"type":39,"coordinates":86},[87,88],120.59538,31.30408,{"lat":88,"lon":87},[91,95],{"name":92,"role":28,"phone":93,"phoneExt":24,"email":94},"Xiaoyan Shi","86-13913532943","314054161@qq.com",{"name":96,"role":51,"phone":24,"phoneExt":24,"email":24},"Jihong Tang",{"type":98,"investigatorFullName":24,"investigatorTitle":24,"investigatorAffiliation":24,"oldNameTitle":24,"oldOrganization":24},"SPONSOR","100502585",false,"NCT05824169","Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 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 1 Patients","Inclusion Criteria:\n\n* Six months of age and younger on day of vector infusion with Type 1 SAM as defined by the following features:\n\n  * Diagnosis of SMA based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and 2 copies of SMN2;\n  * Onset of disease before 6 months of age\n* The 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 a previous gene therapy research trials;\n* Patient who has received Nusinersen and Risdiplam treatment;\n* Patient who has AAV9 neutralizing antibody titer ≥1:200;\n* Patient who requires non-invasive ventilatory support averaging≥16 hours\u002Fday;\n* Patient with a point mutation in SMN2 (c.859G\\>C);\n* Patient who requires non-invasive ventilatory support averaging≥16 hours\u002Fday at screening;\n* Patient who use invasive ventilatory support or pulse oximetry \\\u003C 95% saturation while awake and calm at screening;\n* Patient who is positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody;\n* Abnormal laboratory values considered clinically significant, including gamma-glutamyl transferase(GGT), Aspartate aminotransferase (AST), alanine aminotransferase (ALT), bilirubin \\> 3x upper limit of normal (ULN), Hemoglobin (Hgb)\\\u003C 110 or \\>150 g\u002FL, platelet \\\u003C183x10\\^9\u002FL or 614x10\\^9\u002FL;\n* Class IV patient based on Modified Ross Heart Failure Classification for Children;\n* Patient with a history of glucocorticoid allergy;\n* Contraindication that would interfere with the lumbar puncture procedures;\n* Presence of an untreated active infection requiring systemic antiviral therapy at any time during the screening period;\n* Vaccination less than 2 weeks before infusion of vector;\n* Patient who has any concurrent clinically significant major disease or any other condition that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study.\n\nNote: Other protocol defined inclusion\u002Fexclusion criteria may apply.","ALL","0 Months","6 Months",{"count":109,"type":110},18,"ESTIMATED","INTERVENTIONAL",[113,114],"PHASE1","PHASE2","The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.",[117],"Spinal Muscular Atrophy",[119],"Type 1","2025-07-01",{"date":122,"type":123},"2025-07-03","ACTUAL",{"date":125,"type":123},"2023-02-25",{"date":127,"type":110},"2026-12",{"name":5,"class":6},4]