[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100502585":3},{"organization":4,"armGroups":7,"interventions":18,"overallOfficials":24,"centralContacts":25,"locations":31,"responsibleParty":98,"collaborators":24,"id":100,"slug":101,"hasResults":102,"nctId":103,"briefTitle":104,"officialTitle":105,"acronym":24,"eligibilityCriteria":106,"healthyVolunteers":102,"sex":107,"minAge":108,"maxAge":109,"enrollmentInfo":110,"targetDuration":24,"studyType":113,"phases":114,"briefSummary":117,"conditions":118,"keywords":120,"overallStatus":34,"whyStopped":24,"lastUpdateSubmitDate":122,"lastUpdatePostDateStruct":123,"startDateStruct":126,"completionDateStruct":128,"leadSponsor":130,"locationsCount":131},{"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",[32,53,66,82],{"facility":33,"status":34,"city":35,"state":24,"zip":36,"country":37,"countryCode":38,"cosmosGeoPoint":39,"geoPoint":44,"contacts":45},"Peking University， First Hospital, Department of Pediatrics","RECRUITING","Beijing","100034","China","CN",{"type":40,"coordinates":41},"Point",[42,43],116.39723,39.9075,{"lat":43,"lon":42},[46,50],{"name":47,"role":28,"phone":48,"phoneExt":24,"email":49},"Yanbin Fan","86-13126582705","fanyanbin0323@163.com",{"name":51,"role":52,"phone":24,"phoneExt":24,"email":24},"Hui Xiong","PRINCIPAL_INVESTIGATOR",{"facility":54,"status":34,"city":35,"state":24,"zip":55,"country":37,"countryCode":38,"cosmosGeoPoint":56,"geoPoint":58,"contacts":59},"Bayi Children's Hospital, Seventh Medical Center, PLA general hospital","100700",{"type":40,"coordinates":57},[42,43],{"lat":43,"lon":42},[60,64],{"name":61,"role":28,"phone":62,"phoneExt":24,"email":63},"Xiuwei Ma","86-1361111080","pony007@vip.sina.com",{"name":65,"role":52,"phone":24,"phoneExt":24,"email":24},"Zhicun Feng",{"facility":67,"status":34,"city":68,"state":24,"zip":69,"country":37,"countryCode":38,"cosmosGeoPoint":70,"geoPoint":74,"contacts":75},"Shengjing Hospital of China Medical University","Shenyang","110004",{"type":40,"coordinates":71},[72,73],123.43278,41.79222,{"lat":73,"lon":72},[76,80],{"name":77,"role":28,"phone":78,"phoneExt":24,"email":79},"Ping Ma","86-15501095755","maping@joinn-lab.com",{"name":81,"role":52,"phone":24,"phoneExt":24,"email":24},"Hua Wang",{"facility":83,"status":34,"city":84,"state":24,"zip":85,"country":37,"countryCode":38,"cosmosGeoPoint":86,"geoPoint":90,"contacts":91},"Children's Hospital of Soochow University","Suzhou","215025",{"type":40,"coordinates":87},[88,89],120.59538,31.30408,{"lat":89,"lon":88},[92,96],{"name":93,"role":28,"phone":94,"phoneExt":24,"email":95},"Xiaoyan Shi","86-13913532943","314054161@qq.com",{"name":97,"role":52,"phone":24,"phoneExt":24,"email":24},"Jihong Tang",{"type":99,"investigatorFullName":24,"investigatorTitle":24,"investigatorAffiliation":24,"oldNameTitle":24,"oldOrganization":24},"SPONSOR","100502585","phase-1-evaluation-of-safety-and-efficacy-of-gene-therapy-drug-in-the-treatment-of-spinal-muscular-atrophy-sma-type-1-patients-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":111,"type":112},18,"ESTIMATED","INTERVENTIONAL",[115,116],"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.",[119],"Spinal Muscular Atrophy",[121],"Type 1","2025-07-01",{"date":124,"type":125},"2025-07-03","ACTUAL",{"date":127,"type":125},"2023-02-25",{"date":129,"type":112},"2026-12",{"name":5,"class":6},4]