[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"GeneCradle Inc\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":158},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,46,71,91,110,133],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":34,"lastUpdatePostDateStruct":35,"startDateStruct":38,"completionDateStruct":40,"leadSponsor":42,"locationsCount":45},"100606313","phase-1-phase-iii-clinical-study-to-evaluate-the-safety-tolerability-and-efficacy-of-gc310-injection-in-patients-with-wilsons-disease-wd-100606313",false,"NCT07173933","Phase I\u002FII Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD)","A Multicenter, Open-label, Single-dose, Dose-escalation Phase I\u002FII Clinical Trial Evaluating the Safety, Tolerability, and Efficacy of GC310 Adeno-associated Virus Injection in the Treatment of Patients With Wilson's Disease (WD)","Inclusion Criteria:\n\n* Aged ≥ 18 years, sex unrestricted;\n* Definitive diagnosis of Wilson disease (WD) based on:\n\n  (i) or (ii) + (iii) and (iv), or (i) or (ii) + (v); (i) Neurological and\u002For psychiatric symptoms; (ii) Unexplained liver injury; (iii) Reduced serum ceruloplasmin and\u002For elevated 24-hour urinary copper; (iv) Positive corneal Kayser-Fleischer (K-F) ring; (v) Biallelic pathogenic ATP7B variants confirmed by segregation analysis and variant pathogenicity assessment;\n* Serum ceruloplasmin concentration \\\u003C ½ × lower limit of normal (LLN);\n* Willing and able to comply with all study procedures, and has provided written informed consent.\n\nExclusion Criteria:\n\nSubjects meeting ANY of the following criteria will be excluded:\n\n1. Screening serum anti-AAV5 neutralizing antibody titre \\> 1:100.\n2. Clinically significant laboratory abnormality at screening or baseline:\n\n   1. ALT or AST ≥ 5 × ULN, direct bilirubin \\> 1 × ULN, or albumin \\\u003C 1 × LLN;\n   2. Blood ammonia \\> 1 × ULN.\n3. Renal impairment (any degree).\n4. Current hepatic decompensation or history of hepatic decompensation.\n5. Liver stiffness measurement (LSM) ≥ 15 kPa by transient elastography at screening.\n6. History of acute liver failure from any cause.\n7. Evidence of advanced liver disease defined by either:\n\n   1. MELD score ≥ 12, or\n   2. Child-Pugh score ≥ 7.\n8. Severe neuro-psychiatric manifestations that, in the investigator's opinion, could compromise subject safety or interfere with study participation.\n9. Positive for HIV antibody, hepatitis C antibody, Treponema pallidum antibody, or hepatitis B surface antigen.\n10. Contraindications to glucocorticoid therapy judged by the investigator (e.g., uncontrolled hypertension, systemic fungal infection, glaucoma, osteoporosis, active tuberculosis).\n11. Concurrent conditions that may interfere with study conduct or assessment, including significant gastrointestinal, cardiovascular, cerebrovascular, renal, endocrine, haematological, immunological, neurological or psychiatric disorders other than Wilson disease.\n12. Pregnant or lactating women.\n13. Women of child-bearing potential or fertile men who plan to conceive within 1 year after dosing or are unwilling to use highly effective contraception.\n14. Body-mass index ≥ 24 kg\u002Fm².\n15. History of severe hypersensitivity to foods or drugs, including recombinant proteins.\n16. Vaccination within 2 weeks prior to planned dosing.\n17. Prior exposure to any gene-therapy product.\n18. Participation in any other clinical trial (WD-related or not) within 3 months before screening.\n19. Any other condition or circumstance that, in the opinion of the investigator, renders the subject unsuitable for the study (e.g., poor compliance).","ALL","18 Years",{"count":19,"type":20},15,"ESTIMATED","INTERVENTIONAL",[23,24],"PHASE1","PHASE2","The goal of this clinical trial is to learn if GC310 (AAV5-ATP7B) gene therapy can treat Wilson's Disease (WD) in patients over the age of 18 years old. The main questions it aims to answer are:\n\nIs GC310 safe and tolerable to WD patients? What is the recommended phase II dose (RP2D)? What is the change from baseline in 24-hour urinary copper concentration after 52 weeks of administration?\n\nParticipants will be administrated GC310 intravenously and be followed up for 52 weeks to observe drug safety, tolerability and efficacy .",[27],"Wilson Disease",[29,30,31,32,27],"gene therapy","Genecradle","AAV5","ATP7B","NOT_YET_RECRUITING","2025-09-08",{"date":36,"type":37},"2025-09-15","ACTUAL",{"date":39,"type":20},"2025-10",{"date":41,"type":20},"2027-10",{"name":43,"class":44},"GeneCradle Inc","INDUSTRY",1,{"id":47,"slug":48,"hasResults":11,"nctId":49,"briefTitle":50,"officialTitle":51,"acronym":4,"eligibilityCriteria":52,"healthyVolunteers":11,"sex":16,"minAge":53,"maxAge":4,"enrollmentInfo":54,"targetDuration":4,"studyType":21,"phases":56,"briefSummary":57,"conditions":58,"keywords":60,"overallStatus":62,"whyStopped":4,"lastUpdateSubmitDate":63,"lastUpdatePostDateStruct":64,"startDateStruct":66,"completionDateStruct":68,"leadSponsor":70,"locationsCount":45},"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.","2 Years",{"count":55,"type":20},21,[23,24],"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.",[59],"Spinal Muscular Atrophy Type 3",[61],"SMA type 3","RECRUITING","2025-07-01",{"date":65,"type":37},"2025-07-03",{"date":67,"type":37},"2024-05-10",{"date":69,"type":20},"2028-12",{"name":43,"class":44},{"id":72,"slug":73,"hasResults":11,"nctId":74,"briefTitle":75,"officialTitle":76,"acronym":4,"eligibilityCriteria":77,"healthyVolunteers":11,"sex":16,"minAge":78,"maxAge":4,"enrollmentInfo":79,"targetDuration":4,"studyType":21,"phases":81,"briefSummary":82,"conditions":83,"keywords":4,"overallStatus":62,"whyStopped":4,"lastUpdateSubmitDate":63,"lastUpdatePostDateStruct":85,"startDateStruct":86,"completionDateStruct":88,"leadSponsor":90,"locationsCount":45},"100546192","phase-1-evaluation-of-the-safety-and-efficacy-of-late-onset-pompe-disease-gene-therapy-drug-100546192","NCT06391736","Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug","A Multi-centered, Single Arm, Open Labeled, Study to Evaluate the Safety, Tolerability, and Efficacy of an Adeno-associated Virus Vector Expressing the Human Acid Alpha-glucosidase (GAA) Transgene Intravenous Injection in Patients With Late-onset Pompe Disease","Inclusion Criteria:\n\n* Age ≥ 6 years, males or females;\n* Patient has a diagnosis of LOPD;\n* Patient has upright FVC ≥ 30% of predicted normal value;\n* A 6MWT ≥ 40 meters, assistive device allowed;\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 any history or concurrent clinical organic disease, including cardiovascular and liver diseases, respiratory system, nervous system disease, or any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in the study.\n* Patient who requires invasive mechanical ventilation, or rely on noninvasive non-non-invasive assisted ventilation when sitting upright;\n* Patient who is positive for human immunodeficiency (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody;\n* Patient with a history of glucocorticoid allergy;\n* Patient who has a contraindication to study drug or to corticosteroids, or has demonstrated hypersensitivity to any of the components of the study drug;\n* Patient who has AAV9 neutralizing antibody titer ≥ 1:100；\n* Patient who has participated in a previous gene therapy research trial;\n* Pregnant or lactating female participants;\n* Patients who have fertility plans within 6 months from screening to the end of the study and are unwilling to take effective physical contraceptive measures (such as a condom, intrauterine device, contraceptive ring, ligation, abstinence, etc.) for contraception (including the subject's partner);","6 Years",{"count":80,"type":20},33,[23,24],"This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with late-onset Pompe disease （LOPD） who are ≥ 6 years old will be studied.",[84],"Pompe Disease (Late-onset)",{"date":65,"type":37},{"date":87,"type":37},"2024-04-19",{"date":89,"type":20},"2026-12",{"name":43,"class":44},{"id":92,"slug":93,"hasResults":11,"nctId":94,"briefTitle":95,"officialTitle":96,"acronym":4,"eligibilityCriteria":97,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":98,"enrollmentInfo":99,"targetDuration":4,"studyType":21,"phases":101,"briefSummary":102,"conditions":103,"keywords":4,"overallStatus":62,"whyStopped":4,"lastUpdateSubmitDate":63,"lastUpdatePostDateStruct":105,"startDateStruct":106,"completionDateStruct":108,"leadSponsor":109,"locationsCount":45},"100505377","phase-1-safety-evaluation-of-gene-therapy-drug-in-the-treatment-of-primary-hypertriglyceridemic-patients-with-recurrent-pancreatitis-100505377","NCT05860569","Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic Patients With Recurrent Pancreatitis","A Multi-center, Open Label, Multi-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Tolerance of Gene Therapy Drug GC304 in the Treatment of Primary Hypertriglyceridemia Patients With History of Acute Pancreatitis","Inclusion Criteria:\n\n* Diagnosed as primary hypertriglyceridemia poorly managed by regular treatment and dietary control, with episode of acute pancreatitis twice or once of severe acute pancreatitis within 5 years;\n* Fasting plasma triglycerides (TG) levels above 5.65 mmol\u002FL (intake of dietary fat \\\u003C30 g within 24 hours before blood taken);\n* Homozygous or heterozygous mutations in GPIHBP1 or LPL genes by genetic screening;\n* The patients within reproductive age take effective contraceptive measures voluntarily entering screening stage until 6 months after the trial;\n* The patients fully understand and are able to comply with the requirements of the treatment and are willing to complete the trial as planned, including voluntary compliance with the trial procedures, acceptance of low-fat dietary requirements, and provide of biological samples.\n* Be able to understand the procedures and methods of the trial and voluntarily participate with the signature of the informed consent by the patient or his\u002Fher guardian.\n\nExclusion Criteria:\n\n* Patient who is known to be allergic to any ingredient of a trial drug (including immunosuppressants) or has any disease prohibited from the treatment;\n* Patient who is having active bacteria, fungi, viruses or other infections;\n* Patient who is intolerant of immunosuppressive drugs or steroids;\n* Patient who is with any of the following clinical history of serious illness or existing serious illness:\n\n  1. unrelieved abdominal pain caused by acute onset of pancreatitis or by other causes；\n  2. disease history of malignancy or currently suffering from any malignant tumor;\n  3. autoimmune diseases;\n  4. disease history of epilepsy or mental illness (e.g. schizophrenia, depression, mania, anxiety, etc.);\n  5. heart diseases: cardiomyopathy and myocarditis; structural heart diseases; coronary heart disease (acute coronary syndrome, myocardial infarction); pericardial disease; severe arrhythmias (severe tachycardia requiring pacemakers, severe rapid arrhythmias, and other arrhythmias beyond the control of medications) ; New York Heart Association (NYHA) classification heart function grading ≥III or Left Ventricular Ejection Fraction (LVEF) ≤50%;\n  6. poorly controlled diabetes (fasting blood glucose ≥11.1mmol\u002FL);\n  7. with systolic blood pressure (SBP) \\> 150mmHg and\u002For diastolic blood pressure (DBP) \\> 100mmHg after treatment with a stable dose (at least 4 weeks) of antihypertensive drugs;\n* The results of the laboratory examination at screening meet either of the following:\n\n  1. Aspartate transaminase (AST) or alanine transaminase (ALT) \\> 2 × upper limit of normals (ULN);\n  2. Total bilirubin \\> upper limit of normals (ULN);\n  3. Creatinine \\> upper limit of normals (ULN);\n  4. Phosphatase kinase \\> 2 × upper limit of normals (ULN);\n  5. Glomerular filtration rate estimate \\\u003C 50 mL\u002Fmin (estimated by the Cockroft-Gault formula);\n  6. Positive hepatitis B surface antigen, positive hepatitis C antibody, positive HIV antibody or positive syphilis spiral antibody before or during screening;\n  7. A positive blood pregnancy test;\n* AAV5 neutralizing antibody levels above 1:100\n* Person who has used a clinical trial drug within 1 month (30 days) prior to screening, or who plans to participate in other clinical trials during the trial period;\n* Blood loss\u002Fdonation of more than 400 mL (except for female physiological blood loss) within 3 months (90 days) before screening, and receiving blood transfusion or using blood products;\n* Person who has undergone major surgery within 3 months (90 days) prior to screening, or who has undergone surgery that could significantly affect the course or safety evaluation of the trial drug;\n* Alcohol consumption was high in the first 3 months (90 days), i.e. the average alcohol intake was greater than 3 units\u002Fday (Male) or 2 units\u002Fdays (female) (1 unit = 18ml alcohol, such as beer 360 ml with 5% alcohol, 12% wine 150ml, 40% liquor 45ml); or who cannot abstain from drinking during the trial;\n* Women who are pregnant, pregnant or breastfeeding, or all persons of reproductive age who are unable to take effective contraceptives until 3 months after the completion of the study;\n* Patients who have poor compliance or who may not be able to complete the test for other reasons, or whom the investigator considers inappropriate to participate in the trial.","60 Years",{"count":100,"type":20},7,[23],"The study will evaluate safety and tolerance of intravenous delivery of GC304 gene therapy drug as a treatment of primary hypertriglyceridemic patients with previous onset of acute pancreatitis.",[104],"Hypertriglyceridemia, Familial",{"date":65,"type":37},{"date":107,"type":37},"2024-09-20",{"date":69,"type":20},{"name":43,"class":44},{"id":111,"slug":112,"hasResults":11,"nctId":113,"briefTitle":114,"officialTitle":115,"acronym":4,"eligibilityCriteria":116,"healthyVolunteers":11,"sex":16,"minAge":117,"maxAge":118,"enrollmentInfo":119,"targetDuration":4,"studyType":21,"phases":121,"briefSummary":122,"conditions":123,"keywords":125,"overallStatus":62,"whyStopped":4,"lastUpdateSubmitDate":63,"lastUpdatePostDateStruct":127,"startDateStruct":128,"completionDateStruct":130,"leadSponsor":131,"locationsCount":132},"100502585","phase-1-evaluation-of-safety-and-efficacy-of-gene-therapy-drug-in-the-treatment-of-spinal-muscular-atrophy-sma-type-1-patients-100502585","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.","0 Months","6 Months",{"count":120,"type":20},18,[23,24],"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.",[124],"Spinal Muscular Atrophy",[126],"Type 1",{"date":65,"type":37},{"date":129,"type":37},"2023-02-25",{"date":89,"type":20},{"name":43,"class":44},4,{"id":134,"slug":135,"hasResults":11,"nctId":136,"briefTitle":137,"officialTitle":138,"acronym":4,"eligibilityCriteria":139,"healthyVolunteers":11,"sex":16,"minAge":53,"maxAge":140,"enrollmentInfo":141,"targetDuration":4,"studyType":21,"phases":143,"briefSummary":145,"conditions":146,"keywords":148,"overallStatus":62,"whyStopped":4,"lastUpdateSubmitDate":150,"lastUpdatePostDateStruct":151,"startDateStruct":153,"completionDateStruct":155,"leadSponsor":157,"locationsCount":100},"100590720","phase-3-safety-and-efficacy-evaluation-of-gc101-gene-therapy-via-intrathecal-it-injectionin-the-treatment-of-patients-with-type-2-spinal-muscular-atrophy-sma---phase-iii-100590720","NCT06971094","Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III","A Multicenter, Randomized, Open-Label, Standard-of-Care-Controlled, Phase III Clinical Trial to Evaluate the Safety and Efficacy of Intrathecal (IT) Injection of GC101 Adeno-Associated Virus Injection in the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA)","Inclusion Criteria:\n\n* Patients with a confirmed diagnosis of Type 2 5q-SMA through clinical phenotype and genetic testing.\n* Patients who have been receiving regular treatment with nusinersen for more than one year prior to screening.\n* Patients who have not received treatment with risdiplam within 2 months prior to screening and have no plans to receive risdiplam treatment within 12 months after enrollment.\n* Patients who can sit independently but cannot walk independently at the time of screening (according to the definitions of independent sitting and walking in the WHO-MGRS motor milestones scale), and have an HFMSE score of ≥10 points.\n* Patients and\u002For their legal guardians are able to understand and are willing to comply with the requirements and procedures of the trial protocol, and voluntarily participate and sign the informed consent form\n\nExclusion Criteria:\n\n* Patients with serum anti-AAV9 neutralizing antibody titers \\> 1:50 at the time of screening.\n* Patients who have received nusinersen treatment within 2 months prior to enrollment.\n* Patients with any medical conditions that may affect the interpretation of study results or pose a risk to the safety of the participants, including but not limited to organ dysfunction of any cause, acute infectious diseases, primary\u002Facquired immunodeficiency diseases, severe cardiovascular\u002Fcerebrovascular diseases, gastrointestinal diseases, diabetes, known epilepsy, meningitis, seizure or convulsion history, or a family history of psychiatric disorders; and those with cerebrospinal fluid circulation disorders.\n* Patients with severe liver injury\u002Fhepatic insufficiency of any cause, including but not limited to alanine aminotransferase (ALT), aspartate aminotransferase (AST) ≥3 times the upper limit of normal (ULN); total bilirubin (TBil) ≥1.5 times the ULN.\n* Patients deemed by the investigator to have contraindications to glucocorticoid use, such as severe hypertension, diabetes, systemic infectious diseases, fungal infections, glaucoma, osteoporosis, peptic ulcer disease, tuberculosis, etc.\n* Patients with contraindications to lumbar puncture or intrathecal injection therapy.\n* Patients with any medical conditions that may affect the assessment of motor function, such as severe scoliosis, severe joint contracture deformities, planned spinal correction surgery during the trial period, severe osteoporosis, or a history of fractures.\n* Patients positive for hepatitis B surface antigen (HBsAg), human immunodeficiency virus (HIV) antibodies, hepatitis C virus (HCV) antibodies, or syphilis antibodies.\n* Patients who have received vaccinations within 2 weeks prior to dosing.\n* Patients who have previously received gene therapy or participated in any clinical trial within 3 months prior to screening.\n* Patients deemed by the investigator to be unsuitable for participation in this study.","12 Years",{"count":142,"type":20},50,[144],"PHASE3","This trial employs a multicenter, randomized, open-label, standard-of-care-controlled design and plans to enroll 50 patients with Type 2 SMA aged 2 to 12 years who have previously received nusinersen. The primary objective of the trial is to evaluate the efficacy of GC101 in treating Type 2 SMA. The secondary objectives are to assess the efficacy, safety, and pharmacokinetic (PK) profile of GC101 in treating Type 2 SMA.",[147],"SMA - Spinal Muscular Atrophy",[149,30],"SMA, type II, AAV gene therapy","2025-06-04",{"date":152,"type":37},"2025-06-05",{"date":154,"type":37},"2025-05-27",{"date":156,"type":20},"2026-12-31",{"name":43,"class":44},""]