[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100538660":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":19,"centralContacts":20,"locations":19,"responsibleParty":29,"collaborators":19,"id":33,"slug":34,"hasResults":35,"nctId":36,"briefTitle":37,"officialTitle":38,"acronym":19,"eligibilityCriteria":39,"healthyVolunteers":35,"sex":40,"minAge":41,"maxAge":42,"enrollmentInfo":43,"targetDuration":19,"studyType":46,"phases":47,"briefSummary":49,"conditions":50,"keywords":53,"overallStatus":56,"whyStopped":19,"lastUpdateSubmitDate":57,"lastUpdatePostDateStruct":58,"startDateStruct":61,"completionDateStruct":63,"leadSponsor":65,"locationsCount":19},{"fullName":5,"class":6},"Suzhou Municipal Hospital","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"NGGT006","EXPERIMENTAL","3 doses of NGGT006 will be administered according to the principle of dose escalation",[13],"Drug: NGGT006",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"DRUG","Single intravenous infusion of NGGT006 at low dose (7.5e12vg\u002Fkg), medium dose (1.5e13vg\u002Fkg) and high dose (3e13vg\u002Fkg).",[9],null,[21,26],{"name":22,"role":23,"phone":24,"phoneExt":19,"email":25},"Jun Zhang, MD","CONTACT","+86 0512-62363323","zhangjun0808@njmu.edu.cn",{"name":27,"role":23,"phone":24,"phoneExt":19,"email":28},"Yan Chen, PhD","Chenyandoc@163.com",{"type":30,"investigatorFullName":31,"investigatorTitle":32,"investigatorAffiliation":5,"oldNameTitle":19,"oldOrganization":19},"PRINCIPAL_INVESTIGATOR","Yan Chen","President of Suzhou Municipal Hospital","100538660","early-phase-1-safety-and-efficacy-study-of-nggt006-in-refractory-hypercholesterolemia-patients-100538660",false,"NCT06293729","Safety and Efficacy Study of NGGT006 in Refractory Hypercholesterolemia Patients","A Clinical Study for the Safety and Efficacy of Intravenous Infusion of NGGT006 in Treatment of Refractory Hypercholesterolemia","Inclusion Criteria:\n\n1. 18 ≤ age ≤ 55 years old;\n2. A patient with a clear diagnosis of refractory hypercholesterolemia and confirmed by genetic testing to be familial hypercholesterolemia;\n3. AAV binding antibody titer ≤1:80 and AAV neutralizing antibody ≤1:5;\n4. 18≤BMI (body mass index)≤35;\n5. During the screening period, the subjects have received stable maximum tolerated dose of lipid-lowering drug treatment, but LDL-C was still ≥70mg\u002FdL with clinical atherosclerotic cardiovascular disease; or LDL-C level was ≥ 100 mg\u002FdL without clinical atherosclerotic cardiovascular disease: the highest tolerated dose refers to (the following must be met at the same time):\n\n   ① Moderate to high doses of statins for ≥4 weeks, whether used alone or in combination with other lipid-lowering drugs; exceptions: subjects cannot tolerate statins; or subjects cannot receive statin treatment due to other reasons, such as low BMI, etc.;\n\n   ② Ezetimibe ≥ 4 weeks;\n\n   ③ Alirocumab 150mg Q2W or 300mg Q4W; evolocumab 140mg Q2W or 420mg Q4W; ≥8 weeks; And during the clinical trial process, any adjustment involving the type and dosage of lipid-lowering drugs must be approved by the researcher;\n6. Stable healthy diet for ≥12 weeks, and can adhere to a healthy diet throughout the entire clinical trial;\n7. Voluntarily sign the informed consent form and be willing to comply with the trial visit plan;\n8. Willing to maintain a similar amount and intensity of exercise during the study period as during the baseline period;\n9. Maintain good living habits, have no history of alcoholism or alcohol dependence (ICD-10 diagnosis is F10)\n10. No new or recurring cardiovascular events (myocardial infarction, cerebral infarction, etc.) within half a year;\n11. No stent implantation plan within three months;\n12. Female subjects have not had sexual intercourse for 14 days before administration, and their blood tests indicate that they are not pregnant;\n13. Subjects of childbearing age agree to use highly effective contraceptive measures for at least 365 days from the time of NGGT006 administration.\n\nExclusion Criteria:\n\n1. Secondary hyperlipidemia;\n2. Use of other drugs or nutritional products that may affect blood lipids (such as fibrates) within 6 weeks;\n3. Have received low-density lipoprotein apheresis (LDL apheresis) within the past 2 months;\n4. Large weight fluctuations (≥5kg) in the past 2 months;\n5. Positive for hepatitis B surface antigen, hepatitis C, human immunodeficiency virus (HIV)，syphilis test or other infections (such as Epstein-Barr virus, Mycoplasma pneumoniae, tuberculosis virus, HPV, Chlamydia pneumoniae, respiratory syncytial virus, Adenovirus and coxsackievirus group B, etc.);\n6. Clinically significant abnormalities in liver function test: alanine aminotransferase (ALT) \\>2 × upper limit of normal (ULN) and\u002For aspartate aminotransferase (AST) \\>2 × ULN;\n7. RR at the baseline \\>160\u002F100mmHg (one repeated measurement is allowed);\n8. Uncontrollable myocardial infarction or heart failure, and those planning surgery within one year; or new acute coronary syndrome in the past six months;\n9. Diabetes diagnosed within 3 months or with poor control (HbA1c \\>9%);\n10. Abnormal thyroid function, or those using thyroid hormone replacement therapy but poorly controlled (TSH within the normal range for \\\u003C12 weeks);\n11. Acute or chronic renal insufficiency;\n12. Hemoglobin (Hb) \\\u003C 120g\u002FL (male), Hb \\\u003C 110 (female);\n13. Abnormal platelet counts or morphology;\n14. History or laboratory tests suggestive of thrombosis;\n15. Had contraindications to glucocorticoid (e.g., epilepsy, severe schizophrenia, active peptic ulcer)；\n16. Used systemic glucocorticoid treatment within 6 weeks before enrollment;\n17. Life expectancy less than 1 year;\n18. Suffering from malignant tumors such as liver cancer; liver fibrosis;\n19. Previous gene therapy treatment;\n20. Hypersensitivity to AAV preparations (for example trehalose) or cortisone or immunosuppressants (sirolimus, rituximab, tacrolimus);\n21. Suffering from immunodeficiency disease\n22. Participation in any other clinical trial within 3 months;\n23. Breastfeeding females;\n24. Any other condition that may not be appropriate for the study in the opinion of the Investigator.","ALL","18 Years","55 Years",{"count":44,"type":45},9,"ESTIMATED","INTERVENTIONAL",[48],"EARLY_PHASE1","This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT006 in patients with refractory Hypercholesterolemia diagnosed by gene testing for familial hypercholesterolemia. NGGT006 uses adeno-associated virus (AAV) as a vector, carrying a liver specific promoter and codon optimized human LDLR gene, driving the expression of LDLR protein with normal function and promoting the clearance of low-density lipoprotein cholesterol (LDL-C).",[51,52],"Refractory Hypercholesterolemia","Familial Hypercholesterolemia",[54,55],"Gene therapy","Low-density lipoprotein cholesterol","NOT_YET_RECRUITING","2024-04-16",{"date":59,"type":60},"2024-04-17","ACTUAL",{"date":62,"type":45},"2024-06-01",{"date":64,"type":45},"2029-03-01",{"name":5,"class":6}]