[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100525754":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":25,"locations":35,"responsibleParty":56,"collaborators":19,"id":58,"slug":59,"hasResults":60,"nctId":61,"briefTitle":62,"officialTitle":63,"acronym":19,"eligibilityCriteria":64,"healthyVolunteers":60,"sex":65,"minAge":66,"maxAge":67,"enrollmentInfo":68,"targetDuration":19,"studyType":71,"phases":72,"briefSummary":74,"conditions":75,"keywords":77,"overallStatus":37,"whyStopped":19,"lastUpdateSubmitDate":81,"lastUpdatePostDateStruct":82,"startDateStruct":85,"completionDateStruct":87,"leadSponsor":89,"locationsCount":90},{"fullName":5,"class":6},"First Affiliated Hospital Xi'an Jiaotong University","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"NGGT006","EXPERIMENTAL","4 doses of NGGT006 will be administered according to the principle of dose escalation",[13],"Genetic: NGGT006",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"GENETIC","Single intravenous infusion of NGGT006 at dose 1 (7.5e12vg\u002Fkg), dose 2 (1.5e13vg\u002Fkg) , dose 3 (3e13vg\u002Fkg) and dose 4 (4e13vg\u002Fkg).",[9],null,[21],{"name":22,"affiliation":23,"role":24},"Zuyi Yuan, M.D.","First Affiliated Hospital of Xian Jiaotong University","STUDY_CHAIR",[26,31],{"name":27,"role":28,"phone":29,"phoneExt":19,"email":30},"Tao Zheng, M.D.","CONTACT","086-15229218127","zhengtao900305@163.com",{"name":32,"role":28,"phone":33,"phoneExt":19,"email":34},"Ge Gao, M.M.","086-18229068097","gaoge1030@163.com",[36],{"facility":23,"status":37,"city":38,"state":39,"zip":40,"country":41,"countryCode":42,"cosmosGeoPoint":43,"geoPoint":48,"contacts":49},"RECRUITING","Xi'an","Shaanxi","710061","China","CN",{"type":44,"coordinates":45},"Point",[46,47],108.92861,34.25833,{"lat":47,"lon":46},[50,52],{"name":51,"role":28,"phone":29,"phoneExt":19,"email":30},"Tao Zheng, MD",{"name":53,"role":28,"phone":54,"phoneExt":19,"email":55},"Yue Wu, MD","086-18092826334","yue.wu@xjtu.edu.cn",{"type":57,"investigatorFullName":19,"investigatorTitle":19,"investigatorAffiliation":19,"oldNameTitle":19,"oldOrganization":19},"SPONSOR","100525754","early-phase-1-nggt006-gene-therapy-for-homozygous-familial-hypercholesterolemia-100525754",false,"NCT06125847","NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia","A Clinical Study for the Safety and Efficacy of Intravenous Infusion of NGGT006 in Treatment of Homozygous Familial Hypercholesterolemia With LDLR Mutations","Inclusion Criteria:\n\n* Voluntarily sign informed consent form;\n* Male or female, 12 ≤ age ≤ 55 years (first patient≥ 18 years), diagnosed as homozygous familial hypercholesterolemia with genetic confirmation of two mutant alleles of the LDL receptor (LDLR) gene;\n* AAV8 neutralizing antibodies can be negative or reduced to negative levels through methods such as plasma exchange.\n* Untreated LDL-C ≥10 mmol\u002FL (386mg\u002F dL) or treated LDL-C ≥7 mmol\u002FL (270mg\u002F dL) together with cutaneous or tendon xanthoma before age 18 years;\n* Had been on stable medication for ≥30 days if receiving lipid-lowering therapy (or ≥60 days if receiving alirocumab or evolocumab) prior to screening and not scheduled for addition of new drugs or dose adjustments during the study;\n* Agreed to follow a low-fat diet and comply with all study procedures;\n* Agreed to maintain a similar exercise volume and intensity to baseline during the study period;\n* Agreed to maintain good lifestyle habits;\n* No history of alcohol abuse or alcohol dependence (diagnosed as F10 in ICD-10 code);\n* No sexual activity for 14 days prior to administration and negative serum pregnancy test in female participants;\n* Participants of childbearing potential agreed to use highly effective contraception for at least 365 days from administration of NGGT006;\n* No plan of stent implantation within 3 months.\n\nExclusion Criteria:\n\n* Positive for hepatitis B surface antigen, hepatitis C, human immunodeficiency virus (HIV) or syphilis test;\n* Clinically significant abnormalities in liver function test: alanine aminotransferase (ALT) ≥2 × upper limit of normal (ULN) and\u002For aspartate aminotransferase (AST) ≥2 × ULN;\n* Baseline blood pressure ≥160\u002F100 mmHg (1 repeat measurement is allowed);\n* Uncontrolled myocardial infarction or heart failure, or had surgery plan within 1 year;\n* Diabetes diagnosed within 3 months or with poor control (HbA1c ≥9%);\n* Acute or chronic kidney failure;\n* Hemoglobin (Hb) ≥120g\u002FL (male), Hb ≥110 (female);\n* Abnormal platelet counts or morphology;\n* History or laboratory tests suggestive of thrombosis;\n* Had contraindications to glucocorticoid (e.g., epilepsy, severe schizophrenia, active peptic ulcer);\n* Life expectancy less than 1 year;\n* With malignant tumors;\n* Liver fibrosis or liver cancer;\n* Previous gene therapy treatment;\n* Hypersensitivity to AAV or cortisone or immunosuppressants (sirolimus, rituximab, tacrolimus);\n* Participation in any other clinical trial within 3 months;\n* History of stent implantation within 1 month or myocardial infarction within 3 months;\n* Breastfeeding females;\n* Any other condition that may not be appropriate for the study in the opinion of the Investigator.","ALL","12 Years","55 Years",{"count":69,"type":70},12,"ESTIMATED","INTERVENTIONAL",[73],"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 homozygous familial hypercholesterolemia (HoFH) patients with LDLR mutations. NGGT006 is an adeno-associated viral (AAV) vector carrying 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).",[76],"Homozygous Familial Hypercholesterolemia",[78,79,80],"Homozygous familial hypercholesterolemia","Gene therapy","Low-density lipoprotein cholesterol","2024-12-04",{"date":83,"type":84},"2024-12-09","ACTUAL",{"date":86,"type":84},"2023-10-29",{"date":88,"type":70},"2028-11",{"name":5,"class":6},1]