[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100525754":3},{"organization":4,"outcomesModule":7,"designInfo":29,"detailedDescription":35,"studyPopulation":28,"armGroups":36,"interventions":43,"overallOfficials":48,"centralContacts":53,"locations":63,"responsibleParty":84,"collaborators":28,"id":86,"slug":28,"hasResults":87,"nctId":88,"briefTitle":89,"officialTitle":90,"acronym":28,"eligibilityCriteria":91,"healthyVolunteers":87,"sex":92,"minAge":93,"maxAge":94,"enrollmentInfo":95,"targetDuration":28,"studyType":98,"phases":99,"briefSummary":101,"conditions":102,"keywords":104,"overallStatus":65,"whyStopped":28,"lastUpdateSubmitDate":108,"lastUpdatePostDateStruct":109,"startDateStruct":112,"completionDateStruct":114,"leadSponsor":116,"locationsCount":117},{"fullName":5,"class":6},"First Affiliated Hospital Xi'an Jiaotong University","OTHER",{"primaryOutcomes":8,"secondaryOutcomes":16,"otherOutcomes":28},[9,13],{"measure":10,"description":11,"timeFrame":12},"Incidence of treatment-related adverse events (AE) and serious adverse events (SAE)","Incidence of AE and SAE, as assessed by physical examinations, clinical laboratory parameters and adverse event reporting","52 weeks",{"measure":14,"description":15,"timeFrame":12},"Absolute change and percent change in LDL-C","Change in LDL-C concentration from baseline to week 52",[17,20,22,24,26],{"measure":18,"description":19,"timeFrame":12},"Absolute change and percent change in apoB","Change in lipid concentrations from baseline to week 52",{"measure":21,"description":19,"timeFrame":12},"Absolute change and percent change in TC",{"measure":23,"description":19,"timeFrame":12},"Absolute change and percent change in HDL-C",{"measure":25,"description":19,"timeFrame":12},"Absolute change and percent change in TG",{"measure":27,"description":19,"timeFrame":12},"Absolute change and percent change in Lp(a)",null,{"allocation":30,"interventionModel":31,"interventionModelDescription":28,"primaryPurpose":32,"observationalModel":28,"timePerspective":28,"maskingInfo":33},"NA","SEQUENTIAL","TREATMENT",{"masking":34,"maskingDescription":28,"whoMasked":28},"NONE","Homozygous familial hypercholesterolemia (HoFH) is a rare inherited disorder of lipoprotein metabolism, characterized by extreme elevations in low-density lipoprotein cholesterol (LDL-C) and leading to early onset of severe coronary artery disease. This is an early phase 1, open-label, single-center, dose-escalation, pilot trial to evaluate the safety and efficacy of a single intravenous infusion of NGGT006 in 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). 4-15 subjects will be enrolled and divided into 4 groups according to the principle of dose escalation, respectively administered intravenous infusion of NGGT006 at dose group 1 (7.5e12vg\u002Fkg), dose group 2 (1.5e13vg\u002Fkg) , dose group 3 (3e13vg\u002Fkg) and dose group 4 (4e13vg\u002Fkg). The researcher is allowed to extend 0-3 patients. All subjects will undergo 52 weeks of treatment observation and further 260 weeks of long-term follow-up.",[37],{"label":38,"type":39,"description":40,"interventionNames":41},"NGGT006","EXPERIMENTAL","4 doses of NGGT006 will be administered according to the principle of dose escalation",[42],"Genetic: NGGT006",[44],{"type":45,"name":38,"description":46,"armGroupLabels":47,"otherNames":28},"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).",[38],[49],{"name":50,"affiliation":51,"role":52},"Zuyi Yuan, M.D.","First Affiliated Hospital of Xian Jiaotong University","STUDY_CHAIR",[54,59],{"name":55,"role":56,"phone":57,"phoneExt":28,"email":58},"Tao Zheng, M.D.","CONTACT","086-15229218127","zhengtao900305@163.com",{"name":60,"role":56,"phone":61,"phoneExt":28,"email":62},"Ge Gao, M.M.","086-18229068097","gaoge1030@163.com",[64],{"facility":51,"status":65,"city":66,"state":67,"zip":68,"country":69,"countryCode":70,"cosmosGeoPoint":71,"geoPoint":76,"contacts":77},"RECRUITING","Xi'an","Shaanxi","710061","China","CN",{"type":72,"coordinates":73},"Point",[74,75],108.92861,34.25833,{"lat":75,"lon":74},[78,80],{"name":79,"role":56,"phone":57,"phoneExt":28,"email":58},"Tao Zheng, MD",{"name":81,"role":56,"phone":82,"phoneExt":28,"email":83},"Yue Wu, MD","086-18092826334","yue.wu@xjtu.edu.cn",{"type":85,"investigatorFullName":28,"investigatorTitle":28,"investigatorAffiliation":28,"oldNameTitle":28,"oldOrganization":28},"SPONSOR","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":96,"type":97},12,"ESTIMATED","INTERVENTIONAL",[100],"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).",[103],"Homozygous Familial Hypercholesterolemia",[105,106,107],"Homozygous familial hypercholesterolemia","Gene therapy","Low-density lipoprotein cholesterol","2024-12-04",{"date":110,"type":111},"2024-12-09","ACTUAL",{"date":113,"type":111},"2023-10-29",{"date":115,"type":97},"2028-11",{"name":5,"class":6},1]