[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"heterozygous-familial-hypercholesterolemia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:heterozygous-familial-hypercholesterolemia":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,50,79,107],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":29,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":38,"lastUpdatePostDateStruct":39,"startDateStruct":42,"completionDateStruct":44,"leadSponsor":46,"locationsCount":49},"100528742","phase-1-a-study-of-verve-102-in-patients-with-familial-hypercholesterolemia-or-premature-coronary-artery-disease-100528742",false,"NCT06164730","A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease","Open-label, Phase 1b, Single Ascending Dose Study to Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial Hypercholesterolemia or Premature Coronary Artery Disease Who Require Additional Lowering of Low-density Lipoprotein Cholesterol","Inclusion Criteria:\n\n* Diagnosis of HeFH or premature CAD\n\nExclusion Criteria:\n\n* Homozygous familial hypercholesterolemia\n* Active or history of chronic liver disease\n* Current treatment with PCSK9 inhibitor or prior treatment within specified timeframe\n* Clinically significant or abnormal laboratory values as defined by the protocol","ALL","18 Years","70 Years",{"count":20,"type":21},85,"ESTIMATED","INTERVENTIONAL",[24],"PHASE1","VT-10201 is an Open-label, Phase 1b, Single-ascending Dose Study That Will Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial Hypercholesterolemia (HeFH) or Premature Coronary Artery Disease (CAD) Who Require Additional Lowering of LDL-C. VERVE-102 Uses Base-editing Technology Designed to Disrupt the Expression of the PCSK9 Gene in the Liver and Lower Circulating PCSK9 and LDL-C. This Study is Designed to Determine the Safety and Pharmacodynamic Profile of VERVE-102 in This Patient Population.",[27,28],"Heterozygous Familial Hypercholesterolemia","Premature Coronary Heart Disease",[30,31,32,33,34,35,36],"VERVE-102","Familial Hypercholesterolemia","Coronary Artery Disease","Dose Escalation","Gene Editing","Base Editing","heart-2","RECRUITING","2026-07-31",{"date":40,"type":41},"2026-08-03","ACTUAL",{"date":43,"type":41},"2024-04-30",{"date":45,"type":21},"2027-08",{"name":47,"class":48},"Verve Therapeutics, Inc.","INDUSTRY",27,{"id":51,"slug":52,"hasResults":11,"nctId":53,"briefTitle":54,"officialTitle":55,"acronym":4,"eligibilityCriteria":56,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":57,"targetDuration":4,"studyType":22,"phases":59,"briefSummary":61,"conditions":62,"keywords":63,"overallStatus":67,"whyStopped":4,"lastUpdateSubmitDate":68,"lastUpdatePostDateStruct":69,"startDateStruct":71,"completionDateStruct":73,"leadSponsor":75,"locationsCount":78},"100620113","early-phase-1-early-phase-study-of-art002g1-injection-in-hefh-safety-tolerability-and-preliminary-efficacy-100620113","NCT07353398","Early-phase Study of ART002g1 Injection in HeFH: Safety, Tolerability and Preliminary Efficacy","Early-phase Clinical Study on Safety, Tolerability and Preliminary Efficacy of ART002g1 Injection in the Treatment of Heterozygous Familial Hypercholesterolemia","Subjects must meet all the following criteria to be eligible for enrollment:\n\n1. Male or female, aged 18 to 70 years (inclusive) at the time of signing the Informed Consent Form (ICF);\n2. Body weight between 45 and 90 kg (inclusive) at screening;\n3. Definite diagnosis of heterozygous familial hypercholesterolemia (HeFH), meeting either of the following two criteria (1) or (2):\n\n（1) HeFH diagnosed to be caused by mutations in the LDLR, APOB, or PCSK9 gene;\n\n(2) Meeting 2 out of the 3 following criteria for adults per the Dutch Lipid Clinical Network (DLCN) criteria:\n\n1. Serum LDL-C ≥ 4.7 mmol\u002FL without prior lipid-lowering treatment;\n2. Cutaneous or tendinous xanthomas, or arcus cornealis (in subjects \\\u003C 45 years old);\n3. Presence of FH or early-onset atherosclerotic cardiovascular disease (ASCVD) in first-degree relatives.\n\nSubjects must not be enrolled if they meet any one or more of the following exclusion criteria:\n\n1. Diagnosis of compound heterozygous FH, double heterozygous FH, or homozygous FH (HoFH);\n2. Positive for hepatitis B surface antigen (HBsAg), or positive for hepatitis B core antibody (HBcAb) with peripheral blood HBV DNA titer ≥ 1 × 10² copies\u002FL; positive for hepatitis C virus (HCV) antibody with positive peripheral blood HCV RNA; positive for human immunodeficiency virus (HIV) antibody;\n3. Any unstable systemic disease, including but not limited to: unstable angina; cerebrovascular accident or transient ischemic attack (within 6 months prior to screening); myocardial infarction (within 6 months prior to screening); history of heart failure (NYHA Class II-IV); severe arrhythmia requiring pharmacotherapy; liver, kidney, or metabolic diseases; or other unstable systemic diseases as determined by the investigator;\n4. History of percutaneous transluminal coronary angioplasty (PTCA), percutaneous coronary intervention (PCI), or coronary artery bypass grafting (CABG) within 6 months prior to the first dose; or documented severe coronary artery stenosis as confirmed by coronary CT or coronary angiography within 90 days prior to randomization.",{"count":58,"type":21},24,[60],"EARLY_PHASE1","This study is an open-label, single ascending dose (SAD) study designed to evaluate the safety and tolerability of ART002g1 in patients with heterozygous familial hypercholesterolemia (HeFH) who require further reduction in low-density lipoprotein cholesterol (LDL-C). ART002g1 uses base editing technology, which is designed to interfere with the expression of the PCSK9 gene in the liver, thereby reducing the circulating levels of PCSK9 and LDL-C. The primary objectives of this study are to determine the safety and pharmacodynamic (PD) profiles of ART002g1 in this patient population.",[27],[64,65,66],"ART002g1","LNP","HeFH","NOT_YET_RECRUITING","2026-03-15",{"date":70,"type":41},"2026-03-17",{"date":72,"type":21},"2026-03-11",{"date":74,"type":21},"2027-07",{"name":76,"class":77},"Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine","OTHER",1,{"id":80,"slug":81,"hasResults":11,"nctId":82,"briefTitle":83,"officialTitle":84,"acronym":85,"eligibilityCriteria":86,"healthyVolunteers":11,"sex":16,"minAge":87,"maxAge":4,"enrollmentInfo":88,"targetDuration":4,"studyType":90,"phases":4,"briefSummary":91,"conditions":92,"keywords":93,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":97,"lastUpdatePostDateStruct":98,"startDateStruct":100,"completionDateStruct":102,"leadSponsor":104,"locationsCount":106},"100564821","assessment-of-the-prevalence-of-steatotic-liver-disease-associated-with-metabolic-dysfunction-in-patients-with-heterozygous-familial-hypercholesterolemia-100564821","NCT06634160","Assessment of the Prevalence of Steatotic Liver Disease Associated With Metabolic Dysfunction in Patients With Heterozygous Familial Hypercholesterolemia","Assessment of Metabolic Dysfunction-associated Steatotic Liver Disease (MASLD) Prevalence in Patients With Heterozygous Familial Hypercholesterolemia (HeFH): the STEATO-FH Study","STEATO-FH","Inclusion Criteria:\n\n* Patient aged 35 or over\n* Consultation at Nantes, Rennes or Angers University Hospital during the inclusion period\n* With a diagnosis of familial hypercholesterolemia defined by the presence of a genetic variant, ACMG classes 4 \\& 5 on LDLR, APOB or PCSK9\n* Patient not objecting to inclusion in study (no written objection)\n\nExclusion Criteria:\n\n* Protected patients: minors, adults under guardianship, curatorship and\u002For safeguard of justice\n* Pregnant or breast-feeding\n* Active viral hepatitis\n* Hemochromatosis\n* Other genetic or autoimmune hepatitis\n* Current treatment with a drug likely to cause hepatic steatosis, including amiodarone, carbamazepine, tamoxifen, valproate, clozapine, anti-retrovirals\n* Current oral corticosteroid therapy unless dose has been stable for ≥ 3 months\n* Current pathological alcohol consumption (≥ 60 g\u002Fday in men and ≥ 50 g\u002Fday in women)","35 Years",{"count":89,"type":21},200,"OBSERVATIONAL","The main goal of the STEATO-FH study is to determine the prevalence of liver steatosis within the Heterozygous Familial Hypercholesterolemia patient population.",[27],[94,27,95,96],"MASLD","steatosis","fibrosis","2025-02-05",{"date":99,"type":41},"2025-02-06",{"date":101,"type":41},"2025-01-30",{"date":103,"type":21},"2028-01-30",{"name":105,"class":77},"Nantes University Hospital",3,{"id":108,"slug":109,"hasResults":11,"nctId":110,"briefTitle":111,"officialTitle":112,"acronym":4,"eligibilityCriteria":113,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":114,"targetDuration":4,"studyType":22,"phases":116,"briefSummary":118,"conditions":119,"keywords":126,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":130,"lastUpdatePostDateStruct":131,"startDateStruct":132,"completionDateStruct":134,"leadSponsor":136,"locationsCount":138},"100559771","phase-3-a-study-on-efficacy-and-safety-of-hst101-in-chinese-patients-with-hypercholesterolemia-100559771","NCT06568471","A Study on Efficacy and Safety of HST101 in Chinese Patients with Hypercholesterolemia","A Randomized, Double-blind, Placebo-controlled Phase 3 Clinical Study to Evaluate the Efficacy and Safety of HST101 in Chinese Patients with Hypercholesterolemia","Inclusion Criteria:\n\n* Provision of written and signed informed consent form prior to any study-specific procedure;\n* Male or female participants ≥18 years of age at the screening visit;\n* Body weight ≥ 40 kg and body mass index (BMI) ≥18 and ≤35 kg\u002Fm2;\n* On a stable diet and lipid-lowering oral drugs (such as statins, ezetimibe or Hybutimibe, omega-3 compounds, fenofibrate, nicotinic acid, etc.) for at least 4 weeks prior to the first drug administration\n* LDL-C≥1.8 mmol\u002FL (70 mg\u002FdL) and TG≤4.52 mmol\u002FL (400 mg\u002FdL) at screening for ASCVD patients or those at very (ultra)-high risk for ASCVD, including patients with HeFH; LDL-C ≥ 2.6 mmol\u002FL (100 mg\u002FdL) and TG ≤ 4.52 mmol\u002FL (400 mg\u002FdL) at screening for patients at high-risk for ASCVD including patients with HeFH;\n* Patients on a PCSK9 mAb at a dose of 75 mg, 140 mg, or 150 mg Q2W must undergo a washout period of ≥6 weeks after the last dose; for those on 300 mg or 420 mg Q4W, the washout period is ≥10 weeks following last dose;\n* Female of childbearing potential must have a negative pregnancy test at the last screening visit and consent to use highly effective contraceptives during the trial and 3 months after the last dose of investigational drug.\n\nExclusion Criteria:\n\n* Documented history of homozygous familial hypercholesterolemia (HoFH);\n* Estimated glomerular filtration rate (eGFR)\\\u003C30 mL\u002Fmin\u002F1.73m2;\n* Active liver disease or hepatic dysfunction, history of liver transplant, and\u002For ALT or AST \\>2.5 × ULN at screening;\n* Poorly controlled thyroid disorder including hypothyroidism or hyperthyroidism;\n* Poorly controlled Type 1 or Type 2 diabetes mellitus defined as fasting blood glucose ≥11.0 mmol\u002FL (200 mg\u002FdL) and glycosylated hemoglobin (HbA1c) ≥ 9%;\n* Serious arrhythmia, MI, unstable angina pectoris, PCI, CABG, implantable cardioverter defibrillator, aortic valve surgery or stroke within 3 months prior to the first dose;\n* Planned cardiac surgery or revascularization during the study period;\n* New York Heart Association (NYHA) Class III-IV heart failure;\n* Pregnant or lactating women;\n* Poorly controlled hypertension (SBP≥160 mmHg or DBP≥100 mmHg in a sitting position)\n* Unexplained creatine kinase (CK) \\> 5 x ULN (retested once is needed if suspected to be related to excessive exercise or abnormal activity);\n* LDL apheresis or plasma exchange within 2 months prior to the first dose;\n* HIV, Treponema pallidum, or HCV antibody test positive, or HBV-DNA \\>ULN at screening;\n* History of prescription drug abuse, illicit drug use or alcohol abuse within 6 months prior to screening;\n* History of any major drug allergy, including allergy to protein biologics;\n* Participate another clinical trial within 30 days or less than 5 half-lifes (drug) before screening, whichever is longer",{"count":115,"type":21},210,[117],"PHASE3","This randomized study is to assess LDL-C reductions at Week 12 with monthly (Q4W \\[≤31 days\\]) dosing of HST101 (lerodalcibep) 300 mg administered subcutaneously (SC) compared to placebo in patients with atherosclerotic cardiovascular disease (ASCVD) or very-high\u002Fhigh risk for ASCVD including Heterozygous familial hypercholesterolemia (HeFH) on a stable diet and oral LDL-C lowering drug therapy, followed by 36-week open-label treatment with subsequent 4-week follow-up for total 52-week long-term safety and efficacy evaluation.",[120,121,122,27,123,124,125],"Hypercholesterolemia","Dyslipidemias","Primary Hypercholesterolemia","Hyperlipidemia; Mixed","Metabolic Disease","ASCVD",[127,128,129],"PCSK9 inhibitor","Lerodalcibep","LDL-C","2025-02-04",{"date":99,"type":41},{"date":133,"type":41},"2024-11-16",{"date":135,"type":21},"2026-05",{"name":137,"class":48},"Hasten Biopharmaceutical Co., Ltd.",18]