[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"familial-hypercholesterolemia---homozygous\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:familial-hypercholesterolemia---homozygous":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,57],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":29,"overallStatus":44,"whyStopped":4,"lastUpdateSubmitDate":45,"lastUpdatePostDateStruct":46,"startDateStruct":49,"completionDateStruct":51,"leadSponsor":53,"locationsCount":56},"100561964","phase-3-study-to-evaluate-safety-tolerability-and-efficacy-of-inclisiran-in-children-with-homozygous-familial-hypercholesterolemia-100561964",false,"NCT06597006","Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia","Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Children (2 to Less Than 12 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol","ORION-19","Inclusion Criteria:\n\n* Male or female participants, 2 to \\\u003C12 years of age at screening\n* HoFH diagnosed by genetic confirmation\n\n  \\- Note: Participants with known null (negative) mutations in both LDLR alleles are not eligible (see also exclusion criteria)\n* Fasting LDL-C \\>130 mg\u002FdL (3.4 mmol\u002FL) at screening\n* On an optimal dose of statin (investigator's discretion), unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe)\n* Participants on lipid-lowering therapies (such as e.g. statins, ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation\n* Participants on a documented regimen of LDL-apheresis for ≥ 3 months before screening will be allowed to continue the apheresis during the study, if needed. The apheresis schedule\u002Fsettings\u002Fduration must be stable prior to screening, are not allowed to change during the double-blind period of the trial and must permit that an apheresis coincides with each study visit.\n\nExclusion Criteria:\n\n* Documented evidence of a null (negative) mutation in both LDLR alleles\n* Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9\n* History of poor response to therapy with any monoclonal antibody directed towards PCSK9 (e.g. \\\u003C15% reduction in LDL-C)\n* Treatment with mipomersen or lomitapide (within 5 months of screening)\n* Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome\n* Heterozygous familial hypercholesterolemia (HeFH)\n* Body weight (at the screening and\u002For randomization (Day 1) visit) \\\u003C16 kg for participants 6 to \\\u003C12 years (at screening) or \\\u003C11 kg for participants 2 to \\\u003C6 years (at screening)\n* Active liver disease defined as any known current infectious, neoplastic, or metabolic pathology of the liver or unexplained alanine aminotransferase (ALT), aspartate aminotransferase (AST) elevation \\>3x ULN, or total bilirubin elevation \\>2x ULN (except patients with Gilbert's syndrome)\n* Pregnant or nursing females\n* Recent and\u002For planned use of other investigational medicinal products or devices","ALL","2 Years","11 Years",{"count":21,"type":22},9,"ESTIMATED","INTERVENTIONAL",[25],"PHASE3","This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to \\\u003C12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDLC).",[28],"Familial Hypercholesterolemia - Homozygous",[30,31,32,33,34,35,36,37,38,39,40,41,42,43],"Homozygous familial hypercholesterolemia (HoFH),","LDL-cholesterol (LDL-C), children, pediatric,","small interfering ribonucleic acid (siRNA),","inclisiran,","Familial Hypercholesterolemia,","Homozygous FH,","Hypercholesterolemia,","Lipoprotein(a),","Hyperlipidemia,","Dyslipidemia,","Cardiovascular Diseases,","Heart Failure,","Cholesterol,","Aortic Stenosis","RECRUITING","2026-02-18",{"date":47,"type":48},"2026-02-20","ACTUAL",{"date":50,"type":48},"2025-02-28",{"date":52,"type":22},"2029-04-15",{"name":54,"class":55},"Novartis Pharmaceuticals","INDUSTRY",18,{"id":58,"slug":59,"hasResults":11,"nctId":60,"briefTitle":61,"officialTitle":62,"acronym":63,"eligibilityCriteria":64,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":65,"enrollmentInfo":66,"targetDuration":4,"studyType":68,"phases":4,"briefSummary":69,"conditions":70,"keywords":73,"overallStatus":44,"whyStopped":4,"lastUpdateSubmitDate":79,"lastUpdatePostDateStruct":80,"startDateStruct":82,"completionDateStruct":84,"leadSponsor":86,"locationsCount":5},"100390972","early-detection-of-familial-hypercholesterolemia-in-children-100390972","NCT04370899","Early Detection of Familial Hypercholesterolemia in Children","Early Detection of Familial Hypercholesterolemia in Children in Tarragona Province","DECOPIN","Inclusion Criteria:\n\n* Children between 2 and 18 years of age.\n* LDL-C level above 135 mg\u002FdL\n* Previously, the pediatrician will have discarded secondary causes (hypercholesterolaemia such as hypothyroidism, nephrotic syndrome, diabetes, renal insufficiency).\n\nAfter confirmation that one of the parents has a genetic mutation (Lipoxip\u002FLiponext) or clinical diagnosis (DLCN ≥ 8), the child will be studied. The progenitor with hypercholesterolemia will be considered as an index case, in this way we will demonstrate the vertical transmission of the genetic disease.\n\nExclusion Criteria:\n\n* The child population under 2 and over the age of 18 and children.\n* Children with high cholesterol but by secondary causes.","18 Years",{"count":67,"type":22},400,"OBSERVATIONAL","Heterozigous FH is an underdiagnosed disease in the paediatric population. Its early detection, would allow us to initiate lifestyle therapeutical changes and early pharmacological therapy if necessary. This is a key fact to reduce atherosclerosis progression and cardiovascular risk in adulthood. Moreover, it will allow, detecting the first and second degree affected relatives.",[71,72,28],"Familial Hypercholesterolemia","Familial Hypercholesterolemia - Heterozygous",[71,74,75,76,77,78],"Childhood","cardiovascular risk","lifestyle assessment","genetic disorder","LDL cholesterol","2025-04-17",{"date":81,"type":48},"2025-04-18",{"date":83,"type":48},"2013-03-14",{"date":85,"type":22},"2030-07-01",{"name":87,"class":88},"Institut Investigacio Sanitaria Pere Virgili","OTHER"]