[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT06597006":3,"trial-entities:NCT06597006":292,"trial-summary:NCT06597006":296},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":20,"study_type":35,"primary_purpose":36,"phases":37,"enrollment_info":39,"interventions":42,"primary_outcomes":56,"secondary_outcomes":61,"sex":84,"minimum_age":85,"maximum_age":86,"healthy_volunteers":87,"eligibility_criteria":88,"std_ages":108,"locations":110,"central_contacts":281,"overall_officials":287,"references":290,"see_also_links":291},"NCT06597006","CKJX839C12304","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","RECRUITING","2029-04-15","2026-07","2026-07-24","2025-02-28","Novartis Pharmaceuticals","INDUSTRY",true,"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).","This is a two-part (1 year double-blind inclisiran versus placebo \u002F 1 year open-label inclisiran) multicenter 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 (LDL-C) on stable standard of care background lipid-lowering therapy.",[19],"Familial Hypercholesterolemia - Homozygous",[21,22,23,24,25,26,27,28,29,30,31,32,33,34],"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","INTERVENTIONAL","TREATMENT",[38],"PHASE3",{"count":40,"type":41},9,"ESTIMATED",[43,50],{"type":44,"name":45,"description":46,"armGroupLabels":47,"otherNames":48},"DRUG","Inclisiran","Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg, inclisiran sodium 180 mg s.c. for participants with body weight \\\u003C23 kg to ≥16 kg, or inclisiran sodium 100 mg s.c. for participants with body weight \\\u003C16 kg. The dose level is based on the participant's body weight on Day 1 (for Part 1) and Day 360 (for Part 2), respectively.",[45],[49],"KJX839",{"type":44,"name":51,"description":52,"armGroupLabels":53,"otherNames":54},"Placebo","Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)",[51],[55],"saline solution",[57],{"measure":58,"description":59,"timeFrame":60},"Percentage change in LDL-C from baseline to Day 330 (Year 1)","Evaluate the effect of inclisiran compared to placebo on reducing LDL-C \\[percent change\\] at Day 330","Baseline and Day 330",[62,66,70,72,74,76,78,80,82],{"measure":63,"description":64,"timeFrame":65},"Time-adjusted percent change in LDL-C from baseline after Day 90 and up to Day 330 (Year 1)","Evaluate the effect of inclisiran compared to placebo on reducing LDL-C \\[time-adjusted percent change\\] over Year 1","Baseline, after Day 90 up to Day 330",{"measure":67,"description":68,"timeFrame":69},"Percent change in LDL-C, total cholesterol, non-HDL-C, triglycerides, HDL-C, VLDL-C from baseline to each assessment time up to Day 720 (Year 2)","Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time","Baseline, up to Day 720",{"measure":71,"description":68,"timeFrame":69},"Percent change in PCSK9 from baseline to each assessment time up to Day 720 (Year 2)",{"measure":73,"description":68,"timeFrame":69},"Percent change in Apo B, Apo A1 from baseline to each assessment time up to Day 720 (Year 2)",{"measure":75,"description":68,"timeFrame":69},"Absolute change in LDL-C, total cholesterol, non-HDL-C, triglycerides, HDL-C, VLDL-C from baseline to each assessment time up to Day 720 (Year 2)",{"measure":77,"description":68,"timeFrame":69},"Absolute change in PCSK9 from baseline to each assessment time up to Day 720 (Year 2)",{"measure":79,"description":68,"timeFrame":69},"Absolute change in Apo B, Apo A1 from baseline to each assessment time up to Day 720 (Year 2)",{"measure":81,"description":68,"timeFrame":69},"Percent change in Lp(a) from baseline to each assessment time up to Day 720 (Year 2)",{"measure":83,"description":68,"timeFrame":69},"Absolute change in Lp(a) from baseline to each assessment time up to Day 720 (Year 2)","ALL","2 Years","11 Years",false,{"inclusion":89,"exclusion":92,"raw_text":107},[90,91],"Male or female participants, 2 to \\\u003C12 years of age at screening","HoFH diagnosed by genetic confirmation",[93,94,95,96,97,98,99,100,101,102,103,104,105,106],"Fasting LDL-C \\>130 mg\u002FdL (3.4 mmol\u002FL) at screening","On an optimal dose of statin (investigator's discretion), unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe)","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","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.","Documented evidence of a null (negative) mutation in both LDLR alleles","Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9","History of poor response to therapy with any monoclonal antibody directed towards PCSK9 (e.g. \\\u003C15% reduction in LDL-C)","Treatment with mipomersen or lomitapide (within 5 months of screening)","Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome","Heterozygous familial hypercholesterolemia (HeFH)","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)","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)","Pregnant or nursing females","Recent and\u002For planned use of other investigational medicinal products or devices","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",[109],"CHILD",[111,129,144,158,172,180,188,196,203,209,217,225,233,240,247,253,261,268,274],{"facility":112,"status":8,"city":113,"state":114,"zip":115,"country":116,"contacts":117,"geoPoint":126},"UC San Francisco Medical Center","San Francisco","California","94143-0348","United States",[118,123],{"name":119,"role":120,"phone":121,"email":122},"Luis Gay","CONTACT","+1 415 476 8338","luis.gay@ucsf.edu",{"name":124,"role":125},"Martin Thelin","PRINCIPAL_INVESTIGATOR",{"lat":127,"lon":128},37.77493,-122.41942,{"facility":130,"status":8,"city":131,"state":132,"zip":133,"country":116,"contacts":134,"geoPoint":141},"Childrens National Hospital","Washington D.C.","District of Columbia","20010",[135,139],{"name":136,"role":120,"phone":137,"email":138},"Desiree Tobechukwu Nwanze","+1 202 476 5000","dnwanze@childrensnational.org",{"name":140,"role":125},"Sarah Clauss",{"lat":142,"lon":143},38.89511,-77.03637,{"facility":145,"status":8,"city":146,"state":147,"zip":148,"country":116,"contacts":149,"geoPoint":155},"Washington Univ School Of Medicine","St Louis","Missouri","63110",[150,153],{"name":151,"role":120,"email":152},"Jodi Pagano","jpagano@wustl.edu",{"name":154,"role":125},"Anne Goldberg",{"lat":156,"lon":157},38.62727,-90.19789,{"facility":159,"status":8,"city":160,"state":161,"zip":162,"country":116,"contacts":163,"geoPoint":169},"Primary Childrens Medical Center","Salt Lake City","Utah","84113",[164,167],{"name":165,"role":120,"email":166},"Linda Lambert","Linda.lambert@hsc.utah.edu",{"name":168,"role":125},"Adam Ware",{"lat":170,"lon":171},40.76078,-111.89105,{"facility":173,"status":8,"city":174,"zip":175,"country":176,"geoPoint":177},"Novartis Investigative Site","Vienna","1090","Austria",{"lat":178,"lon":179},48.20849,16.37208,{"facility":173,"status":8,"city":181,"state":182,"zip":183,"country":184,"geoPoint":185},"Beijing","Beijing Municipality","100013","China",{"lat":186,"lon":187},39.9075,116.39723,{"facility":173,"status":8,"city":189,"state":190,"zip":191,"country":192,"geoPoint":193},"Frankfurt am Main","Hesse","60590","Germany",{"lat":194,"lon":195},50.11552,8.68417,{"facility":173,"status":8,"city":197,"zip":198,"country":199,"geoPoint":200},"Ioannina","455 00","Greece",{"lat":201,"lon":202},39.66341,20.85187,{"facility":173,"status":8,"city":204,"zip":205,"country":199,"geoPoint":206},"Thessaloniki","546 42",{"lat":207,"lon":208},40.64072,22.93493,{"facility":173,"status":8,"city":210,"state":211,"zip":212,"country":213,"geoPoint":214},"Kota Bharu","Kelantan","16150","Malaysia",{"lat":215,"lon":216},6.12361,102.24333,{"facility":173,"status":8,"city":218,"state":219,"zip":220,"country":221,"geoPoint":222},"Amsterdam","North Holland","1105 AZ","Netherlands",{"lat":223,"lon":224},52.37403,4.88969,{"facility":173,"status":8,"city":226,"state":227,"zip":228,"country":229,"geoPoint":230},"Bloemfontein","Free State","9301","South Africa",{"lat":231,"lon":232},-29.12107,26.214,{"facility":173,"status":8,"city":234,"state":235,"zip":236,"country":229,"geoPoint":237},"Johannesburg","Gauteng","2193",{"lat":238,"lon":239},-26.20227,28.04363,{"facility":173,"status":8,"city":241,"zip":242,"country":243,"geoPoint":244},"Taichung","407219","Taiwan",{"lat":245,"lon":246},24.1469,120.6839,{"facility":173,"status":8,"city":248,"zip":249,"country":243,"geoPoint":250},"Taipei","111045",{"lat":251,"lon":252},25.05306,121.52639,{"facility":173,"status":8,"city":254,"state":255,"zip":256,"country":257,"geoPoint":258},"Adana","Saricam","01330","Turkey (Türkiye)",{"lat":259,"lon":260},36.98615,35.32531,{"facility":173,"status":8,"city":262,"state":263,"zip":264,"country":257,"geoPoint":265},"Ankara","Yenimahalle","06500",{"lat":266,"lon":267},39.91987,32.85427,{"facility":173,"status":8,"city":269,"zip":270,"country":257,"geoPoint":271},"Izmir","35100",{"lat":272,"lon":273},38.41273,27.13838,{"facility":173,"status":8,"city":275,"zip":276,"country":277,"geoPoint":278},"Southampton","SO16 6YD","United Kingdom",{"lat":279,"lon":280},50.90395,-1.40428,[282,285],{"name":13,"role":120,"phone":283,"email":284},"1-888-669-6682","novartis.email@novartis.com",{"name":13,"role":120,"phone":286},"+41613241111",[288],{"name":13,"affiliation":13,"role":289},"STUDY_DIRECTOR",[],[],{"nct_id":4,"conditions":293,"biomarkers":295},[294],"Hyperlipoproteinemia, Type IIa",[],{"nct_id":4,"found":15,"summary":297,"prompt_version":307},{"design":298,"status":299,"heading":300,"summary":301,"follow_up":302,"word_count":303,"commitments":304,"compensation":305,"drugs_mentioned":306},"This is a two-part study: one year where participants receive either inclisiran or a placebo, followed by one year where all participants receive inclisiran. It is a multicenter study.","completed","Study of Inclisiran for Children with Homozygous Familial Hypercholesterolemia","This study is looking at the safety, how well it's tolerated, and how effective inclisiran is in children aged 2 to under 12 years old who have homozygous familial hypercholesterolemia (HoFH). HoFH is a genetic condition that causes very high levels of \"bad\" cholesterol (LDL-C). Inclisiran works by targeting a specific protein to help lower cholesterol. Some participants will receive inclisiran, while others will receive a placebo (an inactive substance like saline). The main goal is to see how much inclisiran can lower LDL-C levels after about one year. To join, you must have HoFH confirmed by genetic testing and high LDL-C levels. The study plans to enroll 9 children.","The primary endpoint measures changes in LDL-C from the start of the study to Day 330 (approximately one year).",110,"Not specified in the trial record.","Not stated in the trial record.",[45,51],"v2"]