[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"familial-hypercholesterolemia---heterozygous\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:familial-hypercholesterolemia---heterozygous":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,59,86],{"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":46,"whyStopped":4,"lastUpdateSubmitDate":47,"lastUpdatePostDateStruct":48,"startDateStruct":51,"completionDateStruct":53,"leadSponsor":55,"locationsCount":58},"100561965","phase-3-study-to-evaluate-efficacy-and-safety-of-inclisiran-in-children-with-heterozygous-familial-hypercholesterolemia-100561965",false,"NCT06597019","Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous 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 (6 to Less Than 12 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL- Cholesterol","ORION-20","Inclusion Criteria:\n\n* Male or female participants, 6 to \\\u003C12 years of age at screening\n* HeFH diagnosed either by genetic testing or on phenotypic criteria\n* Fasting LDL-C \\>130 mg\u002FdL (3.4 mmol\u002FL) at screening\n* For participants 8 to \\\u003C12 years, on an optimal dose of statin (investigator's discretion) unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe). For participants \\\u003C8 years, the use of background lipid-lowering treatment is based on investigator's discretion.\n* Participants on lipid-lowering therapies (such as statin and\u002For e.g. ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation.\n\nExclusion Criteria:\n\n* Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9\n* Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome\n* Homozygous familial hypercholesterolemia (HoFH)\n* Body weight \\\u003C16 kg at the screening and\u002For randomization (Day 1) visit\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","6 Years","11 Years",{"count":21,"type":22},60,"ESTIMATED","INTERVENTIONAL",[25],"PHASE3","This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to \\\u003C12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDLC).",[28],"Familial Hypercholesterolemia - Heterozygous",[30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45],"Heterozygous familial hypercholesterolemia (HeFH),","LDL-cholesterol (LDL-C),","Children,","pediatric,","small interfering ribonucleic acid (siRNA),","inclisiran,","Familial Hypercholesterolemia,","Heterozygous FH,","Hypercholesterolemia,","Lipoprotein(a),","Hyperlipidemia,","Dyslipidemia,","Heart Failure,","Cardiovascular Diseases,","Cholesterol,","Aortic Stenosis","RECRUITING","2026-05-13",{"date":49,"type":50},"2026-05-15","ACTUAL",{"date":52,"type":50},"2024-12-09",{"date":54,"type":22},"2029-04-15",{"name":56,"class":57},"Novartis Pharmaceuticals","INDUSTRY",66,{"id":60,"slug":61,"hasResults":11,"nctId":62,"briefTitle":63,"officialTitle":64,"acronym":65,"eligibilityCriteria":66,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":67,"enrollmentInfo":68,"targetDuration":4,"studyType":23,"phases":70,"briefSummary":71,"conditions":72,"keywords":73,"overallStatus":76,"whyStopped":4,"lastUpdateSubmitDate":77,"lastUpdatePostDateStruct":78,"startDateStruct":80,"completionDateStruct":82,"leadSponsor":84,"locationsCount":5},"100600824","phase-3-randomized-placebo-controlled-double-blind-phase-3b-study-to-evaluate-the-efficacy-and-safety-of-lerodalcibep-in-children-6-to-17-years-with-heterozygous-fh-100600824","NCT07102511","Randomized, Placebo-Controlled, Double-Blind, Phase 3b Study to Evaluate the Efficacy and Safety of Lerodalcibep in Children 6 to 17 Years, With Heterozygous FH","Randomized, Placebo-Controlled, Double-Blind, Phase 3b Study to Evaluate the Efficacy and Safety of Lerodalcibep in Children and Adolescents, 6 to 17 Years of Age, With Heterozygous Familial Hypercholesterolemia on Stable Diet and Oral Lipid-Lowering Therapy","LIBerate Kids","Inclusion Criteria:\n\n1. Provision of written and signed informed consent\u002Fassent prior to any study-specific procedure;\n2. Male or female, 6 to 17 years of age (defined as from 6 to less than 18 years of age), at the first Screening Visit;\n3. Weight of more than 18 kg (40 lbs) and BMI more than 17 and less than42 kg\u002Fm2;\n4. Diagnosis of definite, probable, or possible HeFH based on clinical criteria (SB Register criteria or genotyping and at the defined eligibility visit (Screening Visit or post washout\u002Fstabilization); a calculated LDL-C (Friedewald) equal or above 130 mg\u002FdL or, if the patient has additional risk factors as defined , a calculated LDL-C (Friedewald) equal or above100 mg\u002FdL; and TG below 400 mg\u002FdL while on stable lipid-lowering oral drug therapy (eg, maximally tolerated statin with or without ezetimibe); Note: Patients unable to tolerate approved doses of a statin may take lower than approved doses and less frequently than daily as long as the dose and dosing frequency is consistent per the Investigator's judgment. Patients with documented intolerance to statins may also participate;\n5. On a stable diet and lipid-lowering oral therapies (statins, ezetimibe, bile-acid sequestrants) or combinations thereof for at least 6 weeks (excluded oral lipid-lowering agents include mipomersen, lomitapide, and gemfibrozil);\n6. Patients on a PCSK9 mAb must undergo a washout period of \\>8 weeks after the last dose. For patients who have received an siRNA PCSK9 inhibitor the washout period is 360 days post last dose;\n7. Females of childbearing potential must be using a highly effective form of contraception if sexually active and have negative urine pregnancy test at the last Screening Visit; Note: Highly effective methods of birth control include refraining from heterosexual sexual intercourse during the entire period of risk, birth control pills or patches, intrauterine devices (IUDs), sexual activity with a male partner who has had a vasectomy, condom or diaphragm or cervical cap with spermicide or IUD, oral, implantable, or injectable contraceptives.\n8. Male patients will either be surgically sterile or agree to use the following forms of contraception: male or female condom with spermicide and a female partner who is sterile or who agrees to use the following contraceptives: diaphragm or cervical cap with spermicide; or IUD, oral, implantable, or injectable contraceptives; and\n9. Male patients must refrain from sperm donation until 90 days following the last dose of study drug.\n\nExclusion Criteria:\n\n1. Use of prohibited oral lipid-lowering agents mipomersen or lomitapide within 6 months of screening or gemfibrozil within 6 weeks of the Screening Visit;\n2. LDL or plasma apheresis within 2 months prior to Day 1;\n3. Documented history of HoFH defined as clinical and\u002For genetic with true HoFH (ie, identical pathogenic variants), compound heterozygous (ie, 2 different pathogenic LDLR variants) or combined heterozygous (2 different pathogenic FH variants such as LDLR plus ApoB or LDLR plus PCSK9 gain-of function);\n4. History of any prior or active clinical condition or acute and\u002For unstable systemic disease compromising patient inclusion, at the discretion of the Investigator, including but not limited to clinically significant pulmonary, hematologic, gastrointestinal, endocrine (excluding diabetes), immunologic, dermatologic, neurologic, or psychiatric disease, which in the Investigator's opinion, would not be suitable for the study from a patient safety consideration or could interfere with the results of the study;\n5. Females of childbearing potential who are sexually active, not using or unwilling to use a highly effective form of contraception, pregnant or breastfeeding, or who have a positive urine pregnancy test at the last Screening Visit; Note: Highly effective methods of birth control include refraining from heterosexual sexual intercourse during the entire period of risk, birth control pills or patches, IUDs, sexual activity with a male partner who has had a vasectomy, condom or diaphragm or cervical cap with spermicide or IUD, oral, implantable, or injectable contraceptives.\n6. Moderate to severe renal dysfunction, defined as an estimated glomerular filtration rate \\\u003C30 mL\u002Fmin\u002F1.73m2 at the Screening Visit;\n7. Active liver disease or hepatic dysfunction (eg, cirrhosis, alcoholic liver disease, known hepatitis B or hepatitis C, autoimmune hepatitis, liver failure, liver cancer), history of liver transplant, and\u002For AST or ALT \\>2.5 × the ULN based on age as determined by central laboratory analysis at screening (tests that result in ALT or AST up to 3 × ULN may have 1 repeat test to confirm eligibility during the Screening Period);\n8. Uncontrolled thyroid disease: hyperthyroidism or hypothyroidism as defined by thyroid stimulating hormone (TSH) below the lower limit of normal (LLN) or \\>1.5 × ULN, respectively, at the Screening Visit. If TSH is above\u002Fbelow these cut-off points, the patient can enter if the free triiodothyronine (FT3) is within the reference range. If controlled, then treatment should be stable for at least 3 months prior to the Screening Visit;\n9. Uncontrolled Type 1 or Type 2 diabetes mellitus (defined as fasting glucose above 200 mg\u002FdL and HbA1c of above 9%);\n10. Uncontrolled serious cardiac arrhythmia (sustained ventricular tachycardia, frequent non sustained ventricular tachycardia, any ventricular fibrillation episode, wide-complex tachycardia, atrial fibrillation with rapid ventricular response, and severe second-degree or third degree atrioventricular block), myocardial infarction (MI), unstable angina, percutaneous coronary intervention, coronary artery bypass grafting, placement of implantable cardioverter defibrillator or biventricular pacemaker, aortic valve surgery, or stroke within 3 months prior to enrollment (the day patient signs the informed consent\u002Fassent and first procedure is performed);\n11. Planned cardiac surgery or revascularization;\n12. New York Heart Association III-IV heart failure; or patients with last documented left ventricular ejection fraction \\\u003C30% by standard of care assessments (eg, echocardiography, cardiac magnetic resonance imaging, nuclear imaging, computed tomography angiography, or angiography with ventriculogram), within 12 months;\n13. Uncontrolled hypertension defined as a reproducible (ie, repeated 5 minutes apart) sitting blood pressure above 160 mmHg systolic or above 100 mmHg diastolic;\n14. Enrolled in another investigational device or drug study, or less than 30 days or 5 half-lives since ending another investigational device or drug study(ies), or receiving other investigational agent(s); such as PCSK9 or ANGPTL3 or Lp(a) siRNA or locked nucleic acid reducing agents within 12 months of the Screening Visit;\n15. Unexplained CK \\>5 × ULN, unless related to exercise or unusual activity in which case 1 repeat test is allowed;\n16. Patients who cannot be available for Protocol-required study visits or procedures, to the best of the patient's and Investigator's knowledge;\n17. A history, within 6 months prior to screening, of prescription drug abuse, illicit drug use, or alcohol abuse according to medical history;\n18. Donated or lost a significant volume (\\>500 mL) of blood or plasma within 30 days prior to Day 1;\n19. Had a blood transfusion within 4 weeks of randomization or known diagnosis of human immunodeficiency virus;\n20. Previous treatment with lerodalcibep or any adnectin product;\n21. Have any other finding which, in the opinion of the Investigator, would compromise the patient's safety or participation in the study; or\n22. An employee or family member of the Investigator or study site personnel.","17 Years",{"count":69,"type":22},150,[25],"The goal of this clinical trial is to assess the LDL-Cholesterol reductions at Week 12 and Week 24 with monthly dosing of lerodalcibep (Lerochol) 300 mg administered subcutaneously by auto-injector (AI)\u002Fpre-filled pen (PFP) compared to placebo (dummy), in male and female pediatric patients 6 to 17 years of age, with inherited high cholesterol (HeFH) on a stable diet and maximally tolerated oral LDL C lowering drug therapy such as statins.\n\nThe main question\\[s\\] it aims to answer are:\n\nHow effective is Lerochol in reducing LDL cholesterol? How well is it tolerated and are there any safety concerns? Researchers will compare Lerochol to placebo (inert or dummy injection solution).\n\nParticipants will visit the clinic every month for months and be asked to fast overnight, but allowed to drink water, before clinic visits. Undergo physical exams, height and weight measurements, answer questions, have blood drawn from a vein in their arm, have blood pressure measurements, EKC heart tests, and receive monthly injections lasting about 5 seconds in their arms or abdomen with an autoinjector.",[28],[74,75],"inherited high cholesterol","high cholesterol","NOT_YET_RECRUITING","2025-07-28",{"date":79,"type":50},"2025-08-03",{"date":81,"type":22},"2025-09-01",{"date":83,"type":22},"2027-06-30",{"name":85,"class":57},"LIB Therapeutics LLC",{"id":87,"slug":88,"hasResults":11,"nctId":89,"briefTitle":90,"officialTitle":91,"acronym":92,"eligibilityCriteria":93,"healthyVolunteers":11,"sex":17,"minAge":94,"maxAge":95,"enrollmentInfo":96,"targetDuration":4,"studyType":98,"phases":4,"briefSummary":99,"conditions":100,"keywords":103,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":109,"lastUpdatePostDateStruct":110,"startDateStruct":112,"completionDateStruct":114,"leadSponsor":116,"locationsCount":119},"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.","2 Years","18 Years",{"count":97,"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.",[101,28,102],"Familial Hypercholesterolemia","Familial Hypercholesterolemia - Homozygous",[101,104,105,106,107,108],"Childhood","cardiovascular risk","lifestyle assessment","genetic disorder","LDL cholesterol","2025-04-17",{"date":111,"type":50},"2025-04-18",{"date":113,"type":50},"2013-03-14",{"date":115,"type":22},"2030-07-01",{"name":117,"class":118},"Institut Investigacio Sanitaria Pere Virgili","OTHER",2]