[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"heterozygous-familial-hypercholesterolemia-hefh\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:heterozygous-familial-hypercholesterolemia-hefh":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,42,75,111,134,163],{"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":26,"conditions":27,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":41},"100597406","phase-2-a-study-of-enlicitide-decanoate-mk-0616-an-oral-pcsk9-inhibitor-in-children-and-adolescents-with-heterozygous-familial-hypercholesterolemia-mk-0616-029-100597406",false,"NCT07058077","A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)","An Operationally Seamless Phase 2\u002F3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Enlicitide Decanoate in Pediatric Participants With Heterozygous Familial Hypercholesterolemia","Inclusion Criteria:\n\nInclusion criteria include, but are not limited to:\n\n* Has possible or definite diagnosis of HeFH based on a locally accepted diagnostic algorithm or diagnosis by genetic testing results\n* Has a fasted LDL-C value (evaluated by the central laboratory) that is ≥130 mg\u002FdL\n* Is receiving either:\n\n  * An optimized daily dose of statin (± nonstatin LLT)\n  * A nonstatin LLT with documented intolerance to at least 2 different statins, or documented intolerance to 1 statin plus refusal of statin therapy by the participant or legally acceptable representative\n* Is on a stable dose of all background LLTs for at least 30 days prior to screening, with no medication or dose changes planned during participation in Part A or Part B\n\nExclusion Criteria:\n\nExclusion criteria include, but are not limited to:\n\n* Has a history of homozygous FH based on genetic or clinical criteria, or history of known compound heterozygous FH, or double heterozygous FH\n* Has a history of nephrotic syndrome\n* Has any clinically significant malabsorption condition based on investigator assessment\n* Was previously treated\u002Fis being treated with certain other cholesterol lowering medications, including proprotein convertase subtilisin\u002Fkexin type 9 (PCSK9) inhibitors without adequate washout","ALL","6 Years","17 Years",{"count":20,"type":21},153,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE2","PHASE3","This study is designed to learn if enlicitide decanoate is safe and effective to treat children and adolescents with heterozygous familial hypercholesterolemia (HeFH) and high amounts of low-density lipoprotein cholesterol (LDL-C) in the blood.\n\nThe goals of this study are to learn about the safety of enlicitide and if children tolerate it, what happens to enlicitide in a child's body over time, and if enlicitide works to lower cholesterol levels in children more than a placebo.",[28],"Heterozygous Familial Hypercholesterolemia (HeFH)","RECRUITING","2026-07-01",{"date":32,"type":33},"2026-07-02","ACTUAL",{"date":35,"type":33},"2025-08-21",{"date":37,"type":21},"2037-01-23",{"name":39,"class":40},"Merck Sharp & Dohme LLC","INDUSTRY",33,{"id":43,"slug":44,"hasResults":11,"nctId":45,"briefTitle":46,"officialTitle":47,"acronym":48,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":16,"minAge":50,"maxAge":51,"enrollmentInfo":52,"targetDuration":4,"studyType":22,"phases":54,"briefSummary":56,"conditions":57,"keywords":58,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":64,"lastUpdatePostDateStruct":65,"startDateStruct":67,"completionDateStruct":69,"leadSponsor":71,"locationsCount":74},"100625810","detection-and-follow-up-of-coronary-lesions-in-hefh-destiny-fh-study-100625810","NCT07427472","\"Detection and Follow-up of Coronary Lesions in HeFH (DESTINY-FH Study)\"","Detection and Longitudinal Follow-up of Non-calcified and Calcified Coronary Lesions in Heterozygous Familial Hypercholesterolemia (DESTINY-FH)","DESTINY-FH","Inclusion Criteria:\n\n1. Patients with heterozygous familial hypercholesterolemia\n2. Aged 30 to 60 years.\n3. Patients who underwent a CAC score and a coronary CT angiogram at least 5 years ago, exclusively at the same imaging center (ICT de la Pitié Salpetrière).\n4. Patient asymptomatic for exertional chest pain at the time of CCTA\n5. Clinical examination performed\n6. Beneficiary of a social protection scheme or entitled person (excluding AME)\n7. Patient informed and consent form signed\n\nExclusion Criteria:\n\n1. Patient under guardianship, or unable to give consent\n2. Pregnancy, breast-feeding\n3. Technical contraindication: weight \\> 250 kg\n4. Simultaneous participation in other interventional research involving the human body, or period of exclusion following previous research involving the human body still in progress.\n5. Adults subject to a legal protection order\n6. Contraindication to esmolol and\u002For atenolol\n7. Only for patients who need to undergo a CT angiogram and a thoraco-abdominal-pelvic CT scan:\n\n   * Renal insufficiency (LC\\\u003C60)\n   * Unbalanced diabetes when acquiring a previous coronary angioscanner\n   * Personal history of cardiovascular disease and myocardial infarction at the time of CCTA\n   * Contraindication to iodinated contrast media\n   * Patient having already had an adverse event (AE) during the acquisition of a prior coronary angioscanner","30 Years","60 Years",{"count":53,"type":21},300,[55],"NA","This multicenter, non-randomized interventional study aims to assess coronary artery disease progression over 5 years in patients with genetically confirmed heterozygous familial hypercholesterolemia (HeFH), using coronary computed tomography angiography (CCTA).\n\nThe primary endpoint is the visual evaluation of coronary stenosis using CAD-RADS v2.0, identifying changes between baseline (2018-2022) and study inclusion. The study will enroll 300 patients (100 protected, 200 non-protected) from La Pitié-Salpêtrière hospital and Saint Antoine Hospital (Paris). Participation lasts up to one week. Total study duration is 2 years, with extended follow-up through routine care data over 10 years.",[28],[59,60,61,62,63],"Coronary Artery Disease","Atherosclerosis","Coronary Plaque Vulnerability","Cardiovascular Risk","Cardiovascular Events","2026-06-22",{"date":66,"type":33},"2026-06-24",{"date":68,"type":33},"2026-06-19",{"date":70,"type":21},"2036-06-19",{"name":72,"class":73},"Assistance Publique - Hôpitaux de Paris","OTHER",3,{"id":76,"slug":77,"hasResults":11,"nctId":78,"briefTitle":79,"officialTitle":80,"acronym":4,"eligibilityCriteria":81,"healthyVolunteers":11,"sex":16,"minAge":82,"maxAge":83,"enrollmentInfo":84,"targetDuration":4,"studyType":22,"phases":86,"briefSummary":88,"conditions":89,"keywords":99,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":101,"lastUpdatePostDateStruct":102,"startDateStruct":104,"completionDateStruct":106,"leadSponsor":108,"locationsCount":110},"100630707","phase-1-a-safety-and-tolerability-trial-evaluating-ctx310-in-participants-with-refractory-dyslipidemias-100630707","NCT07491172","A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias","A Phase 1 Open-label, Multicenter, First-in-human, Ascending Dose Trial Evaluating the Safety and Tolerability of a Lipid Nanoparticle Formulation of CRISPR-Guide RNA-Cas9 Nuclease (CTX310) for In Vivo Editing of the Angiopoietin-like 3 (ANGPTL3) Gene in Participants With Refractory Dyslipidemias","Key Inclusion Criteria:\n\n1. Age of ≥18 and ≤75 years at the time of signing the informed consent.\n2. Able to provide written informed consent.\n3. Participants diagnosed with persistent dyslipidemias defined by TG ≥150 mg\u002FdL - and LDL-C ≥70 mg\u002FdL in participants with ASCVD, or LDL-C ≥70 or 100mg\u002FdL in participants with or without ASCVD respectively, or TG ≥500 mg\u002FdL.\n4. Refractory to the maximal intensity or MTD of standard of care lines of lipid-lowering therapies available through routine clinical care, for at least 12 weeks prior to screening\n5. Female participants must be postmenopausal or surgically sterile.\n6. All male participants and their female partners must agree to the use of an acceptable method of effective contraception for the duration of the study.\n\nExclusion Criteria:\n\n1. Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply.\n2. Evidence of liver disease, defined as but not limited to:\n\n   LFTS \\>2 × upper limit of normal (ULN), or total bilirubin \\>2 × ULN, or INR \\>1.5 × ULN, or liver stiffness measured by liver elastography\n3. Abnormal or compromised function of kidney, heart, blood or liver.\n4. Acute coronary syndrome event or stroke within 24 weeks prior to Day 1. Acute pancreatitis within 12 weeks prior to Day 1.\n5. Current use or use within 365 days from Day 1 of any hepatocyte-targeted small interfering RNA (except inclisiran).\n6. Positive serology for HIV, hepatitis B or hepatitis C (antibody, surface antigen orNAT). Serology consistent with prior immunization will be eligible for the trial.\n7. Any prior malignancy within the past 5 years, or current malignancy (exceptions for resected or removed basal cell carcinoma, squamous cell carcinoma in situ and carcinoma in situ of the cervix or breast).\n8. Women of childbearing potential.\n\nNote: Other protocol defined Inclusion\u002FExclusion criteria may apply.\n\nNote: The inclusion and exclusion criteria listed represent the global protocol. Additional or modified eligibility criteria may apply in certain countries in accordance with local regulatory and ethics committee requirements and the approved country-specific protocol.","18 Years","75 Years",{"count":85,"type":21},90,[87],"PHASE1","This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are refractory to available treatments.",[90,91,92,93,94,28,95,96,97,98],"Cardiovascular","Metabolic Disease","Dyslipidemias","Lipid Disorder","Hypertriglyceridemia","Homozygous Familial Hypercholesterolemia (HoFH)","Severe Hypertriglyceridemia (sHTG)","Mixed Hyperlipemia","Hypercholesterolaemia",[100],"Refractory Dyslipidemias","2026-05-19",{"date":103,"type":33},"2026-05-22",{"date":105,"type":33},"2024-06-21",{"date":107,"type":21},"2028-06",{"name":109,"class":40},"CRISPR Therapeutics AG",18,{"id":112,"slug":113,"hasResults":11,"nctId":114,"briefTitle":115,"officialTitle":116,"acronym":4,"eligibilityCriteria":117,"healthyVolunteers":118,"sex":16,"minAge":82,"maxAge":4,"enrollmentInfo":119,"targetDuration":4,"studyType":22,"phases":121,"briefSummary":122,"conditions":123,"keywords":4,"overallStatus":124,"whyStopped":4,"lastUpdateSubmitDate":125,"lastUpdatePostDateStruct":126,"startDateStruct":128,"completionDateStruct":130,"leadSponsor":132,"locationsCount":4},"100628716","phase-3-a-study-to-evaluate-the-syh2053-injection-in-patients-with-heterozygous-familial-hypercholesterolemia-hefh-100628716","NCT07465263","A Study to Evaluate the SYH2053 Injection in Patients With Heterozygous Familial Hypercholesterolemia (HeFH)","A Study to Evaluate the SYH2053 Injection in Patients With Heterozygous Familial Hypercholesterolemia (HeFH): A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase Ⅲ Clinical Trial","Inclusion Criteria:\n\n1. Male or female participants ≥18 years of age.\n2. HeFH.\n3. Stable moderate-to-high intensity statin therapy (± cholesterol absorption inhibitors) .\n4. Maintained a low-fat diet for ≥4 weeks before signing the ICF.\n5. Fasting LDL-C at screening ≥ 2.6 mmol\u002FL or ≥ 1.4 mmol\u002FL without ASCVD or with ASCVD.\n6. Fasting TG ≤5.6 mmol\u002FL at screening.\n\nExclusion Criteria:\n\n1. HoFH or suspected HoFH.\n2. Use of medications that significantly affect LDL-C levels.\n3. Hypersensitivity or suspected allergy to oligonucleotide drugs or excipients of the investigational product.\n4. Major adverse cardiovascular events (MACE) within 180 days before signing the ICF; history of hemorrhagic stroke; or extreme-risk ASCVD at screening.\n5. Uncontrolled (by medication\u002Fablation) or severe arrhythmias within 180 days before signing the ICF.\n6. NYHA Class III-IV heart failure or LVEF \\\u003C40% within 1 year before signing ICF or at screening.\n7. Type 1 diabetes.\n8. Uncontrolled severe illness or conditions that may interfere with study results\u002Fincrease risk at screening, according to investigator's judgment.\n9. History of malignancy or underlying malignancy within 5 years before signing ICF or at screening.\n10. Major surgery within 180 days before signing ICF or planned during the study.\n11. History of drug\u002Falcohol abuse within 5 years before signing ICF.\n12. Participation in another clinical trial within 90 days or 5 half-lives (whichever is longer) before signing ICF, or planned during the study.\n13. Any of the following at screening:\n\n1）SBP ≥160 mmHg or DBP ≥100 mmHg. 2）ALT\u002FAST \\>3× ULN, or total bilirubin \\>1.5× ULN. 3）CK \\>2.5× ULN. 4）QTcF interval: \\>450 ms for male, \\>470 ms for female. 5）eGFR \\\u003C30 mL\u002Fmin\u002F1.73 m². 6）HBsAg positive with HBV DNA positive; or HCV\u002Fsyphilis\u002FHIV antibody positive. 7）TSH\\\u003CLLN, or TSH\\>ULN. 8）HbA1c \\>8.5%. 14.Pregnancy\u002Flactation or planned parenthood, and\u002For without effective contraception for female and male participants of childbearing potential from the study to 3 months after the end of treatment.\n\n\\-",true,{"count":120,"type":21},135,[25],"Heterozygous Familial Hypercholesterolemia (HeFH) is an autosomal dominant disorder characterized by markedly elevated low-density lipoprotein cholesterol (LDL-C) and increased risk of atherosclerotic cardiovascular disease (ASCVD). This trial aims to evaluate the SYH2053 Injection in patients with HeFH.",[28],"NOT_YET_RECRUITING","2026-03-17",{"date":127,"type":33},"2026-03-19",{"date":129,"type":21},"2026-03-31",{"date":131,"type":21},"2028-05-31",{"name":133,"class":40},"CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.",{"id":135,"slug":136,"hasResults":11,"nctId":137,"briefTitle":138,"officialTitle":139,"acronym":140,"eligibilityCriteria":141,"healthyVolunteers":11,"sex":16,"minAge":82,"maxAge":4,"enrollmentInfo":142,"targetDuration":4,"studyType":22,"phases":144,"briefSummary":145,"conditions":146,"keywords":148,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":153,"lastUpdatePostDateStruct":154,"startDateStruct":156,"completionDateStruct":158,"leadSponsor":160,"locationsCount":162},"100629136","the-origin-fh-study-100629136","NCT07470723","The ORIGIN-FH Study","Opportunity to Reach Individuals With Genetic Dyslipidemia During Infancy and the Newborn Period to Find Familial Hypercholesterolemia","ORIGIN-FH","Inclusion Criteria - parent participant:\n\n* Individuals in the expectant partnership providing informed consent are at least 18 years of age.\n* Ability to understand and willingness to sign a written informed consent document.\n* Willingness to comply with all study procedures and be available for the duration of the study.\n* Expectant parent (currently pregnant with fetus ≥12 weeks gestation) where one or both partners have been diagnosed with possible or definite HoFH or HeFH based on Dutch Lipid Clinic Network (DLCN) or confirmed diagnosis from a healthcare provider. At minimum, at least one parent with HoFH or HeFH must be willing to consent to study participation. However, both parents will be invited to participate.\n* Parent(s) commit to using local laboratory services for infant blood samples, with mobile phlebotomy used as an alternative if available in their area.\n\nInclusion Criteria - newborn participant:\n\n* Newborn does not have any congenital abnormalities or medical conditions that may interfere with collection of dried blood spot (DBS) specimen and newborn does not require admission to neonatal intensive care unit.\n\nExclusion Criteria - parent and newborn participants:\n\n* Expectant partnership where neither partner meets diagnostic criteria for HeFH or HoFH.\n* Parent refuses consent for newborn's study participation.\n* Newborn has medical condition precluding DBS specimen collection, or a newborn's DBS specimen is not collected by 1 week of age.\n* Not suitable for study participation due to other reasons at the discretion of the investigators.",{"count":143,"type":21},70,[55],"The goal of this clinical trial is to identify different types of Familial Hypercholesterolemia (FH) in infants and newborns. Participants will:\n\n* undergo a cheek swab for genetic testing (parents only)\n* have 5 blood samples collected\n\nParticipants can expect to be in the trial for 2 years.",[28,95,147],"Familial Hypercholesterolemia",[149,150,151,152],"cholesterol","newborn screening","pediatric","infant","2026-03-09",{"date":155,"type":33},"2026-03-13",{"date":157,"type":33},"2026-02-14",{"date":159,"type":21},"2029-02-14",{"name":161,"class":73},"University of Wisconsin, Madison",1,{"id":164,"slug":165,"hasResults":11,"nctId":166,"briefTitle":167,"officialTitle":168,"acronym":4,"eligibilityCriteria":169,"healthyVolunteers":11,"sex":16,"minAge":82,"maxAge":170,"enrollmentInfo":171,"targetDuration":4,"studyType":22,"phases":173,"briefSummary":174,"conditions":175,"keywords":4,"overallStatus":124,"whyStopped":4,"lastUpdateSubmitDate":176,"lastUpdatePostDateStruct":177,"startDateStruct":179,"completionDateStruct":181,"leadSponsor":183,"locationsCount":4},"100627572","phase-3-qlc7401-in-participants-with-heterozygous-familial-hypercholesterolemia-hefh-100627572","NCT07450378","QLC7401 in Participants With Heterozygous Familial Hypercholesterolemia (HeFH)","Evaluation of Efficacy and Safety of QLC7401 in Participants With Heterozygous Familial Hypercholesterolemia on a Stable Lipid-lowering Therapy: A Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Clinical Study","Inclusion Criteria:\n\n* Aged 18-80 years (inclusive)\n* Participants diagnosed with heterozygous familial hypercholesterolemia by Dutch Lipid Clinic\n* Network criteria or genetic testing;\n* Serum low density lipoprotein cholesterol ≥2.6 millimoles (mmol)\u002Fliter (L) at screening.\n* Fasting triglyceride \\\u003C4.5 mmol\u002FL at screening.\n* Participants on lipid-lowering therapies should be on a stable dose for ≥4 weeks before screening with no planned medication or dose change during study participation.\n* Estimated glomerular filtration Rate (eGFR) \\>30 mL\u002Fmin\u002F1.73 m².\n* Willing to provide informed consent.\n\nExclusion Criteria:\n\n* New York Heart Association (NYHA) class III or IV heart failure.\n* Uncontrolled cardiac arrhythmia.\n* Cardiovascular Diseases within 3 months.\n* Uncontrolled hypertension.\n* Poorly controlled type 2 diabetes.\n* Significant liver or kidney disease.\n* Pregnancy or lactation.\n* Other conditions deemed unsuitable by investigator.","80 Years",{"count":172,"type":21},120,[25],"This Phase III clinical trial aims to assess the efficacy and safety of QLC7401 Treatment in adults with Heterozygous Familial Hypercholesterolemia on a stable lipid-lowering therapy. Participants will be randomly assigned to receive subcutaneous injections of either QLC7401 or a placebo according to the study schedule.",[28],"2026-02-27",{"date":178,"type":33},"2026-03-04",{"date":180,"type":21},"2026-05",{"date":182,"type":21},"2028-07",{"name":184,"class":40},"Qilu Pharmaceutical Co., Ltd."]