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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Study of Zodasiran in Adolescent Participants With Homozygous Familial HypercholesterolemiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, homozygous familial hypercholesterolemia, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseRecruitingPhase 32026-10-06NCT07473843
Computerized Decision Support for Identification and Management of Familial HypercholesterolemiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Hypercholesterolemia, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseRecruitingNot applicable2026-09-29NCT06743659
Cascade Testing In Identifying At-risk Family Members Of Individuals With Familial Hypercholesterolemia (FH) In PCPCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseNot yet recruitingNot applicable2026-09-18NCT07653555
A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory DyslipidemiasCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Hypercholesterolemia, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, homozygous familial hypercholesterolemia, hyperlipidemia, hyperlipoproteinemia, hyperlipoproteinemia type V, hypertriglyceridemia, inborn errors of metabolism, metabolic diseaseRecruitingPhase 12026-09-18NCT07491172
Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 StudiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, homozygous familial hypercholesterolemia, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseRecruitingPhase 32026-09-18NCT05682378
The Spanish Familial Hypercholesterolaemia Cohort StudyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, cardiovascular disorder, familial hypercholesterolemia, hereditary disease, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseRecruitingNot stated2026-09-04NCT02693548
NGGT006 Gene Therapy for Homozygous Familial HypercholesterolemiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, homozygous familial hypercholesterolemia, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseRecruitingEarly phase 12026-09-03NCT06125847
A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery DiseaseArterial Occlusive Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, arteriosclerosis disorder, cardiovascular disorder, coronary artery disorder, familial hypercholesterolemia, heart disorder, hereditary disease, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic disease, myocardial ischemia, vascular disorderRecruitingPhase 12026-09-02NCT06164730
An Observational Study to Evaluate Treatment Patterns in High, Very High and Extreme Cardiovascular Risk Patients With Hypercholesterolemia, Including Familial HypercholesterolemiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Hypercholesterolemia, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseNot yet recruitingNot stated2026-08-21NCT07606118
Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Coronary Plaque Volumes Measured by Coronary Computed Tomography Angiography (CCTA) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)Arterial Occlusive Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, arteriosclerosis disorder, atherosclerosis, cardiovascular disorder, familial hypercholesterolemia, hereditary disease, homozygous familial hypercholesterolemia, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic disease, vascular disorderRecruitingNot stated2026-08-21NCT07447648
Study of ARO-DIMERPA in Adult Participants With Mixed HyperlipidemiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseRecruitingPhase 1 / Phase 22026-08-18NCT07223658
IBI306 Monotherapy in Non-Familial Hypercholesterolemia and Mixed HyperlipidemiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseActive, not recruitingPhase 32026-08-14NCT07473960
A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, homozygous familial hypercholesterolemia, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseActive, not recruitingPhase 32026-08-12NCT07037771
2-Hydroxybenzylamine (2-HOBA) to Reduce HDL Modification and Improve HDL Function in Familial Hypercholesterolemia (FH)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseRecruitingPhase 22026-08-07NCT04941599
Specified Drug-use Survey of Leqvio for s.c. Injection.Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Hypercholesterolemia, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, homozygous familial hypercholesterolemia, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseActive, not recruitingNot stated2026-08-06NCT06275724
Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial HypercholesterolemiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Hypercholesterolemia, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, Ventricular Outflow Obstruction, aortic valve disorder, aortic valve stenosis, cardiovascular disorder, familial hypercholesterolemia, heart disorder, heart failure, heart valve disorder, hereditary disease, homozygous familial hypercholesterolemia, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseRecruitingPhase 32026-07-24NCT06597006
A Study of SYH2070 Injection in Participants With Mixed HyperlipidemiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseRecruitingPhase 22026-07-22NCT07695597
Phase 1b Study of VERVE-201 in Patients With Refractory HyperlipidemiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Hypercholesterolemia, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, hyperlipidemia, hyperlipoproteinemia, hypertriglyceridemia, inborn errors of metabolism, metabolic diseaseRecruitingPhase 12026-07-17NCT06451770
A Real-World Study of Long-Term Adherence and Persistence to Inclisiran, Evolocumab, and AlirocumabArterial Occlusive Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, arteriosclerosis disorder, atherosclerosis, atherosclerotic cardiovascular disease, cardiovascular disorder, familial hypercholesterolemia, hereditary disease, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic disease, vascular disorderRecruitingNot stated2026-07-15NCT07543731
A Dose-exploration Study of EDP167 in HoFHCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, homozygous familial hypercholesterolemia, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseRecruitingPhase 22026-05-20NCT07489209
Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial HypercholesterolemiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Hypercholesterolemia, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, Ventricular Outflow Obstruction, aortic valve disorder, aortic valve stenosis, cardiovascular disorder, familial hypercholesterolemia, heart disorder, heart failure, heart valve disorder, hereditary disease, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseRecruitingPhase 32026-05-15NCT06597019
Child-Parent Familial Hypercholesterolemia ScreeningCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseRecruitingNot stated2026-05-15NCT04529967
An Adapted Brazilian Cardioprotective Diet, Phytosterols and Krill Oil in Familial Hypercholesterolemia (DICA-FH)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseActive, not recruitingNot applicable2026-05-12NCT06331195
National Network for Cardiovascular Genomics: Advancing Cardiovascular Healthcare for Hereditary Diseases in Brazil's Unified Health System Through a Multicenter RegistryAbnormalities, Multiple, Aneurysm, Aortic Dissection, Arrhythmias, Cardiac, Bone Diseases, Developmental, Brugada syndrome, Cardiac Conduction System Disease, Cardiomegaly, Cardiovascular Abnormalities, Collagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Death, Death, Sudden, Death, Sudden, Cardiac, Dissection, Blood Vessel, Dyslipidemias, Ehlers-Danlos syndrome, Ehlers-Danlos syndrome, vascular type, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Loeys-Dietz syndrome, Marfan syndrome, Musculoskeletal Abnormalities, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Polymorphic Catecholaminergic Ventricular Tachycardia, Skin Abnormalities, Skin and Connective Tissue Diseases, Tachycardia, aortic aneurysm, aortic disorder, aortic valve disorder, aortic valve stenosis, arrhythmogenic right ventricular cardiomyopathy, bone disorder, cardiac arrest, cardiomyopathy, cardiovascular disorder, congenital heart disease, connective tissue disorder, dilated cardiomyopathy, familial hypercholesterolemia, heart disorder, heart valve disorder, hematologic disorder, hemorrhagic disease, hereditary disease, hereditary skin disorder, hyperlipidemia, hyperlipoproteinemia, hypertrophic cardiomyopathy, inborn errors of metabolism, laminopathy, metabolic disease, musculoskeletal system disorder, non-compaction cardiomyopathy, restrictive cardiomyopathy, short QT syndrome, skin disorder, subvalvular aortic stenosis, vascular disorder, vascular hemostatic disease, ventricular tachycardiaRecruitingNot stated2026-05-08NCT06546137
A Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of VSA003 in Chinese HoFH PatientsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, familial hypercholesterolemia, hereditary disease, homozygous familial hypercholesterolemia, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic diseaseActive, not recruitingPhase 32026-05-05NCT06712771