C
Heterozygous familial hypercholesterolemia
Genetic & Family HistoryDisease / conditionrisknon modifiable
18
evidence score
Also known as: heterozygous familial hypercholesterolaemia
PREVENT status
Not a PREVENT input variable.
Causal support
trial evidence
Score components
lit volume: 3meta-analyses: 0trials: 14.6genetic: 0MR bonus:
References (1)
0 meta-analyses / systematic reviews- Fasting is not routinely required for determination of a lipid profile: clinical and laboratory implications including flagging at desirable concentration cut-points-a joint consensus statement from the European Atherosclerosis Society and European Federation of Clinical Chemistry and Laboratory Medicine. — European heart journal 2016 · PMID 27122601
Clinical trials (28)
| NCT | Title | Status | Type | Interventions |
|---|---|---|---|---|
| NCT07058077 | An Operationally Seamless Phase 2/3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Enlicitide Decanoate in Pediatric Participants With Heterozygous Familial Hypercholesterolemia | RECRUITING | INTERVENTIONAL | Enlicitide Decanoate, Placebo |
| NCT06597019 | 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 | RECRUITING | INTERVENTIONAL | Inclisiran, Placebo |
| NCT05952869 | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of MK-0616 in Adults With Heterozygous Familial Hypercholesterolemia. | COMPLETED | INTERVENTIONAL | Enlicitide Decanoate, Placebo |
| NCT04797104 | Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Long-Term Efficacy and Safety of LIB003 in Heterozygous FH Patients on Stable Lipid-Lowering Therapy Requiring Additional LDL-C Reduction | COMPLETED | INTERVENTIONAL | lerodalcibep |
| NCT04666298 | A Placebo-controlled, Double-blind, Randomized Trial to Evaluate the Effect of Different Doses of Inclisiran Given as Subcutaneous Injections in Japanese Participants With High Cardiovascular Risk and Elevated LDL-C | COMPLETED | INTERVENTIONAL | Inclisiran sodium, Placebo |
| NCT04759534 | Phase III Clinical Study to Evaluate the Efficacy and Safety of PCSK9 Inhibitors in the Prevention of Chinese Heterozygous Familial Hypercholesterolemia | UNKNOWN | INTERVENTIONAL | protein convertase subtilisin/kexin type 9 inhibitor |
| NCT03814187 | An Open-label Extension Trial of the Phase III Lipid-lowering Trials to Assess the Effect of Long Term Dosing of Inclisiran Given as Subcutaneous Injections in Subjects With High Cardiovascular Risk and Elevated LDL-C | COMPLETED | INTERVENTIONAL | Inclisiran Sodium |
| NCT03694197 | Long Term Safety Study of PRALUENT in Patients With Heterozygous Familial Hypercholesterolemia or With Non-Familial Hypercholesterolemia at High and Very High Cardiovascular Risk and Previously Enrolled in the Neurocognitive Function Trial | TERMINATED | INTERVENTIONAL | Praluent |
| NCT03510884 | A Randomized, Double-Blind, Placebo-Controlled Study Followed by an Open Label Treatment Period to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia | COMPLETED | INTERVENTIONAL | Alirocumab SAR236553 (REGN727), Rosuvastatin, Atorvastatin |
| NCT02957682 | A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Effect of Praluent on Neurocognitive Function in Patients With Heterozygous Familial Hypercholesterolemia or With Non-Familial Hypercholesterolemia at High and Very High Cardiovascular Risk | COMPLETED | INTERVENTIONAL | Praluent (Alirocumab), Placebo |
| NCT02890992 | An 8-Week Open-Label, Sequential, Repeated Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia Followed by an Extension Phase | COMPLETED | INTERVENTIONAL | alirocumab SAR236553 (REGN727), statins, ezetimibe |
| NCT02624869 | Open-label, Single-Arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) | COMPLETED | INTERVENTIONAL | Evolocumab |
| NCT02392559 | Double-blind, Randomized, Multicenter, Placebo-Controlled Study to Characterize the Efficacy, Safety, and Tolerability of 24 Weeks of Evolocumab for LDL-C Reduction in Pediatric Subjects 10 to 17 Years of Age With HeFH | COMPLETED | INTERVENTIONAL | Evolocumab, Placebo |
| NCT02460159 | A Phase III, Clinical Trial to Assess the Long Term Safety and Tolerability of MK-0653C in Japanese Patients With Hypercholesterolemia Who Have Inadequate LDL-C Control on Ezetimibe or Atorvastatin Calcium Monotherapy | COMPLETED | INTERVENTIONAL | EZ 10 mg/Atorva 20 mg FDC, EZ 10 mg/Atorva 10 mg FDC |
| NCT02326220 | A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Alirocumab in Patients With Heterozygous Familial Hypercholesterolemia Undergoing Lipid Apheresis Therapy | COMPLETED | INTERVENTIONAL | Placebo, Alirocumab |
| NCT02107898 | A Randomized, Double-blind, Placebo-controlled, Parallel Group, Multicenter Study to Evaluate the Efficacy and Safety of Alirocumab in Heterozygous Familial Hypercholesterolemia or High Cardiovascular Risk Patients With Hypercholesterolemia Not Adequately Controlled With Their Lipid Modifying Therapy | COMPLETED | INTERVENTIONAL | Placebo (for alirocumab), Alirocumab, Lipid-Modifying Therapy (LMT) |
| NCT01954394 | Open-Label Extension Study of EFC12492, R727-CL-1112, EFC12732 and LTS11717 Studies to Assess the Long-Term Safety and Efficacy of Alirocumab in Patients With Heterozygous Familial Hypercholesterolemia | COMPLETED | INTERVENTIONAL | Alirocumab, Lipid-Modifying Therapy (LMT) |
| NCT01763918 | A Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 on LDL-C in Subjects With Heterozygous Familial Hypercholesterolemia | COMPLETED | INTERVENTIONAL | Evolocumab, Placebo |
| NCT01709500 | A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of REGN727/SAR236553 in Patients With Heterozygous Familial Hypercholesterolemia Not Adequately Controlled With Their Lipid-Modifying Therapy | COMPLETED | INTERVENTIONAL | LMT (atorvastatin, simvastatin, or rosuvastatin), alirocumab, Placebo |
| NCT01623115 | A Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of SAR236553/REGN727 in Patients With Heterozygous Familial Hypercholesterolemia Not Adequately Controlled With Their Lipid-Modifying Therapy | COMPLETED | INTERVENTIONAL | Alirocumab, Placebo (for alirocumab), Lipid Modifying Therapy (LMT) |
| NCT01617655 | A Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of SAR236553/REGN727 in Patients With Heterozygous Familial Hypercholesterolemia and LDL-C Higher or Equal to 160mg/dL With Their Lipid-Modifying Therapy | COMPLETED | INTERVENTIONAL | Alirocumab, Placebo (for alirocumab), Lipid Modifying Therapy (LMT) |
| NCT01576484 | A Phase 2, Open-Label Extension of Study R727-CL-1003 to Evaluate the Long-Term Safety and Efficacy of REGN727 Administered by Subcutaneous Injection in Patients With Heterozygous Familial Hypercholesterolemia | COMPLETED | INTERVENTIONAL | Alirocumab, Placebo Matched to Alirocumab |
| NCT01524289 | A 1-Year, Worldwide, Multicenter, Double-Blind, Randomized, Parallel, Placebo-Controlled Study to Assess the Efficacy and Tolerability of Anacetrapib When Added to Ongoing Statin Therapy With or Without Other Lipid Modifying Medication(s) in Patients With Heterozygous Familial Hypercholesterolemia | COMPLETED | INTERVENTIONAL | Anacetrapib, Placebo |
| NCT01375751 | A Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Tolerability and Efficacy of AMG 145 on LDL-C in Subject With Heterozygous Familial Hypercholesterolemia | COMPLETED | INTERVENTIONAL | Evolocumab, Placebo |
| NCT01266876 | A Randomized, Double-Blind, Placebo-Controlled, 12-Week Study of the Safety and Efficacy of REGN727 in Patients With Heterozygous Familial Hypercholesterolemia | COMPLETED | INTERVENTIONAL | Alirocumab, Placebo |
| NCT06958315 | An Observational Prospective Study to Assess the Effectiveness of Inclisiran(Leqvio®)in Patients With Atherosclerotic Vascular Disease and/or Heterozygous Familial Hypercholesterolemia Treated in Spanish Clinical Practice:the INSPIRE Study | ACTIVE NOT RECRUITING | OBSERVATIONAL | Inclisiran |
| NCT06112327 | Long-term Follow-up Study of Investigational Gene-editing Therapies in Participants With or at High Risk for Cardiovascular Disease | ENROLLING BY INVITATION | OBSERVATIONAL | |
| NCT02976818 | Relationships Between Lipoprotein(a) Levels and Aortic Valve Calcification in Patients With Heterozygous Familial Hypercholesterolemia | COMPLETED | OBSERVATIONAL |
Genetic associations (0)
None linked.