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. 2024 Jan 8;9:13. doi: 10.1038/s41392-023-01690-3

Table 3.

Important clinical trials for the development of PCSK9-iTs

PCSK9-iT for CVD NCT Number Study Name Study Title Phases Study Status Conditions Interventions Sponsor Start Date Primary Completion Date

Evolocumab

Anti-PCSK9 mAb

Approved by EMA and the US FDA in 2015

 NCT01516879 DESCARTES Durable Effect of PCSK9 Antibody CompARed wiTh placEbo Study 3 COMPLETED Hypercholesterolemia

BIOLOGICAL: Evolocumab

BIOLOGICAL: Placebo

DRUG: Atorvastatin

DRUG: Ezetimibe

OTHER: Diet Only

 Amgen Jan-12 Oct-13
NCT01588496 TESLA Trial Evaluating PCSK9 Antibody in Subjects With LDL Receptor Abnormalities 2/3 COMPLETED Homozygous Familial Hypercholesterolemia

BIOLOGICAL: Evolocumab

DRUG: Placebo

 Amgen Apr-12 Jan-14
NCT01624142 TAUSSIG Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders 2/3 COMPLETED Severe Familial Hypercholesterolemia BIOLOGICAL: Evolocumab Amgen Jun-12 May-18
NCT01763827 MENDEL-2 Monoclonal Antibody Against PCSK9 to Reduce Elevated LDL-C in Subjects Currently Not Receiving Drug Therapy for Easing Lipid Levels-2 3 COMPLETED Hyperlipidemia

BIOLOGICAL: Evolocumab

DRUG: Ezetimibe

BIOLOGICAL: Placebo to Evolocumab

OTHER: Placebo to Ezetimibe

 Amgen Jan-13 Oct-13
NCT01763866 LAPLACE-2 LDL-C Assessment With PCSK9 Monoclonal Antibody Inhibition Combined With Statin Therapy-2 3 COMPLETED Hyperlipidemia

BIOLOGICAL: Evolocumab

DRUG: Ezetimibe

DRUG: Placebo to Evolocumab

DRUG: Placebo to Ezetimibe

DRUG: Atorvastatin

DRUG: Rosuvastatin

DRUG: Simvastatin

 Amgen Jan-13 Nov-13
NCT01763905 GAUSS-2 Goal Achievement After Utilizing an Anti-PCSK9 Antibody in Statin Intolerant Subjects -2 3 COMPLETED Hyperlipidemia

BIOLOGICAL: Evolocumab

DRUG: Placebo to Evolocumab

DRUG: Ezetimibe

DRUG: Placebo to Ezetimibe

 Amgen Jan-13 Nov-13
NCT01763918 RUTHERFORD-2 Reduction of LDL-C With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder Study-2 3 COMPLETED Hyperlipidemia

BIOLOGICAL: Evolocumab

DRUG: Placebo

 Amgen Feb-13 Nov-13
NCT01764633 FOURIER Further Cardiovascular Outcomes Research With PCSK9 Inhibition in Subjects With Elevated Risk 3 COMPLETED Dyslipidemia

BIOLOGICAL: Evolocumab

DRUG: Placebo

 Amgen Feb-13 Nov-16
NCT01813422 GLAGOV GLobal Assessment of Plaque reGression With a PCSK9 antibOdy as Measured by intraVascular Ultrasound 3 COMPLETED Hypercholesterolemia

BIOLOGICAL: Evolocumab

DRUG: Placebo

 Amgen Apr-13 July-16
NCT01849497 n/a Study to Assess In-home Use of Evolocumab (AMG 145) Using a Prefilled Syringe or a Prefilled Autoinjector/Pen 3 COMPLETED Primary Hypercholesterolemia|Mixed Dyslipidemia

BIOLOGICAL: Evolocumab Pre-filled Syringe

BIOLOGICAL: Evolocumab AI/pen

 Amgen Apr-13 Sep-13
NCT01854918 OSLER-2 Open-label Extension Study of Evolocumab (AMG 145) in Adults With Hyperlipidemia and Mixed Dyslipidemia 3 COMPLETED Hyperlipidemia and Mixed Dyslipidemia

BIOLOGICAL: Evolocumab

DRUG: Standard of Care

 Amgen Apr-13 May-18
NCT01879319 n/a Study to Assess in Home Use of Evolocumab (AMG 145) Administration Using Either an Automated Mini-doser or a Prefilled Autoinjector/Pen 3 COMPLETED Primary Hypercholesterolemia|Mixed Dyslipidemia

BIOLOGICAL: Evolocumab AMD

BIOLOGICAL: Evolocumab AI/pen

 Amgen Jul-13 Nov-13
NCT01953328 AMG145 Study of Low-Density Lipoprotein Cholesterol (LDL-C) Reduction Using Evolocumab (AMG 145) in Japanese Patients With Advanced Cardiovascular Risk 3 COMPLETED Hyperlipidemia or Mixed Dyslipidemia at High Risk for Cardiovascular Events

DRUG: Atorvastatin

BIOLOGICAL: Evolocumab

OTHER: Placebo to Evolocumab

 Amgen Oct-13 Jun-14
NCT01984424 GAUSS-3 Goal Achievement After Utilizing an Anti-PCSK9 Antibody in Statin Intolerant Subjects-3 3 COMPLETED Hyperlipidemia

DRUG: Atorvastatin

DRUG: Placebo to Atorvastatin

OTHER: Placebo to Ezetimibe

DRUG: Ezetimibe

OTHER: Placebo to Evolocumab

DRUG: Evolocumab

 Amgen Dec-13 Nov-15
NCT02189837 FLOREY Effects on Lipoprotein Metabolism From PCSK9 Inhibition Utilizing a Monoclonal Antibody 3 COMPLETED Primary Hyperlipidemia and Mixed Dyslipidemia

BIOLOGICAL: Evolocumab

DRUG: Atorvastatin

DRUG: Placebo to Evolocumab

DRUG: Placebo to Atorvastatin

 Amgen Jul-14 Feb-15
NCT02207634 EBBINGHAUS Evaluating PCSK9 Binding antiBody Influence oN coGnitive HeAlth in High cardiovascUlar Risk Subjects 3 COMPLETED Dyslipidemia

BIOLOGICAL: Evolocumab

DRUG: Placebo

DRUG: Background Statin Therapy

 Amgen Sep-14 Nov-16
NCT02304484 n/a Open-label Extension (OLE) Study to Assess Safety and Efficacy of Evolocumab 3 COMPLETED Hypercholesterolemia BIOLOGICAL: Evolocumab Amgen Nov-14 Mar-18
NCT02392559 HAUSER-RCT Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Pediatric Subjects With Genetic Low-Density Lipoprotein (LDL) Disorders 3 COMPLETED Heterozygous Familial Hypercholesterolemia

DRUG: Evolocumab

DRUG: Placebo

 Amgen Mar-16 Nov-19
NCT02585895 n/a Evolocumab Compared to LDL-C Apheresis in Patients Receiving LDL-C Apheresis Prior to Study Enrollment 3 COMPLETED Hypercholesterolemia

BIOLOGICAL: Evolocumab

PROCEDURE: Low-density Lipoprotein Cholesterol (LDL-C) Apheresis

 Amgen Dec-15 Sep-16
NCT02624869 HAUSER-OLE Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia) 3 COMPLETED Familial Hypercholesterolemia BIOLOGICAL: Evolocumab Amgen Sep-16 Jun-21
NCT02634580 GAUSS-4 Goal Achievement After Utilizing an Anti-PCSK9 Antibody in Statin Intolerant Subjects-4 3 COMPLETED Hypercholesterolemia

BIOLOGICAL: Evolocumab

DRUG: Ezetimibe

DRUG: Placebo to Evolocumab

DRUG: Placebo Ezetimibe

 Amgen Feb-16 Aug-17
NCT02662569 BERSON Safety and Efficacy of Evolocumab in Combination With Statin Therapy in Adults With Diabetes and Hyperlipidemia or Mixed Dyslipidemia 3 COMPLETED Diabetes, Hyperlipidemia, Mixed Dyslipidemia

BIOLOGICAL: Evolocumab

DRUG: Atorvastatin

OTHER: Placebo

 Amgen Apr-16 Dec-17
NCT02729025 ANITSCHKOW Effects of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition on Arterial Wall Inflammation in Patients With Elevated Lipoprotein(a) (Lp(a)) 3 COMPLETED Subjects With Hyperlipidemia, Dyslipidemia

DRUG: Evolocumab

DRUG: Placebo

 Amgen Apr-16 Apr-18
NCT02739984 BANTING Evaluation of Evolocumab (AMG 145) Efficacy in Diabetic Adults With Hypercholesterolemia/Mixed Dyslipidemia 3 COMPLETED

Hypercholesterolemia

Mixed Dyslipidemia

Type 2 Diabetes

BIOLOGICAL: Evolocumab

DRUG: Placebo to Evolocumab

 Amgen May-16 Aug-17
NCT02833844 n/a Safety, Tolerability and Efficacy on Low Density Lipoprotein Cholesterol (LDL-C) of Evolocumab in Participants With Human Immunodeficiency Virus (HIV) and Hyperlipidemia/Mixed Dyslipidemia 3 COMPLETED Subjects With Hyperlipidemia, Dyslipidemia and HIV Infection

DRUG: Evolocumab

DRUG: Placebo

 Amgen May-17 Jul-19
NCT02867813 FOURIER OLE Further Cardiovascular Outcomes Research With PCSK9 Inhibition in Subjects With Elevated Risk Open-label Extension 3 COMPLETED Dyslipidemia BIOLOGICAL: Evolocumab Amgen Sep-16 Mar-22
NCT03403374 RAMAN Safety and Tolerability of Repatha®(Evolocumab) in Indian Participants With Homozygous Familial Hypercholesterolemia 4 COMPLETED Homozygous Familial Hypercholesterolemia HoFH DRUG: evolocumab Amgen Aug-18 Nov-19
NCT03570697 n/a Imaging of Coronary Plaques in Participants Treated With Evolocumab 3 COMPLETED Coronary Artery Disease (CAD)

DRUG: Evolocumab

DRUG: Placebo

DRUG: Statin therapy

 Amgen Nov-18 Dec-20
NCT03872401 VESALIUS-CV Effect of Evolocumab in Patients at High Cardiovascular Risk Without Prior Myocardial Infarction or Stroke 3 ACTIVE_NOT_RECRUITING Coronary Heart Disease (CHD)

DRUG: Evolocumab

DRUG: Placebo

 Amgen Jun-19 Jul-25
NCT05284747 EVOLVE-MI EVOLVE-MI: EVOLocumab Very Early After Myocardial Infarction 4 RECRUITING

Cardiovascular Disease

Myocardial Infarction

Stroke

Coronary Revascularization

DRUG: Evolocumab

DRUG: Routine Lipid Management

 Amgen Oct-22 Jun-27

Alirocumab

Anti-PCSK9 mAb

Approved by EMA and the US FDA in 2015

 NCT01507831 ODYSSEY Long Term Long-term Safety and Tolerability of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in High Cardiovascular Risk Patients With Hypercholesterolemia (ODYSSEY Long Term) 3 COMPLETED Hypercholesterolemia

DRUG: Placebo (for alirocumab)

DRUG: Alirocumab

DRUG: Lipid-Modifying Therapy (LMT)

 Sanofi/Regeneron Jan-12 Nov-14
NCT01617655 ODYSSEY HIGH FH Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With Heterozygous Familial Hypercholesterolemia (ODYSSEY HIGH FH) 3 COMPLETED Hypercholesterolemia

DRUG: Alirocumab

DRUG: Placebo (for alirocumab)

DRUG: Lipid Modifying Therapy (LMT)

 Sanofi/Regeneron Jun-12 May-14
NCT01623115 ODYSSEY FH I Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With Heterozygous Familial Hypercholesterolemia Not Adequately Controlled With Their Lipid-Modifying Therapy 3 COMPLETED Hypercholesterolemia

DRUG: Alirocumab

DRUG: Placebo (for alirocumab)

DRUG: Lipid Modifying Therapy (LMT)

 Sanofi/Regeneron Jul-12 Apr-14
NCT01644175 ODYSSEY COMBO I Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With High Cardiovascular Risk and Hypercholesterolemia (ODYSSEY COMBO I) 3 COMPLETED Hypercholesterolemia

DRUG: Placebo (for alirocumab)

DRUG: Alirocumab

DRUG: Lipid-Modifying Therapy (LMT)

 Sanofi/Regeneron Jul-12 Apr-14
NCT01644188 ODYSSEY COMBO II Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Ezetimibe on Top of Statin in High Cardiovascular Risk Patients With Hypercholesterolemia (ODYSSEY COMBO II) 3 COMPLETED Hypercholesterolemia

DRUG: Alirocumab

DRUG: Placebo (for alirocumab)

DRUG: Ezetimibe

DRUG: Placebo (for ezetimibe)

DRUG: Lipid Modifying Therapy (LMT)

 Sanofi/Regeneron Aug-12 May-14
NCT01644474 ODYSSEY MONO Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Ezetimibe in Patients With Hypercholesterolemia 3 COMPLETED Hypercholesterolemia

DRUG: Alirocumab

DRUG: Ezetimibe

DRUG: Placebo (for Alirocumab)

DRUG: Placebo (for Ezetimibe)

 Sanofi/Regeneron Jul-12 Jul-13
NCT01663402 ODYSSEY Outcomes ODYSSEY Outcomes: Evaluation of Cardiovascular Outcomes After an Acute Coronary Syndrome During Treatment With Alirocumab 3 COMPLETED Atherosclerotic Cardiovascular Disease

DRUG: Alirocumab

DRUG: Placebo

DRUG: LMT

 Sanofi/Regeneron Oct-12 Jan-18
NCT01709500 ODYSSEY FH II Study of Alirocumab (REGN727/SAR236553) in Patients With heFH (Heterozygous Familial Hypercholesterolemia) Who Are Not Adequately Controlled With Their LMT (Lipid-Modifying Therapy) 3 COMPLETED Heterozygous Familial Hypercholesterolemia

DRUG: LMT (atorvastatin, simvastatin, or rosuvastatin)

DRUG: alirocumab

DRUG: Placebo

 Regeneron/Sanofi ######### May-14
NCT01709513 ODYSSEY ALTERNATIVE Study of Alirocumab (REGN727/SAR236553) in Patients With Primary Hypercholesterolemia and Moderate, High, or Very High Cardiovascular (CV) Risk, Who Are Intolerant to Statins (ODYSSEY ALTERNATIVE) 3 COMPLETED Hypercholesterolemia

DRUG: Atorvastatin

DRUG: Ezetimibe

DRUG: Alirocumab

DRUG: Placebo

 Regeneron/Sanofi Sep-12 May-14
NCT01730040 ODYSSEY OPTIONS I Study of the Efficacy and Safety of Alirocumab (REGN727/SAR236553) in Combination With Other Lipid-modifying Treatment (LMT) (ODYSSEY OPTIONS I) 3 COMPLETED Hypercholesterolemia

DRUG: Alirocumab

DRUG: Atorvastatin

DRUG: Ezetimibe

DRUG: Rosuvastatin

DRUG: Placebo

 Regeneron/Sanofi Oct-12 Apr-14
NCT01730053 ODYSSEY OPTIONS II Study of Alirocumab (REGN727/SAR236553) added-on to Rosuvastatin Versus Other Lipid Modifying Treatments (LMT) (ODYSSEY OPTIONS II) 3 COMPLETED Hypercholesterolemia

DRUG: Alirocumab

DRUG: Rosuvastatin

DRUG: Ezetimibe

DRUG: Placebo

 Regeneron/Sanofi Nov-12 Apr-14
NCT01926782 ODYSSEY CHOICE 1 Study to Evaluate the Efficacy and Safety of an Every Four Weeks Treatment Regimen of Alirocumab (REGN727/ SAR236553) in Patients With Primary Hypercholesterolemia (ODYSSEY CHOICE 1) 3 COMPLETED Hypercholesterolemia

DRUG: Placebo (for alirocumab)

DRUG: Alirocumab

DRUG: Statin

 Regeneron/Sanofi Sep-13 Sep-14
NCT01954394 ODYSSEY OLE Open Label Study of Long Term Safety Evaluation of Alirocumab 3 COMPLETED Hypercholesterolemia

DRUG: Alirocumab

DRUG: Lipid-Modifying Therapy (LMT)

 Sanofi/Regeneron Dec-13 Jun-17
NCT02023879 ODYSSEY CHOICE II Phase III Study To Evaluate Alirocumab in Patients With Hypercholesterolemia Not Treated With a Statin (ODYSSEY CHOICE II) 3 COMPLETED Hypercholesterolemia

DRUG: Alirocumab

DRUG: Placebo (for Alirocumab)

DRUG: Non-statin LMT

OTHER: Diet Alone

 Sanofi/Regeneron Dec-13 Oct-14
NCT02107898 ODYSSEY JAPAN Efficacy and Safety Evaluation of Alirocumab in Patients With Heterozygous Familial Hypercholesterolemia or High Cardiovascular Risk Patients With Hypercholesterolemia on Lipid Modifying Therapy (ODYSSEY JAPAN) 3 COMPLETED Hypercholesterolemia

DRUG: Placebo (for alirocumab)

DRUG: Alirocumab

DRUG: Lipid-Modifying Therapy (LMT)

 Sanofi/Regeneron Mar-14 Jan-15
NCT02289963 n/a Evaluation of Alirocumab in Addition to Lipid-Modifying Therapy in Patients With High Cardiovascular Risk and Hypercholesterolemia in South Korea and Taiwan 3 COMPLETED Hypercholesterolemia

DRUG: Placebo (for Alirocumab)

DRUG: Alirocumab

DRUG: Lipid-Modifying Therapy (LMT)

 Sanofi/Regeneron Jan-15 Apr-16
NCT02326220 ODYSSEY ESCAPE Study of Alirocumab (REGN727/SAR236553) in Patients With Heterozygous Familial Hypercholesterolemia (HeFH) Undergoing Low-density Lipoprotein (LDL) Apheresis Therapy 3 COMPLETED Heterozygous Familial Hypercholesterolemia

DRUG: Placebo

DRUG: Alirocumab

 Regeneron/Sanofi Mar-15 Jan-16
NCT02476006 ODYSSEY APPRISE Safety, Tolerability, and Effect of Alirocumab in High Cardiovascular Risk Patients With Severe Hypercholesterolemia Not Adequately Controlled With Conventional Lipid-modifying Therapies (ODYSSEY APPRISE) 3 COMPLETED Hypercholesterolemia

DRUG: ALIROCUMAB SAR236553 (REGN727)

DRUG: placebo (for injection training only)

DRUG: ezetimibe

DRUG: atorvastatin

DRUG: rosuvastatin

DRUG: simvastatin

 Sanofi/Regeneron Jun-15 Apr-19
NCT02584504 ODYSSEY-NIPPON Efficacy and Safety of Alirocumab in Patients With Hypercholesterolemia Not Adequately Controlled With Non-statin Lipid Modifying Therapy or the Lowest Strength of Statin 3 COMPLETED Hypercholesterolemia

DRUG: Alirocumab

DRUG: Placebo

DRUG: Atorvastatin

DRUG: Non-statin Lipid-Modifying Therapy

OTHER: Diet Alone

 Sanofi/Regeneron Nov-15 Apr-17
NCT02585778 ODYSSEY DM - Insulin Efficacy and Safety of Alirocumab Versus Placebo on Top of Maximally Tolerated Lipid Lowering Therapy in Patients With Hypercholesterolemia Who Have Type 1 or Type 2 Diabetes and Are Treated With Insulin (ODYSSEY DM - Insulin) 3 COMPLETED Hypercholesterolemia

DRUG: Alirocumab

DRUG: Placebo

DRUG: Lipid-Modifying Therapy (LMT)

DRUG: Antihyperglycemic Drug

 Sanofi/Regeneron Oct-15 Apr-17
NCT02642159 ODYSSEY DM-Dyslipidemia Efficacy and Safety of Alirocumab Versus Usual Care on Top of Maximally Tolerated Statin Therapy in Patients With Type 2 Diabetes and Mixed Dyslipidemia (ODYSSEY DM-Dyslipidemia) 4 COMPLETED Dyslipidemia

DRUG: Alirocumab

DRUG: Statins

DRUG: Ezetimibe

DRUG: Fenofibrate

DRUG: Nicotinic acid

DRUG: Omega-3 fatty acids

DRUG: Antihyperglycemic Drug

 Sanofi/Regeneron Mar-16 Mar-17
NCT02715726 ODYSSEY EAST Evaluation of Alirocumab Versus Ezetimibe on Top of Statin in Asia in High Cardiovascular Risk Patients With Hypercholesterolemia 3 COMPLETED Hypercholesterolemia

DRUG: Alirocumab

DRUG: Placebo for alirocumab

DRUG: ezetimibe

DRUG: placebo for ezetimibe

DRUG: atorvastatin

DRUG: rosuvastatin

DRUG: simvastatin

 Sanofi/Regeneron Jul-16 Aug-18
NCT02957682 n/a Evaluating Effect of the Study Drug Praluent (Alirocumab) on Neurocognitive Function When Compared to Placebo 4 COMPLETED Hypercholesterolemia

DRUG: Praluent (Alirocumab)

DRUG: Placebo

 Regeneron/Sanofi Nov-16 Mar-20
NCT02984982 ODYSSEY J-IVUS Evaluation of Effect of Alirocumab on Coronary Atheroma Volume in Japanese Patients Hospitalized for Acute Coronary Syndrome With Hypercholesterolemia 4 COMPLETED

Hypercholesterolemia

Acute Coronary Syndrome

DRUG: Alirocumab SAR236553

DRUG: Atorvastatin

DRUG: Rosuvastatin

DRUG: Fenofibrate

DRUG: Bezafibrate

DRUG: Ezetimibe

DRUG: Antiplatelets

DRUG: Anticoagulants

 Sanofi/Regeneron Nov-16 Jul-18
NCT03156621 ODYSSEY HoFH Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH) 3 COMPLETED Homozygous Familial Hypercholesterolemia

DRUG: Alirocumab

DRUG: Placebo

 Regeneron/Sanofi Oct-17 Sep-19
NCT03415178 n/a Usability Study of the Commercial Auto-injector Device and the New Auto-injector Device (SYDNEY) in Patients With High or Very High CV Risk With Hypercholesterolemia Not Adequately Controlled With Their Lipid-Modifying Therapy 3 COMPLETED Hypercholesterolemia

DRUG: Alirocumab SAR236553

DEVICE: Current auto-injector device (AI)

DEVICE: New auto-injector device (SYDNEY)

DRUG: Atorvastatin

DRUG: Rosuvastatin

 Sanofi/Regeneron Mar-18 Aug-18
NCT03510715 n/a An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia 3 COMPLETED Hypercholesterolemia

DRUG: Alirocumab SAR236553 (REGN727)

DRUG: Atorvastatin

DRUG: Simvastatin

DRUG: Fluvastatin

DRUG: Pravastatin

DRUG: Lovastatin

DRUG: Rosuvastatin

DRUG: Ezetimibe

DRUG: Cholestyramine

DRUG: Nicotinic acid

DRUG: Fenofibrate

DRUG: Omega-3 fatty acids

 Sanofi/Regeneron Aug-18 Feb-20
NCT03510884 n/a An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia 3 COMPLETED Hypercholesterolemia

DRUG: Alirocumab SAR236553 (REGN727)

DRUG: Rosuvastatin

DRUG: Atorvastatin

DRUG: Simvastatin

DRUG: Pravastatin

DRUG: Lovastatin

DRUG: Fluvastatin

DRUG: Ezetimibe

DRUG: Cholestyramine

DRUG: Nicotinic acid

DRUG: Fenofibrate

DRUG: Omega-3 fatty acids

DRUG: Placebo

 Sanofi/Regeneron May-18 Jan-21

Inclisiran

A PCSK9-specific siRNA approved by EMA in 2020, and the US FDA in 2021

 NCT02314442 n/a A Phase 1 Study of an Investigational Drug, ALN-PCSSC, in Subjects With Elevated Low Density Lipoprotein Cholesterol (LDL-C) 1 COMPLETED Hypercholesterolemia

DRUG: ALN-PCSSC

DRUG: Sterile Normal Saline (0.9% NaCl)

 Alnylam Pharmaceuticals Dec-14 May-15
NCT02597127 ORION-1 Trial to Evaluate the Effect of ALN-PCSSC Treatment on Low Density Lipoprotein Cholesterol (LDL-C) 2 COMPLETED

Atherosclerotic Cardiovascular Disease

Familial Hypercholesterolemia

Diabetes

DRUG: ALN-PCSSC

DRUG: Normal Saline

 The Medicines Company Jan-16 Jun-17
NCT02963311 ORION-2 A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH) 2 COMPLETED Homozygous Familial Hypercholesterolemia

DRUG: ALN-PCSSC

DRUG: Standard of Care

 The Medicines Company Dec-16 Oct-18
NCT03060577 ORION-3 An Extension Trial of Inclisiran in Participants With Cardiovascular Disease and High Cholesterol 2 COMPLETED

Atherosclerotic Cardiovascular Disease

Symptomatic Atherosclerosis

Type2 Diabetes

Familial Hypercholesterolemia

DRUG: Inclisiran

DRUG: Evolocumab

 Novartis Pharmaceuticals Apr-17 Dec-21
NCT03159416 ORION-7 A Study of Inclisiran in Participants With Renal Impairment Compared to Participants With Normal Renal Function (ORION-7) 1 COMPLETED Renal Impairment DRUG: Inclisiran The Medicines Company Jun-17 Mar-18
NCT03397121 ORION-9 Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH) 3 COMPLETED

Heterozygous Familial Hypercholesterolemia

Elevated Cholesterol

DRUG: Inclisiran

DRUG: Placebo

 The Medicines Company Nov-17 Aug-19
NCT03399370 ORION-10 Inclisiran for Participants With Atherosclerotic Cardiovascular Disease and Elevated Low-density Lipoprotein Cholesterol 3 COMPLETED

ASCVD

Elevated Cholesterol

DRUG: Inclisiran Sodium

DRUG: Placebo

 The Medicines Company Dec-17 Sep-19
NCT03400800 ORION-11 Inclisiran for Subjects With ASCVD or ASCVD-Risk Equivalents and Elevated Low-density Lipoprotein Cholesterol 3 COMPLETED

ASCVD

Risk Factor, Cardiovascular

Elevated Cholesterol

DRUG: Inclisiran Sodium

DRUG: Placebo

 The Medicines Company Nov-17 Jul-19
NCT03705234 ORION-4 A Randomized Trial Assessing the Effects of Inclisiran on Clinical Outcomes Among People With Cardiovascular Disease 3 ACTIVE_NOT_RECRUITING Atherosclerotic Cardiovascular Disease

DRUG: Inclisiran

DRUG: Placebo

 University of Oxford Oct-18 Jul-26
NCT03814187 ORION-8 Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C 3 COMPLETED

ASCVD

Elevated Cholesterol

Heterozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia

 DRUG: Inclisiran Sodium Novartis Pharmaceuticals Apr-19 Feb-23
NCT03851705 ORION-5 A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH) 3 COMPLETED Homozygous Familial Hypercholesterolemia

DRUG: Inclisiran Sodium for injection

DRUG: Placebo

DRUG: Placebos

 Novartis Pharmaceuticals Feb-19 Mar-20
NCT04652726 ORION-16 Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia 3 ACTIVE_NOT_RECRUITING Familial Hypercholesterolemia - Heterozygous

DRUG: Inclisiran

DRUG: Placebo

 Novartis Pharmaceuticals Jan-21 Nov-23
NCT04659863 ORION-13 Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia 3 ACTIVE_NOT_RECRUITING Familial Hypercholesterolemia - Homozygous

DRUG: Inclisiran

DRUG: Placebo

 Novartis Pharmaceuticals Feb-21 Nov-23
NCT04666298 ORION-15 Study of Efficacy and Safety of Inclisiran in Japanese Participants With High Cardiovascular Risk and Elevated LDL-C 2 COMPLETED

Hypercholesterolemia

Heterozygous Familial Hypercholesterolemia

DRUG: Inclisiran sodium

DRUG: Placebo

 Novartis Pharmaceuticals Jan-21 Apr-22
NCT04765657 n/a Study of Efficacy and Safety of Inclisiran in Asian Participants With Atherosclerotic Cardiovascular Disease (ASCVD) or ASCVD High Risk and Elevated Low Density Lipoprotein Cholesterol (LDL-C) 3 ACTIVE_NOT_RECRUITING Hypercholesterolemia

DRUG: inclisiran sodium

DRUG: Placebo

 Novartis Pharmaceuticals Mar-21 Jun-22
NCT04774003 ORION-14 Study of Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inclisiran in Chinese Participants With Elevated Serum LDL-C 1 COMPLETED Hyperlipidemia

DRUG: 100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran)

DRUG: Placebo

DRUG: 300 mg inclisiran sodium (equivalent to 284 mg inclisiran)

 Novartis Pharmaceuticals Feb-21 Oct-21
NCT04807400 SPIRIT Study in Primary Care Evaluating Inclisiran Delivery Implementation + Enhanced Support 3 COMPLETED

Atherosclerotic Cardiovascular Disease

Atherosclerotic Cardiovascular Disease Risk Equivelents

Elevated Low Density Lipoprotein Cholesterol

DRUG: Inclisiran

BEHAVIORAL: Behavioral Support

 Novartis Pharmaceuticals Jul-21 Jan-23
NCT04873934 VICTORION-INCEPTION Management of LDL-cholesterol With Inclisiran + Usual Care Compared to Usual Care Alone in Participants With a Recent Acute Coronary Syndrome 3 RECRUITING Acute Coronary Syndrome DRUG: Inclisiran Novartis Pharmaceuticals Jun-21 Aug-24
NCT04929249 VICTORION-INITIATE A Randomized Study to Evaluate the Effect of an “Inclisiran First” Implementation Strategy Compared to Usual Care in Patients With Atherosclerotic Cardiovascular Disease and Elevated LDL-C Despite Receiving Maximally Tolerated Statin Therapy (VICTORION-INITIATE) 3 ACTIVE_NOT_RECRUITING Atherosclerotic Cardiovascular Disease DRUG: Inclisiran Novartis Pharmaceuticals Jun-21 Sep-23
NCT05030428 VICTORION-2PREVENT Study of Inclisiran to Prevent Cardiovascular (CV) Events in Participants With Established Cardiovascular Disease 3 RECRUITING Atherosclerotic Cardiovascular Disease

DRUG: Inclisiran sodium 300 mg

DRUG: Placebo

 Novartis Pharmaceuticals Nov-21 Oct-27
NCT05192941 VICTORION-DIFFERENCE Study of Efficacy, Safety, Tolerability and Quality of Life of Inclisiran (KJX839) vs Placebo, on Top of Ongoing Individually Optimized Lipid-lowering Therapy, in Participants With Hypercholesterolemia 4 RECRUITING Hypercholesterolemia

DRUG: Inclisiran Sodium

DRUG: Placebo

 Novartis Pharmaceuticals Apr-22 Feb-25
NCT05360446 VICTORION-PLAQUE Coronary Computed Tomography Study to Assess the Effect of Inclisiran in Addition to Maximally Tolerated Statin Therapy on Atherosclerotic Plaque Progression in Participants With a Diagnosis of Non-obstructive Coronary Artery Disease Without Previous Cardiovascular Events 3 RECRUITING Coronary Artery Disease

DRUG: Inclisiran sodium 300 mg

DRUG: Placebo

 Novartis Pharmaceuticals Jul-22 Jan-26
NCT05399992 VICTORION-REAL Study Evaluating Effectiveness and Adherence of Inclisiran Plus Standard of Care (SoC) Lipid-lowering Therapy Compared to SoC in ASCVD n/a RECRUITING

Primary Hypercholesterolemia

Mixed Dyslipidemia

 OTHER: Inclisiran Novartis Pharmaceuticals Sep-22 Apr-27
NCT05682378 VICTORION-PEDS-OLE Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Adolescent ORION-16 or ORION-13 Studies 3 RECRUITING Heterozygous or Homozygous Familial Hypercholesterolemia DRUG: Inclisiran Novartis Pharmaceuticals Feb-23 Dec-27
NCT05739383 n/a A Study of Inclisiran to Prevent Cardiovascular Events in High-risk Primary Prevention Patients. 3 RECRUITING Primary Prevention of Atherosclerotic Cardiovascular Disease

DRUG: Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL

DRUG: Placebo in 1.5 ml

 Novartis Pharmaceuticals Mar-23 Apr-29
NCT05763875 VICTORION-Mono Efficacy and Safety of Inclisiran as Monotherapy in Patients With Primary Hypercholesterolemia Not Receiving Lipid-lowering Therapy. 3 RECRUITING Hypercholesterolemia

DRUG: Inclisiran

DRUG: Ezetimibe

DRUG: Matching Placebo for Inclisiran

DRUG: Matching Placebo for Ezetimibe

 Novartis Pharmaceuticals Mar-23 Aug-24
NCT05888103 VICTORION-Mono China Efficacy and Safety of Inclisiran as Monotherapy in Chinese Adults With Low or Moderate ASCVD Risk and Elevated Low-density Lipoprotein Cholesterol. 3 NOT_YET_RECRUITING Primary Hypercholesterolemia or Mixed Dyslipidemia

DRUG: Inclisiran

DRUG: Matching Placebo for Inclisiran

 Novartis Pharmaceuticals Jul-23 Apr-24

Tafolecimab (IBI306)

Anti-PCSK9 mAb

Approved by China’s NMPA in 2023

 NCT03366688 n/a Single Ascending Dose Study of PCSK-9 Inhibitor (IBI306) in Healthy Subjects. 1 COMPLETED Hypercholesterolemia DRUG: IBI306 | DRUG: placebo Innovent Biologics (Suzhou) Co. Ltd. Nov-17 Nov-18
NCT03815812 n/a Multiple Ascending Dose Study of PCSK-9 Inhibitor (IBI306) in Chinese Patients With Hypercholesterolemia 2 COMPLETED Hypercholesterolemia DRUG: IBI306 | DRUG: placebo Innovent Biologics (Suzhou) Co. Ltd. Mar-19 Dec-19
NCT04031742 n/a A Study to Evaluate Safety and Efficacy of IBI306, a PCSK9 Monoclonal Antibody in Chinese Subjects With Homozygous Familial Hypercholesterolemia 2/3 COMPLETED Homozygous Familial Hypercholesterolemia BIOLOGICAL: IBI306 | BIOLOGICAL: IBI306 Innovent Biologics (Suzhou) Co. Ltd. Sep-19 Dec-21
NCT04179669 CREDIT-2 Safety and Efficacy of IBI306 in HeFH Patients 3 COMPLETED Heterozygous Familial Hypercholesterolemia DRUG: IBI306 | DRUG: placebo Innovent Biologics (Suzhou) Co. Ltd. Dec-19 Jun-21
NCT04289285 CREDIT-1 Safety and Efficacy of IBI306 in Chinese Subjects With Non-familial Hypercholesterolemia 3 COMPLETED Hypercholesterolemia DRUG: IBI306 450 mg SC Q4W | DRUG: Placebo SC Q4W | DRUG: IBI306 600 mg SC Q6W | OTHER: Placebo SC Q6W Innovent Biologics (Suzhou) Co. Ltd. Apr-20 Feb-22
NCT04709536 CREDIT-4 A Study of IBI306 in Participants With Hypercholesterolemia 3 UNKNOWN Hypercholesterolemia DRUG: IBI306 | DRUG: Placebo Innovent Biologics (Suzhou) Co. Ltd. Feb-21 May-21
NCT04759534 n/a Application of PCSK9 Inhibitors in Patients With Heterozygous Familial Hypercholesterolemia 3 UNKNOWN Efficacy and Safety|Heterozygous Familial Hypercholesterolemia|PCSK9 BIOLOGICAL: protein convertase subtilisin/kexin type 9 inhibitor Shenzhen People’s Hospital Sep-20 Oct-21
NCT04948008 n/a Evaluate the Efficacy and Safety of IBI306 in Subjects With Homozygous Familial Hypercholesterolemia 2/3 UNKNOWN Familial Hypercholesterolemia - Homozygous|Lipid Metabolism Disorders|Proprotein Convertase Subtilisin/Kexin 9 BIOLOGICAL: IBI306 Shenzhen People’s Hospital Nov-19 Jul-20
NCT05792917 n/a Bioequivalence Study of Tafolecimab Injections in Chinese Healthy Male Volunteers 1 COMPLETED Healthy Male Subjects DRUG: tafolecimab (a modified manufacturing process)|DRUG: tafolecimab (a original manufacturing process) Innovent Biologics (Suzhou) Co. Ltd. Mar-23 Mar-23
PCSK9-iT for other disorders NCT Number Study Title Phases Study Status Conditions Interventions Sponsor Start Date Primary Completion Date
Infection NCT02833844 Safety, Tolerability and Efficacy on Low Density Lipoprotein Cholesterol (LDL-C) of Evolocumab in Participants With Human Immunodeficiency Virus (HIV) and Hyperlipidemia/Mixed Dyslipidemia 3 COMPLETED Subjects With Hyperlipidemia, Dyslipidemia and HIV Infection

DRUG: Evolocumab

DRUG: Placebo

 Amgen May-17 Jul-19
NCT03139630 COPANA - A09 PCSK 9 Substudy: Impact of Protease Inhibitors on PCSK9 Levels in Naive HIV-Infected Patients n/a COMPLETED

HIV Seropositivity

Dyslipidemias

PCSK9

 n/a Franck Boccara Mar-16 Sep-16
NCT03207945 Effect of PCSK9 Inhibition on Cardiovascular Risk in Treated HIV Infection (EPIC-HIV Study) 3 RECRUITING

Dyslipidemias

Cardiovascular Diseases

HIV Infections

DRUG: Alirocumab

OTHER: Placebo

 University of California, San Francisco Apr-18 Jul-24
NCT03500302 Effect of Evolocumab on Coronary Endothelial Function 2 COMPLETED

Human Immunodeficiency Virus

Coronary Artery Disease

 DRUG: Evolocumab Johns Hopkins University May-18 Nov-19
NCT03634293 Treatment of Severe Infection With Antihyperlipidemia Drug 2/3 UNKNOWN

Sepsis

Septic Shock

DRUG: Alirocumab Injectable Product

DRUG: Saline Solution

 Wolfson Medical Center Jan-19 Jan-21
NCT03869073 Evolocumab for PCSK9 Lowering in Early Acute Sepsis (The PLEASe Study) 2 UNKNOWN Sepsis

DRUG: Evolocumab

DRUG: Placebo

 University of British Columbia Feb-19 Feb-21
NCT04941105 Impact of PCSK9 Inhibition on Clinical Outcome in Patients During the Inflammatory Stage of the COVID-19 3 COMPLETED Sars-CoV-2 Infection

DRUG: Evolocumab

DRUG: Saline solution

 Collegium Medicum w Bydgoszczy Jun-21 May-22
NCT05469347 Alirocumab in Patients With Sepsis 1 RECRUITING Sepsis

DRUG: Alirocumab

DRUG: Placebo

 Jonathan Sevransky Jan-23 Apr-24
Autoimmune disorder NCT05191342 Proprotein Convertase Subtilisin Kexin 9 in Rheumatoid Arthritis n/a RECRUITING PCSK9 DIAGNOSTIC_TEST: Enzyme-linked immunosorbent assay for PCSK9 First Affiliated Hospital of Harbin Medical University Nov-21 Nov-22
Alcohol use disorder (AUD) NCT04781322 Safety, Tolerability, and Bioeffects of Alirocumab in Non-treatment Seeking Heavy Drinkers 1 RECRUITING

Alcohol Associated Liver Disease

Heavy Drinking Behavior

DRUG: Alirocumab

OTHER: Placebo

 National Institute on Alcohol Abuse and Alcoholism (NIAAA) Oct-21 Dec-23
Cancer NCT03337698 A Study Of Multiple Immunotherapy-Based Treatment Combinations In Participants With Metastatic Non-Small Cell Lung Cancer (Morpheus- Non-Small Cell Lung Cancer) 1/2 RECRUITING Carcinoma, Non-Small-Cell Lung

DRUG: Atezolizumab

DRUG: Cobimetinib

DRUG: RO6958688

DRUG: Docetaxel

DRUG: CPI-444

DRUG: Pemetrexed

DRUG: Carboplatin

DRUG: Gemcitabine

DRUG: Linagliptin

DRUG: Tocilizumab

DRUG: Ipatasertib

DRUG: Bevacizumab

DRUG: Sacituzumab Govitecan

OTHER: Radiation

DRUG: Evolocumab

DRUG: Tiragolumab

DRUG: XL092

DRUG: Camonsertib

 Hoffmann-La Roche Jan-18 Aug-25
NCT04862260 Cholesterol Disruption in Combination With FOLFIRINOX in Patients With Advanced Pancreatic Adenocarcinoma early 1 RECRUITING

Pancreatic Ductal Adenocarcinoma

Pancreatic Cancer

Pancreas Cancer

Metastatic Cancer

 DRUG: Cholesterol metabolism disruption CHU de Quebec-Universite Laval Oct-21 Jan-25
NCT04937413 The PCSK9i Inhibitor Evolocumab - a Surgical Trial of Pharamcodynamics and Kinetics Evaluation early 1 RECRUITING

Malignant Glioma

Glioblastoma

 DRUG: Evolocumab Duke University Oct-21 Jun-25
NCT05128539 A Study Explore JS001 + JS002 in Patients With Advanced Cancer 1 RECRUITING Advanced Cancer DRUG: JS001(Toripalimab)+JS002 Shanghai Junshi Bioscience Co., Ltd. Dec-21 Feb-24
NCT05144529 A Randomized Pilot Study of Evolocumab Plus Nivolumab/Ipilimumab in Treatment-Na??ve Patients With Metastatic NSCLC 2 RECRUITING Lung Cancer Metastatic

DRUG: Nivolumab

DRUG: Ipilimumab

DRUG: Evolocumab

 Scott Antonia Mar-22 Dec-23
NCT05553834 PCSK9 Inhibitor and PD-1 Inhibitor in Patients With Metastatic, Refractory To Prior Anti PD-1 Non-small Cell Lung 2 RECRUITING Non-small Cell Lung Cancer (NSCLC) COMBINATION_PRODUCT: Alirocumab and Cemiplimab Duke University May-23 Jan-27
NCT05976893 Study on the Composite Endpoint Event of PCSK9 Inhibitor in Patients With Very High Risk of ASCVD and Cancer 4 NOT_YET_RECRUITING ASCVD|Cancer

DRUG: Evolocumab

DRUG: Statin

 Xiang Xie Aug-23 Dec-25