N/A N=5 Gene Analysis and Treatment Optimization in Chinese Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia
Primary: Number of LDLR Gene Mutations — 7 gene mutations
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
N/A N=11 Familial Hyperlipidemia Family Registry
Familial Hyperlipidemia
Primary: Familial Hypercholesterolemia (FH) Determination for Family Members of School Children With Elevated Cholesterol and Documented FH Variant — 3; 8 participants
Phase 2 N=168 Reduction of Low-Density Lipoprotein Cholesterol (LDL-C) With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder Study
Hypercholesterolemia, Familial
Primary: Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12 — 1.12; -42.70; -55.24 percent change — p=<0.001
Phase 3 N=300 Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders
Severe Familial Hypercholesterolemia
Primary: Number of Participants With Adverse Events — 94; 174; 76; 143 Participants
Phase 3 N=58 Trial Evaluating PCSK9 Antibody in Subjects With LDL Receptor Abnormalities
Homozygous Familial Hypercholesterolemia
Primary: Part A: Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 12 — -16.5 percent change
Phase 3 N=309 A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol
Hypercholesterolemia · Heterozygous Familial
Primary: Percent Change From Baseline To Primary Endpoint Visit (PET) In LDL-C In Cohort 1 — -27.17; -6.77; -22.96; -10.62 Percent
Phase 3 N=51 Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
Lipid Metabolism, Inborn Errors · Hypercholesterolemia, Autosomal Dominant · Hyperlipidemias
Primary: Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Primary Efficacy Time Point — -3.31; -24.66 percentage of baseline — p=<0.001
Phase 2 N=9 Study of REGN1500 in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
Homozygous Familial Hypercholesterolemia
Primary: Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 0) to Week 4 in the Main Study Period — -49.17 Percent Change
Phase 3 N=331 Reduction of LDL-C With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder Study-2
Hyperlipidemia
Primary: Percent Change From Baseline in LDL-C at Week 12 — -2.02; 5.53; -61.25; -55.74 percent change — p=<0.001
Phase 2 N=266 Study of Evinacumab (REGN1500) in Participants With Persistent Hypercholesterolemia
Hypercholesterolemia
Primary: Percent Change From Baseline in Calculated Low Density Lipoprotein Cholesterol (LDL-C) at Week 16 (Intent-to-Treat [ITT] Estimand) — 8.8; -29.7; -44.0; -47.2 Percent…
Phase 3 N=370 A 52 Week Study To Assess The Use Of Bococizumab (PF-04950615; RN316) In Subjects With Heterozygous Familial Hypercholesterolemia
Heterozygous Familial Hypercholesterolemia
Primary: Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 12 — -0.3; -54.2 percent change — p=<0.001
Phase 3 N=29 A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)
Homozygous Familial Hypercholesterolemia
Primary: Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) — -40.1 Percent Change
Phase 2 N=116 Study of MGL-3196 in Patients With Heterozygous Familial Hypercholesterolemia (HeFH)
Heterozygous Familial Hypercholesterolemia
Primary: Mean Percent Change in LDL-C From Baseline To Week 12 — -10.6; 8.2 percent change — p=<0.0001
Phase 3 N=65 Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia
Primary: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 (Intent-to-Treat [ITT] Estimand) — 1.9; -47.1 Percent Change — p=< 0.0001
Phase 3 N=142 An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia
Lipid Metabolism, Inborn Errors · Hypercholesterolemia, Autosomal Dominant · Hyperlipidemias
Primary: Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) — -28.5; -27; -27.3; -27.9 percent change
Phase 2 N=21 Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia
Lipid Metabolism, Inborn Errors · Hypercholesterolemia, Autosomal Dominant · Hyperlipidemias
Primary: Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) — -16.2; -2.6; -32.4; 30.6 percentage of baseline
Phase 2 N=77 Study of the Safety and Efficacy of REGN727/SAR236553 in Patients With HeFH Hypercholesterolemia
Hypercholesterolemia
Primary: Percent Change From Baseline in Calculated LDL-C at Week 12 - On-treatment Analysis — -10.7; -28.9; -31.5; -42.5 percent change — p=0.0000
Phase 2 N=3 Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)
Familial Chylomicronemia Syndrome · Lipoprotein Lipase Deficiency · Hyperlipoproteinemia Type 1
Primary: Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG) — -550.00 mg/dL
Phase 1 N=39 8-Week PK/PD Atorvastatin Study In Children And Adolescents With Heterozygous Familial Hypercholesterolemia
Pediatric Heterozygous Hypercholesterolemia
Primary: Parent-metabolite Population Pharmacokinetic (PK) Model for Atorvastatin and Its Metabolites: Atorvastatin Apparent Clearance (CL/F) — 553; 543 L/hr
Phase 3 N=407 Study of Obicetrapib & Ezetimibe Fixed Dose Combination on Top of Maximum Tolerated Lipid-Modifying Therapies
Dyslipidemias · Hypercholesterolemia · Familial Hypercholesterolemia
Primary: Effect of Fixed-Dose Combination (FDC) Compared to Placebo on LDL-C — -45.58; 3.03 percent change from baseline — p=<.0001
Phase 3 N=303 A Study of Enlicitide Decanoate (MK-0616 Oral PCSK9 Inhibitor) in Adults With Heterozygous Familial Hypercholesterolemia (MK-0616-017/CORALreef HeFH)
Hypercholesterolemia · Familial Hypercholesterolemia
Primary: Mean Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 — -58.2; 2.6 Percent Change — p=<0.001
Phase 3 N=124 Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease
Heterozygous Familial Hypercholesterolemia · Coronary Artery Disease
Primary: Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point — 5.17; -28.02 percentage of Baseline — p=<0.001
Phase 3 N=163 Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia)
Familial Hypercholesterolemia
Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 36; 69; 7; 25 Participants
N/A N=8 Acute Microvascular Changes With LDL Apheresis
Hyperlipidemia
Primary: Myocardial Perfusion at Rest — 55; 135 IU/s
Phase 2 N=36 Safety Study of AEM-28 to Treat Refractory Hypercholesterolemia
Hypercholesterolemia · Hyperlipoproteinemia Type II
Primary: Number of Participants Who Incurred at Least One Treatment Emergent Event — 33; 10 participants
Phase 3 N=45 A Randomized, Double-blind, Placebo Controlled Study to Assess Efficacy, Safety and Tolerability of LCQ908 in Subjects With Familial Chylomicronemia Syndrome
Familial Chylomicronemia Syndrome (FCS)
Primary: Percent Change in Fasting Triglycerides From Baseline to 12 Weeks — 45.6; 3.7; -13.9 percent change — p=0.0538
Phase 3 N=249 Study of Alirocumab (REGN727/SAR236553) in Patients With heFH (Heterozygous Familial Hypercholesterolemia) Who Are Not Adequately Controlled With Their LMT (Lipid-Modifying Therapy)
Heterozygous Familial Hypercholesterolemia
Primary: Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent--to--Treat (ITT) Analysis — -48.7; 2.8 percent change — p=<0.0001
Phase 3 N=56 A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
Homozygous Familial Hypercholesterolemia
Primary: Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 150 — 2.39; 0.70 Percentage change — p=0.9047
Phase 2 N=619 Phase 2 Dose-Ranging Efficacy and Safety Trial of SCH 900271 in Participants With Primary Hypercholesterolemia or Mixed Hyperlipidemia (P05675)
Primary Hypercholesterolemia · Mixed Hyperlipidemia
Primary: Change From Baseline in Direct LDL-C at Week 8 — -2.0; -1.5; -4.2; 0.2 Percent change — p=0.06
Phase 2 N=9 A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
Homozygous Familial Hypercholesterolemia
Primary: Percentage Change From Day 1 to Day 90 in LDL-C — -12.26 percent change