Table 3.
Study | Patients | Design | Outcome | Key results | Adverse events |
---|---|---|---|---|---|
Raal et al36 | Homozygous FH on stable low-fat diet and maximum lipid-lowering drugs n = 51 |
Randomized, placebo-controlled; mipomersen 200 mg/week for 26 weeks |
Primary outcome LDL-C reduction Secondary outcome Change in apoB, total cholesterol, HDL-C, lipoprotein(a), triglycerides |
Placebo LDL-C −3.3% Lp(a) −7.9% ApoB100 −2.5% Mipomersen LDL-C −24.7%; P = 0.0003 Lp(a) −31.1%; P = 0.0013 ApoB100 −26.8%; P = 0.0001 |
Injection site reactions Placebo 24% Mipomersen 70% Flu-like symptoms Placebo 24% Mipomersen 29% Elevated transaminases (≥3 × ULN) Placebo 0% Mipomersen 12% |
Stein et al37 (abstract) | Heterozygous FH and cardiovascular disease on low-fat diet and stable lipid-lowering drugs n = 124 |
Randomized, placebo-controlled; mipomersen 200 mg/week for 26 weeks |
Primary outcome LDL-C reduction Secondary outcome Change in apoB, total cholesterol, HDL-C, lipoprotein(a), triglycerides |
Placebo LDL-C −5.2% Lp(a) 2.4% ApoB100 7.0% Mipomersen LDL-C −34.0%; P = 0.001 Lp(a) −20.4% P = 0.001 ApoB100 −26.3%; P = 0.001 |
Injection site reactions Placebo 42% Mipomersen 93% Flu-like symptoms Placebo 39% Mipomersen 42% Elevated transaminases (≥3 × ULN) Placebo 0% Mipomersen 6% |
Tardif et al38 (abstract) | Severe heterozygous FH and high risk for atherosclerosis on maximally tolerated statin therapy n = 58 |
Randomized, placebo-controlled; mipomersen 200 mg/week for 26 weeks |
Primary outcome LDL-C reduction Secondary outcome Change in apoB, total cholesterol, HDL-C, lipoprotein(a), triglycerides |
Placebo LDL-C 12.5% Mipomersen LDL-C −35.9%; P = 0.001 |
Injection site reactions Placebo 32% Mipomersen 90% Flu-like symptoms Placebo 21% Mipomersen 46% Elevated transaminases (≥3 × ULN) Placebo 0% Mipomersen 15% |
Cromwell et al35 (abstract) | Hypercholesterolemia and high risk for atherosclerosis on low-fat diet and maximally tolerated statin therapy n = 158 |
Randomized, placebo-controlled; mipomersen 200 mg/week for 26 weeks |
Primary outcome LDL-C reduction Secondary outcome Change in apoB, total cholesterol, HDL-C, lipoprotein(a), triglycerides |
Placebo LDL-C −4.5% Mipomersen LDL-C −42.2%; P = 0.001 |
Injection site reaction Placebo 31% Mipomersen 78% Flu-like symptoms Placebo 21% Mipomersen 34% Elevated transaminases (≥3 × ULN) Placebo 0% Mipomersen 14% |
Visser et al39 | High-risk patients in tolerant to statins n = 33 |
Randomized, placebo-controlled; mipomersen 200 mg/week for 26 weeks |
Primary outcome LDL-C reduction Secondary outcome Change in apoB, total cholesterol, HDL-C, lipoprotein(a), triglycerides |
Mipomersen LDL-C −47.3%; P = 0.01 Lp(a) −27.1%; P < 0.001 ApoB −46.2%; P < 0.001 |
Discontinuation Placebo 17% Mipomersen 18% |
Abbreviations: ApoB, apolipoprotein B; FH, familial hypercholesterolemia; LDL-C, low-density lipoprotein cholesterol; HDL-C, high-density lipoprotein cholesterol; Lp(a), lipoprotein(a); TG, triglycerides; ULN, upper limit of normal.