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. 2012 May 31;7:29–38. doi: 10.2147/CE.S25239

Table 3.

Phase III studies evaluating mipomersen

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.