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Phase 3 Completed N=58 Randomized Quadruple-blind Treatment

Safety and Efficacy of Mipomersen in Patients With Severe Hypercholesterolemia on a Maximally Tolerated Lipid-Lowering Regimen and Who Are Not on Apheresis

Source: ClinicalTrials.gov NCT00794664 ↗
Enrolled (actual)
58
Serious AEs
10.3%
Results posted
Mar 2013
Primary outcomePrimary: Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Primary Efficacy Time Point — 12.5; -35.9 percentage of baseline — p=<0.001

Summary

The purpose of the study is to evaluate the safety and efficacy of dosing with mipomersen for 26 weeks in treating severely hypercholesterolemic patients who are on a maximally tolerated lipid-lowering regimen and who are not on apheresis.

Outcome Measures

OutcomeResultp-value
PRIMARY
Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Primary Efficacy Time Point
12.5; -35.9 <0.001 sig
PRIMARY
LDL-C at Baseline and the Primary Efficacy Time Point (PET)
249.4; 276.1; 263.9; 174.9
SECONDARY
Percent Change From Baseline in Apolipoprotein B (Apo-B) at Primary Efficacy Time Point
11.41; -35.88 <0.001 sig
SECONDARY
Apo-B at Baseline and the Primary Efficacy Time Point (PET)
182.8; 202.1; 193.7; 126.8
SECONDARY
Percentage Change From Baseline in Total Cholesterol at Primary Efficacy Time Point (PET)
11.13; -28.31 <0.001 sig
SECONDARY
Total Cholesterol at Baseline and the Primary Efficacy Time Point (PET)
320.6; 356.8; 341.5; 251.5
SECONDARY
Percentage Change From Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Primary Efficacy Time Point (PET)
14.17; -33.95 <0.001 sig
SECONDARY
Non-HDL-C at Baseline and the Primary Efficacy Time Point (PET)
277.5; 305.6; 296.7; 198.1

Eligibility Criteria

Inclusion Criteria

  • Fasting LDL-C ≥200 mg/dL (5.1 mmol/L) at screening and the presence of at least 1 of the following criteria:
  • Myocardial infarction (MI)
  • Percutaneous coronary intervention (PCI) or coronary artery bypass graft (CABG)
  • Coronary artery disease documented by angiography or any other accepted imaging technique
  • Positive exercise test (≥1 mm ST-depression at maximal exercise or test terminated because of angina) or a perfusion defect (e.g., thallium or single photon emission computed tomography)
  • Other clinical atherosclerotic diseases: peripheral artery disease, symptomatic carotid artery disease, abdominal aortic aneurysm
  • Or, if alternative above were not met, fasting LDL-C ≥300 mg/dL (7.8 mmol/L)
  • On stable, maximally tolerated statin therapy for 8 weeks
  • On stable, medication from an additional class of hypolipidemic agents for 8 weeks.
  • On stable, low fat diet for 12 weeks
  • Stable weight for 6 weeks

Exclusion Criteria

  • Significant health problems in the recent past including heart attack, stroke, coronary syndrome, unstable angina, heart failure, significant arrhythmia, hypertension, blood disorders, liver disease, cancer, digestive disorders, Type I diabetes, or uncontrolled Type II diabetes
  • Apheresis within 3 months prior to Screening or expected to start apheresis during the treatment phase
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00794664). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.

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