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Phase 3 N=124 Randomized Quadruple-blind Treatment

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

Enrolled (actual)
124
Serious AEs
6.5%
Results posted
Mar 2013
Primary outcome: 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

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
mipomersen sodium (Drug); placebo (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Kastle Therapeutics, LLC
Primary completion
Dec 2009

Outcome Measures

OutcomeResultp-value
PRIMARY
Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point
5.17; -28.02 <0.001 sig
PRIMARY
LDL Cholesterol at Baseline and at the Primary Efficacy Time Point
142.9; 152.9; 146.4; 103.9
SECONDARY
Percent Change From Baseline in Apolipoprotein B at the Primary Efficacy Time Point
7.02; -26.31 <0.001 sig
SECONDARY
Apolipoprotein B at Baseline and at the Primary Efficacy Time Point
126.8; 132.8; 133.8; 95.0
SECONDARY
Percent Change From Baseline in Total Cholesterol at the Primary Efficacy Time Point
3.85; -19.43 <0.001 sig
SECONDARY
Total Cholesterol at Baseline and at the Primary Efficacy Time Point
213.4; 225.3; 219.0; 176.0
SECONDARY
Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol at the Primary Efficacy Time Point
3.74; -25.05 <0.001 sig
SECONDARY
Non-High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point
165.3; 175.5; 168.2; 125.2

Summary

The purpose of this study is to determine whether mipomersen safely and effectively lowers low-density lipoprotein cholesterol (LDL-C) in patients with Heterozygous Familial Hypercholesterolemia (HeFH) and coronary artery disease (CAD) who are already on a stable dose of other lipid-lowering agents (including maximally tolerated statin therapy).

Eligibility Criteria

Inclusion Criteria

  • Diagnosis of Heterozygous Familial Hypercholesterolemia (HeFH)
  • Diagnosis of Coronary Artery Disease (CAD)
  • Stable lipid-lowering therapy for 12 weeks
  • On maximally tolerated statin therapy with at least 1 statin at a dose greater than zero, per Investigator judgment
  • Stable low-fat diet for 8 weeks
  • Stable weight for 6 weeks

Exclusion Criteria

  • Significant health problems in recent past including heart attack, stroke, coronary syndrome, unstable angina, heart failure, significant arrhythmia, orthostatic hypotension, uncontrolled hypertension, blood disorders, liver disease, cancer, or digestive problems
  • Receiving apheresis treatment or last apheresis treatment within 8 weeks
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00706849). 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.

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