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

Completed · Phase 3 · Has a placebo group

Conditions studied: Hypercholesterolemia, Heterozygous Familial

In brief

Primary objective: Determine whether mipomersen (ISIS 301012) significantly reduces atherogenic lipid levels in patients with severe heterozygous familial hypercholesterolemia (severe HeFH), defined as low-density lipoprotein cholesterol (LDL-C) levels ≥200 mg/dL plus the presence of coronary heart disease (CHD)/risk equivalents or LDL-C levels ≥300 mg/dL regardless of the presence of CHD/risk equivalents (referred to as Cohort 1) compared to placebo. Two different mipomersen dosing regimens will be studied: subcutaneous (SC) mipomersen 200 mg once weekly versus placebo, and SC mipomersen 70 mg thrice weekly versus placebo. Secondary Objectives: * Determine whether there are qualitative differences between the safety profiles of the 2 dosing regimens and placebo in Cohort 1, patients with HeFH with LDL-C levels ≥160 mg/dL and \<200 mg/dL plus the presence of CHD/risk equivalents (referred to as Cohort 2), and the overall study population * Determine whether there are qualitative differences between the tolerability of the 2 dosing regimens and placebo in Cohort 1, Cohort 2, and the overall study population * Further characterize the pharmacokinetics (PK) of the 2 dosing regimens in Cohort 1, Cohort 2, and the overall study population * Determine whether the 2 mipomersen dosing regimens significantly reduce atherogenic lipid levels in Cohort 2 compared to placebo * Obtain additional data regarding ongoing safety and efficacy of mipomersen in patients with FH and inadequately controlled LDL-C who complete the primary efficacy assessment visit (PET) in the Blinded Treatment Period and continue treatment in Open-Label Continuation Period

Key facts

Study ID

NCT01475825

Run by

Kastle Therapeutics, LLC

People needed

309

Starts

2011-12-01

Expected to finish

2015-12-29

Last updated by the study team

2019-03-26

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of severe hypercholesterolemia (LDL-C ≥300 mg/dL (7.77 mmol/L) or LDL-C ≥200 mg/dL (5.18 mmol/L) with documented coronary heart disease (CHD) or CHD risk equivalents, or diagnosis of Heterozygous Familial Hypercholesterolemia and LDL-C ≥160 mg/dL (4.14 mmol/L) and <200 mg/dL (5.18 mmol/L))
• On stable, maximally tolerated, statin therapy for at least 12 weeks or if statin intolerant, on at least 1 medication from another class of hypolipidemic agents (i.e., bile acid sequestrants, niacin/nicotinic acid, cholesterol absorption inhibitors, fibrates).
• On stable, low fat diet for 12 weeks
• Body mass index (BMI) ≤40 kg/m2 and stable weight for > 6 weeks

You may not qualify if…

• 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

Where it is running

• Study site — Aurora, Colorado, United States
• Study site — Cooper City, Florida, United States
• Study site — Winter Park, Florida, United States
• Study site — Indianapolis, Indiana, United States
• Study site — Kansas City, Kansas, United States
• Study site — Boston, Massachusetts, United States
• Study site — Ann Arbor, Michigan, United States
• Study site — Grandville, Michigan, United States
• Study site — Rochester, Minnesota, United States
• Study site — St Louis, Missouri, United States
• Study site — Omaha, Nebraska, United States
• Study site — Summit, New Jersey, United States
• Study site — North Massapequa, New York, United States
• Study site — Portland, Oregon, United States
• Study site — Lancaster, Pennsylvania, United States
• Study site — Charleston, South Carolina, United States
• Study site — Nashville, Tennessee, United States
• Study site — Dallas, Texas, United States
• Study site — Norfolk, Virginia, United States
• Study site — Buenos Aires, Argentina
• Study site — Córdoba, Argentina
• Study site — Perth, Australia
• Study site — South Brisbane, Australia
• Study site — Edegem, Belgium
• Study site — Mission Viejo, California, United States

Full record on ClinicalTrials.gov

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