Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

21 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-05-31

Study Completion Date

2011-07-31

Brief Summary

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The purpose of this study is to evaluate the safety and efficacy of extended dosing of mipomersen in patients with familial hypercholesterolemia on lipid-lowering therapy who have completed either the 301012-CS8 (NCT00280995) or 301012-CS9 (NCT00281008) clinical drug trials.

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Detailed Description

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Familial Hypercholesterolemia (FH) is an autosomal dominant metabolic disorder characterized by markedly elevated low density lipoprotein (LDL), premature onset of atherosclerosis, and development of xanthomata. There are two distinct subpopulations that have a high unmet medical need due to the lack of alternative therapy: homozygotes, who have two defective LDL receptor (LDL-R) genes, and heterozygotes with a history of cardiovascular disease (CVD) on maximally tolerated therapy. Treatment for FH is directed at lowering plasma levels of LDL-C.

Mipomersen is an antisense drug targeted to human apolipoprotein B (apoB), the principal apolipoprotein of atherogenic LDL-C and its metabolic precursor, very low density lipoprotein (VLDL). Mipomersen is complimentary to the coding region of the messenger ribonucleic acid (mRNA) for apo-B. Inhibition of apo-B would be expected to impair VLDL synthesis and result in lowered levels of LDL-C.

In early clinical trials, mipomersen has been shown to reduce levels of LDL-C to recommended target levels in some participants.

This was an open-label extension study, which consisted of a ≤2-week screening period, up to 3 years of treatment with mipomersen, and a 24-week post-treatment follow-up period. Patients who participated in Cohorts A, B, or C in study 301012-CS9 were randomized in a 1:1 ratio to mipomersen 200 mg once a week (QW) or 200 mg mipomersen every other week (QOW) for up to 3 years. Patients randomized to mipomersen 200 mg QOW were allowed to receive mipomersen 200 mg QW at the Investigator's discretion after the first 52 weeks of the treatment period. Patients who participated in study 301012-CS8 or Cohort D of study 301012-CS9 received 200 mg mipomersen QW for up to 3 years.

Conditions

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Lipid Metabolism, Inborn Errors Hypercholesterolemia, Autosomal Dominant Hyperlipidemias Metabolic Diseases Hyperlipoproteinemia Type II Metabolism, Inborn Errors Genetic Diseases, Inborn Infant, Newborn, Diseases Metabolic Disorder Congenital Abnormalities Hypercholesterolemia Hyperlipoproteinemias Dyslipidemias Lipid Metabolism Disorders

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Mipomersen 200 mg per week

Participants received 200 mg mipomersen once a week by subcutaneous injection, for up to 3 years.

Group Type EXPERIMENTAL

mipomersen sodium

Intervention Type DRUG

200 mg/ml, in 1 ml solution for subcutaneous injection.

Mipomersen 200 mg every other week

Participants received 200 mg mipomersen every other week by subcutaneous injection, for up to 3 years. Participants could receive mipomersen 200 mg once a week at the Investigator's discretion after the first 52 weeks of the treatment period.

Group Type EXPERIMENTAL

mipomersen sodium

Intervention Type DRUG

200 mg/ml, in 1 ml solution for subcutaneous injection.

Interventions

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mipomersen sodium

200 mg/ml, in 1 ml solution for subcutaneous injection.

Intervention Type DRUG

Other Intervention Names

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ISIS 301012 Kynamro™

Eligibility Criteria

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Inclusion Criteria

\- Satisfactory completion of dosing and Week 7 or Week 15 assessments (depending on the treatment and dose received) in their initial study (Protocol 301012-CS8 (NCT00280995) or 301012-CS9 (NCT00281008)).

Exclusion Criteria

\- Have a new condition or worsening of existing condition which in the opinion of the Investigator would make the patient unsuitable for enrollment, or could interfere with patient's participation in or completion of the study.

Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No