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| Sponsors and Collaborators: |
Genzyme Isis Pharmaceuticals |
|---|---|
| Information provided by: | Genzyme |
| ClinicalTrials.gov Identifier: | NCT00477594 |
Purpose
The purpose of this study is to evaluate the safety and efficacy of mipomersen in subjects with familial hypercholesterolemia on lipid-lowering therapy who have completed either the 301012-CS8 or 301012-CS9 clinical drug trials.
| Condition | Intervention | Phase |
|---|---|---|
|
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 |
Drug: mipomersen sodium |
Phase II |
| Study Type: | Interventional |
| Study Design: | Treatment, Randomized, Open Label, Uncontrolled, Parallel Assignment, Safety/Efficacy Study |
| Official Title: | An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 301012 in Subjects With Familial Hypercholesterolemia |
| Estimated Enrollment: | 52 |
| Study Start Date: | May 2007 |
| Estimated Study Completion Date: | June 2010 |
| Estimated Primary Completion Date: | March 2010 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
1: Experimental
mipomersen 200 mg per week
|
Drug: mipomersen sodium
200 mg s.c. once a week.
|
|
2: Experimental
mipomersen 200 mg every other week
|
Drug: mipomersen sodium
200 mg s.c. once every other week
|
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. These are the 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 apoB-100, the principal apolipoprotein of atherogenic LDL-C and its metabolic precursor, VLDL. Mipomersen is complimentary to the coding region of the mRNA for apoB-100. Inhibition of apoB-100 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 subjects.
The intent of this trial is to gather additional information to evaluate the overall benefit-risk relationship of the drug, and to provide ongoing therapy for FH subjects who participated in two prior mipomersen trials (301012-CS8 and 301012-CS9).
Eligibility| Ages Eligible for Study: | 12 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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 or 301012-CS9).
Exclusion Criteria:
- Have a new condition or worsening of existing condition which in the opinion of the Investigator would make the subject unsuitable for enrollment, or could interfere with subject's participation in or completion of the study.
Contacts and Locations| United States, Illinois | |
| Chicago, Illinois, United States, 60610 | |
| United States, Maine | |
| Auburn, Maine, United States, 04210 | |
| Scarborough, Maine, United States, 04074 | |
| United States, Ohio | |
| Cincinnati, Ohio, United States, 45212 | |
| Study Chair: | Joanne M Donovan, M.D., PhD | Genzyme |
More Information
| Responsible Party: | Genzyme Corporation. ( Medical Monitor ) |
| Study ID Numbers: | 301012-CS17 |
| Study First Received: | May 22, 2007 |
| Last Updated: | August 16, 2008 |
| ClinicalTrials.gov Identifier: | NCT00477594 |
| Health Authority: | United States: Food and Drug Administration |
|
Familial Hypercholesterolemia Heterozygous Familial Hypercholesterolemia Homozygous Familial Hypercholesterolemia |
ISIS 301012 mipomersen Open Label Extension |
|
Lipid Metabolism, Inborn Errors Hypercholesterolemia, autosomal dominant Hyperlipidemias Metabolic Diseases Hyperlipoproteinemia Type II Metabolism, Inborn Errors Genetic Diseases, Inborn |
Infant, Newborn, Diseases Congenital Abnormalities Metabolic disorder Hypercholesterolemia Dyslipidemias Hyperlipoproteinemias Lipid Metabolism Disorders |
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Pathologic Processes Disease |
