NCT00477594

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

An Open-Label Extension Study to Assess the Long-term Safety and Efficacy of Mipomersen in Subjects With Familial Hypercholesterolemia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 21 (actual)
• Sponsor: Kastle Therapeutics, LLC · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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

• 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

Interventions

Type	Name	Description
DRUG	mipomersen sodium	200 mg/ml, in 1 ml solution for subcutaneous injection.

Timeline

• Start date: 2007-05-01
• Primary completion: 2011-03-01
• Completion: 2011-07-01
• First posted: 2007-05-24
• Last updated: 2016-09-09
• Results posted: 2013-04-05

Locations

5 sites across 1 country: United States