Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia

Completed · Phase 3 · Has a placebo group

Conditions studied: 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

In brief

The purpose of this study is to evaluate the safety and efficacy of mipomersen (ISIS 301012) in subjects with homozygous familial hypercholesterolemia on lipid-lowering therapy. This study consisted of a 26-week treatment period and a 24-week post-treatment follow-up period. Following treatment and Week 28 evaluations, participants could elect to enroll in an open-label extension study (301012-CS6; NCT00694109). Participants who were not eligible or elected not to enroll in the open-label extension study or who discontinued during the 28-week treatment period were followed in this study for 24 weeks from administration of the last dose of study drug.

Key facts

Study ID

NCT00607373

Run by

Kastle Therapeutics, LLC

People needed

51

Starts

2007-07-01

Expected to finish

2009-03-01

Last updated by the study team

2016-09-09

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of Homozygous Familial Hypercholesterolemia (HoFH)
• Stable lipid-lowering therapy for 12 weeks
• Stable weight for 6 weeks
• Stable low fat diet for 8 weeks

You may not qualify if…

• Significant health problems in the recent past including heart attack, stroke, blood disorders, cancer, or digestive problems

Where it is running

• Study site — Charlotte, North Carolina, United States
• Study site — Cincinnati, Ohio, United States
• Study site — São Paulo, São Paulo, Brazil
• Study site — Chicoutimi, Quebec, Canada
• Study site — Ste Foy, Quebec, Canada
• Study site — Mistri Wing, Singapore
• Study site — Observatory, South Africa
• Study site — Parktown, South Africa
• Study site — Taipei, Taiwan
• Study site — London, United Kingdom

Full record on ClinicalTrials.gov

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