Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy

A Phase 2, Open-Label, Dose Escalation Study to Assess the Safety and Efficacy of ISIS 301012 as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects

The aim of this study is to assess the safety and efficacy of varying doses of ISIS 301012 as add-on therapy in subjects with Homozygous Familial Hypercholesterolemia

Inclusion Criteria:

• Weight ≥ 40 kg for Cohorts A, B, & C; Weight ≥ 50 kg for Cohort D
• Diagnosis of Homozygous Familial Hypercholesterolemia.
• Female must be non-pregnant and non-lactating.
• On stable lipid lowering therapy for at least 4 weeks.
• Lipid values that meet the pre-specified criteria.

Exclusion Criteria:

• Subject had heart problems in the prior 6 months.
• Subject has elevated ALT, AST, or CPK.
• History of renal disease, liver disease, or malignancy.
• Use of oral anticoagulants, unless the dose has been stable for 4 weeks
• Have any other conditions, which in the opinion of the Investigator would make the subject unsuitable for enrollment, or could interfere with the subject participating in or completing the study.