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

• Status: Completed
• Trial end date: 2007-08-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Kastle Therapeutics, LLC
• Collaborator: Ionis Pharmaceuticals, Inc.
• Treatments: Mipomersen

Criteria

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.