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

• Status: Completed
• Trial end date: 2009-03-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

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

Criteria

Inclusion Criteria:

• 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

Exclusion Criteria:

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