Study of ISIS 301012 (Mipomersen) in Heterozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy

Phase 2

Completed

• Conditions: Hypercholesterolemia, Familial
• Interventions: Drug: ISIS 301012 or Placebo

• Registration Number: NCT00281008
• Lead Sponsor: Kastle Therapeutics, LLC

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2006-01-20
• Study First Submitted QC: 2006-01-20
• Study First Posted: 2006-01-24
• Study Start: 2006-02
• Primary Completion: 2007-07
• Study Completion: 2007-12
• Status Verified: 2016-08
• Last Update Submitted: 2016-08-01
• Last Update Posted: 2016-08-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 44

Inclusion Criteria

• Weight ≥ 50 kg
• Diagnosis of Heterozygous Familial Hypercholesterolemia.
• Females must be non-pregnant and non-lactating.
• On stable lipid lowering therapy for at least 4 weeks.
• Lipid levels 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cohort A	ISIS 301012 or Placebo	Loading doses followed by weekly maintenance doses
Cohort D	ISIS 301012 or Placebo	Loading doses followed by extended weekly maintenance doses
Cohort C	ISIS 301012 or Placebo	Loading doses followed by weekly maintenance doses
Cohort B	ISIS 301012 or Placebo	Loading doses followed by weekly maintenance doses

Primary Outcome Measures

Name	Time	Method
Percent reduction in LDL-cholesterol from baseline	Week 7 (Cohorts A-C), Week 15 (Cohort D)