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
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)
- Secondary Outcome Measures
Name Time Method