Hypercholesterolemia, Familial
Conditions
Keywords
LDL-cholesterol, apoB-100, Heterozygous Familial Hypercholesterolemia
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
Interventions
50 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36
Sponsors
Ionis Pharmaceuticals, Inc.
Kastle Therapeutics, LLC
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percent reduction in LDL-cholesterol from baseline | Week 7 (Cohorts A-C), Week 15 (Cohort D) |
Countries
Netherlands, United States
Outcome results
None listed