Patients with homozygous and heterozygous familial hypercholesterolemia or severe hypercholesterolemia MedDRA version: 9.1 Level: LLT Classification code 10020604 Term: Hypercholesterolemia
Conditions
Interventions
Product Name: Mipomersen Sodium
Product Code: ISIS 301012
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Mipomersen Sodium
Sponsors
Genzyme Europe B.V.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Satisfactory completion of dosing and Week 28 assessments in their initial study (ISIS 301012-CS5, ISIS 301012-CS7 or MIPO3500108) with an acceptable safety profile, per Investigator judgment 2. Able and willing to participate in a 130-week study 3. Satisfy one of the following: (a) Females: Non-pregnant and non-lactating; surgically sterile, post-menopausal, abstinent, or subject or partner compliant with an acceptable contraceptive regimen for 4 weeks prior to, during, and 24 weeks after the last dose of ISIS 301012 OR (b) Males: Surgically sterile, abstinent or subject or partner is utilizing an acceptable contraceptive method during and 24 weeks after the last dose of ISIS 301012 4. Given informed consent, or, in the case of minors, the subject’s parents/legal guardians must be capable and willing of giving informed consent, and, if appropriate for the subject’s age, the subject must be capable of giving consent/ assent for study participation. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Have any new condition or worsening of existing condition 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. 2. Unwilling to limit alcohol consumption for the entire duration of the study, including follow-up: male subjects to a maximum of 2 drinks (20 g) per day, and 8 drinks (80 g) per week; female subjects to a maximum of 1 drink (10 g) per day, and 4 drinks (40 g) per week.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Secondary Objective: ;Main Objective: To evaluate the safety and efficacy of extended dosing with ISIS 301012 (mipomersen) in patients with familial hypercholesterolemia or severe hypercholesterolemia on concomitant lipid-lowering therapy; Primary end point(s): Safety Endpoints All AEs and SAEs Platelets, liver transaminases, renal function tests Other laboratory results Vital signs and ECG Efficacy Endpoints Percent reduction in LDL-C, apo B, total cholesterol and non-HDL-C Pharmacokinetic Endpoints Mipomersen plasma concentrations at scheduled timepoints | — |
Countries
United Kingdom
Outcome results
None listed