A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol - FOCUS FH

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

CTRI

Registry ID

CTRI/2012/09/003013

Enrollment

480

Registered

2012-09-25

Start date

Unknown

Completion date

Unknown

Last updated

2021-11-24

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Health Condition 1: null- Hypercholesterolemia Coronary Heart Disease Health Condition 2: E780- Pure hypercholesterolemia

Interventions

Intervention1: mipomersen sodium: 200 mg subcutaneous injections administered once every other week for the first 8 weeks followed by once every week for 52 weeks. Intervention2: mipomersen sodium: 7

Eligibility

Inclusion criteria

Inclusion criteria: 1. Severe hypercholesterolemia (LDL-C >=300 mg/dL (7.77 mmol/L) or LDL-C >=200 mg/dL (5.18 mmol/L) with documented coronary heart disease (CHD) or CHD risk equivalents, or diagnosis of Heterozygous Familial Hypercholesterolemia and LDL-C >=160 mg/dL (4.14 mmol/L) and 2. On stable, maximally tolerated, statin therapy for at least 12 weeks or if statin intolerant, on at least 1 medication from another class of hypolipidemic agents (i.e., bile acid sequestrants, niacin/nicotinic acid, cholesterol absorption inhibitors, fibrates). 3. On stable, low fat diet for 12 weeks 4. Body mass index (BMI) In INDIA the upper age limit is 65 YEARS.

Exclusion criteria

Exclusion criteria: •Significant health problems in the recent past including heart attack, stroke, coronary syndrome, unstable angina, heart failure, significant arrhythmia, hypertension, blood disorders, liver disease, cancer, digestive disorders, Type I diabetes, or uncontrolled Type II diabetes •Apheresis within 3 months prior to Screening or expected to start apheresis during the treatment phase

Design outcomes

Primary

Measure	Time frame
Percent change from Baseline in low-density lipoprotein cholesterol (LDL-C) in Cohort 1Timepoint: Baseline and Week 60	—

Secondary

Measure	Time frame
â?¢ Percent Change from Baseline in Apolipoprotein B (Apo B) â?¢ Percent Change from Baseline in Lipoprotein a â?¢ Percent Change from Baseline in LDL-C in Cohort 2 â?¢ Number of Participants with Adverse Events â?¢ Number of Participants with Injection Site Reactions â?¢ Blood plasma concentration of MipomersenTimepoint: Week 60	—

Countries

Argentina, Australia, Belgium, Brazil, Canada, Croatia, Czech Republic, France, Germany, Greece, Hong Kong, Hungary, India, Israel, Italy, Malaysia, Netherlands, New Zealand, Norway, Poland, Russian Federation, Slovakia, South Africa, Spain, Sweden, Taiwan, Turkey, Ukraine, United Kingdom, United States of America

Contacts

Public ContactMs Salini James

Genzyme India ( A Sanofi Company)

salini.james@sanofi.com04442967545

Outcome results

None listed

Source: CTRI (via WHO ICTRP) · Data processed: Feb 4, 2026