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

Phase 3

Active, not recruiting

• Conditions: Pure hypercholesterolemia,

• Registration Number: CTRI/2012/09/003013
• Lead Sponsor: Genzyme India

Brief Summary

This study is a phase 3, randomized, double-blind,placebo-controlled, parallel-group study to assess the safety and efficacy oftwo different regimens of mipomersen in patients with familialhypercholesterolemia and inadequately controlled low-density lipoproteincholesterol. This study will be conducted in about 30 countries globally,enrolling 480 patients. From India,40 patients will be enrolled in to the study at 4 centers.

Theprimary objective of this study is to determine whether mipomersen (ISIS301012) significantly reduces atherogenic lipid levels in patients with severeheterozygous familial hypercholesterolemia (severe HeFH), defined aslow-density lipoprotein cholesterol (LDL-C) levels ≥200 mg/dL plus the presenceof coronary heart disease (CHD)/risk equivalents or LDL-C levels ≥300 mg/dLregardless of the presence of CHD/risk equivalents compared to placebo. Twodifferent mipomersen dosing regimens will be studied: subcutaneous (SC)mipomersen 200 mg once weekly versus placebo, and SC mipomersen 70 mg thriceweekly versus placebo.

Primary outcome measure will be percent change fromBaseline in low-density lipoprotein cholesterol (LDL-C) in Cohort 1 [ TimeFrame: Baseline and Week 60 ]

Detailed Description

Not available

Study Timeline

• Study Start: 2012-01-09
• Last Update Posted: 2018-11-13

Recruitment & Eligibility

• Status: Closed to Recruitment of Participants
• Sex: All
• Target Recruitment: 480

Inclusion Criteria

• 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 <200 mg/dL (5.18 mmol/L)) 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).
• On stable, low fat diet for 12 weeks 4.
• Body mass index (BMI) ≤40 kg/m2 and stable weight for 6 weeks In INDIA the upper age limit is 65 YEARS.

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.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Percent change from Baseline in low-density lipoprotein cholesterol (LDL-C) in Cohort 1	Baseline and Week 60

Secondary Outcome Measures

Name	Time	Method
• Percent Change from Baseline in Apolipoprotein B (Apo B)	• Percent Change from Baseline in Lipoprotein a

Trial Locations

Locations (4)

Choithram Hospital & Research Centre; 🇮🇳 Indore, MADHYA PRADESH, India

Fortis Escort Heart Institute; 🇮🇳 Delhi, DELHI, India

Fortis Hospital, Mohali; 🇮🇳 VIIIMohali,, India

M. V. Hospital and Research Center; 🇮🇳 Lucknow, UTTAR PRADESH, India

Choithram Hospital & Research Centre

🇮🇳Indore, MADHYA PRADESH, India

Dr Vidhut Jain

Principal investigator

09826015216

vidyut.jain213@gmail.com