Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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EUCTR |
Last refreshed on:
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22 December 2015 |
Main ID: |
EUCTR2011-001480-42-IT |
Date of registration:
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22/02/2012 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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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
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Scientific title:
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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 |
Date of first enrolment:
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06/06/2012 |
Target sample size:
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480 |
Recruitment status: |
Not Recruiting |
URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-001480-42 |
Study type:
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Interventional clinical trial of medicinal product |
Study design:
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Controlled: yes
Randomised: yes
Open: no
Single blind: no
Double blind: yes
Parallel group: yes
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: yes
Other: yes
Other specify the comparator: - same IMP used at different dosage
Number of treatment arms in the trial: 4
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Phase:
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Countries of recruitment
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Argentina
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Australia
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Belgium
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Brazil
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Canada
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Czech Republic
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Denmark
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Germany
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Greece
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Hong Kong
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Hungary
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India
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Israel
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Italy
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Malaysia
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Netherlands
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New Zealand
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Poland
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Russian Federation
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Slovakia
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South Africa
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Spain
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Sweden
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Taiwan
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Turkey
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Ukraine
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United Kingdom
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United States
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Contacts
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Name:
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Medical Information Genzyme Europe
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Address:
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Gooimer 10
1411 DD
Naarden
Netherlands |
Telephone:
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NA |
Email:
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eumedinfo@genzyme.com |
Affiliation:
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Genzyme Europe B.V. |
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Name:
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Medical Information Genzyme Europe
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Address:
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Gooimer 10
1411 DD
Naarden
Netherlands |
Telephone:
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NA |
Email:
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eumedinfo@genzyme.com |
Affiliation:
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Genzyme Europe B.V. |
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Key inclusion & exclusion criteria
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Inclusion criteria: Diagnosis of severe hypercholesterolemia (LDL-C =300 mg/dL (7.77 mmol/L) or LDL-C = 200 mg/dL (5.18 mmol/L) and documented coronary heart disease (CHD) or CHD risk equivalent or diagnosis of Heterozygous Familial Hypercholesterolemia and LDL-C =160 mg/dL (4.14 mmol/L) and <200 mg/dL (5.18 mmol/L)) 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 Body mass index (BMI) =40 kg/m2 and stable weight for 6 weeks Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 460 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 20
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
Age minimum:
Age maximum:
Gender:
Female: yes Male: yes
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Health Condition(s) or Problem(s) studied
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Heterozygous familial hypercholesterolemia MedDRA version: 14.1
Level: LLT
Classification code 10057079
Term: Heterozygous familial hypercholesterolemia
System Organ Class: 10010331 - Congenital, familial and genetic disorders
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Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
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Intervention(s)
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Product Name: Mipomersen Product Code: ISIS 301012 Pharmaceutical Form: Solution for injection INN or Proposed INN: mipomersen CAS Number: 629167-92-6 Current Sponsor code: ISIS 301012 Other descriptive name: NA Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- Pharmaceutical form of the placebo: Solution for injection Route of administration of the placebo: Subcutaneous use
Product Name: Mipomersen Product Code: ISIS 301012 Pharmaceutical Form: Solution for injection INN or Proposed INN: mipomersen CAS Number: 629167-92-6 Current Sponsor code: ISIS 301012 Other descriptive name: NA Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 140- Pharmaceutical form of the placebo: Solution for injection Route of administration of the placebo: Subcutaneous use
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Primary Outcome(s)
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Secondary Objective: Determine whether there are qualitative differences between the safety profiles of the 2 dosing regimens and placebo in Cohort 1, patients with HeFH with LDL-C levels =160 mg/dL and <200 mg/dL plus the presence of CHD/risk equivalents (referred to as Cohort 2), and the overall study population (comprising patients in both Cohort 1 and Cohort 2) - Determine whether there are qualitative differences between the tolerability of the 2 dosing regimens and placebo in Cohort 1, Cohort 2, and the overall study population -Further characterize the pharmacokinetics (PK) of the 2 dosing regimens in Cohort 1, Cohort 2, and the overall study population - Determine whether the 2 mipomersen dosing regimens significantly reduce atherogenic lipid levels in Cohort 2 compared to placebo
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Main Objective: Determine whether mipomersen (ISIS 301012) significantly reduces atherogenic lipid levels in patients with severe heterozygous familial hypercholesterolemia (severe HeFH), defined as low-density lipoprotein cholesterol (LDL-C) levels =200 mg/dL plus the presence of coronary heart disease (CHD)/risk equivalents or LDL-C levels =300 mg/dL regardless of the presence of CHD/risk equivalents (referred to as Cohort 1) compared to placebo. Two different mipomersen dosing regimens will be studied: subcutaneous (SC) mipomersen 200 mg once weekly versus placebo, and SC mipomersen 70 mg thrice weekly versus placebo.
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Primary end point(s): Percent change from Baseline in low-density lipoprotein cholesterol in Cohort 1
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Timepoint(s) of evaluation of this end point: Baseline to Week 60
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Secondary Outcome(s)
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Timepoint(s) of evaluation of this end point: Percent change from Baseline in Apolipoprotein : Baseline to Week 60 Percent change from Baseline in Lipoprotein a : Baseline to Week 60 Percent change from Baseline in low-density lipoprotein cholesterol in Cohort 2 : Baseline to Week 60 Number of Participants with Adverse Events : 60 weeks Number of Participants with injection site reactions : 60 weeks Blood plasma concentration of Mipomersen : Pre-dose and at 2, 4, 6, 24, 48 and 72 hours post-dose at Weeks 1,13, 30, 38, 50 and 60.
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Secondary end point(s): Percent change from Baseline in Apolipoprotein Percent change from Baseline in Lipoprotein a Percent change from Baseline in low-density lipoprotein cholesterol in Cohort 2 Number of Participants with Adverse Events Number of Participants with injection site reactions Blood plasma concentration of Mipomersen
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Secondary ID(s)
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MIPO3801011
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2011-001480-42-ES
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Source(s) of Monetary Support
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GENZYME
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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