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CTRI Number  CTRI/2012/09/003013 [Registered on: 25/09/2012] Trial Registered Retrospectively
Last Modified On: 13/11/2018
Post Graduate Thesis  No 
Type of Trial  Interventional 
Type of Study   Biological 
Study Design  Randomized, Parallel Group, Placebo Controlled Trial 
Public Title of Study   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 
Scientific Title of Study   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 
Trial Acronym  FOCUS FH 
Secondary IDs if Any
Modification(s)  
Secondary ID  Identifier 
2011-001480-42  EudraCT 
MIPO3801011 - Protocol Amend.01 dated 18-July-2013  Protocol Number 
 
Details of Principal Investigator or overall Trial Coordinator (multi-center study)  
Name   
Designation   
Affiliation   
Address 




 
Phone    
Fax    
Email    
 
Details of Contact Person
Scientific Query
Modification(s)  
Name  Ms Salini James  
Designation  Senior Clinical Project Leader  
Affiliation  Genzyme India ( A Sanofi Company) 
Address  A-101, Business Square, Sir Mathuradas Vasanji Marg, Chakala, Andheri (E), Mumbai – 400093

Mumbai
MAHARASHTRA
400093
India 
Phone  04442967545  
Fax    
Email  salini.james@sanofi.com  
 
Details of Contact Person
Public Query
Modification(s)  
Name  Ms Salini James  
Designation  Senior Clinical Project Leader  
Affiliation  Genzyme India (A Sanofi Company) 
Address  A-101, Business Square, Sir Mathuradas Vasanji Marg, Chakala, Andheri (E), Mumbai – 400093

Mumbai
MAHARASHTRA
400093
India 
Phone  04442967545  
Fax    
Email  salini.james@sanofi.com  
 
Source of Monetary or Material Support  
Genzyme Corporation 500, Kendall Street, Cambridge, MA 02142 USA  
 
Primary Sponsor  
Name  Genzyme India  
Address  1st floor, Technopolis, Golf Course Road, Sector 54, Gurgaon 122002 (Haryana) India Tel: +91 124 452 8300. Fax: +91 124 452 8400.  
Type of Sponsor  Pharmaceutical industry-Global 
 
Details of Secondary Sponsor  
Name  Address 
NIL  NIL 
 
Countries of Recruitment     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  
Sites of Study  
No of Sites = 4  
Name of Principal Investigator  Name of Site  Site Address  Phone/Fax/Email 
Dr Vidhut Jain  Choithram Hospital & Research Centre  Choithram Hospital & Research Centre, Manik Bagh Road, Indore,Madhya Pradesh, 452 001
Indore
MADHYA PRADESH 		 09826015216
07312470068
vidyut.jain213@gmail.com 
Dr Peeyush Jain   Fortis Escort Heart Institute  Fortis Escort Heart Institute, Academic and Research center, Room No 23 A , 2nd Floor , Residential Tower, Okhla Road , New Delhi, 110025, India.
New Delhi
DELHI 		 919818701043
911126825112
peeyush.jain@fortishealthcare.com 
Dr Harinder Bali  Fortis Hospital, Mohali  Fortis Hospital, Mohali Sector 62, Phase VIII Mohali, 160062

 		 01725091980
01724692221
hkbalipgi@gmail.com 
Dr Sandeep Gupta  M. V. Hospital and Research Center  M. V. Hospital and Research Center, 314/30 Mirza Mandi, Chowk, Lucknow, 226017
Lucknow
UTTAR PRADESH 		 05222258215
05224016051
sandeepkumar.gupta@rediffmail.com 
 
Details of Ethics Committee
Modification(s)  
No of Ethics Committees= 4  
Name of Committee  Approval Status 
Chotiram Hospital and Research CentreEC_Indore_Dr. Vidhut Jain   Submittted/Under Review 
IEC of Fortis Hospital Mohali (Harinder Bali)  Approved 
IEC of Fortis Hospital, Delhi (Peeyush Jain)  Approved 
IEC of MVHRC, Lucknow (Sandeep Gupta)  Approved 
 
Regulatory Clearance Status from DCGI  
Status 
Approved/Obtained 
 
Health Condition / Problems Studied
Modification(s)  
Health Type  Condition 
Patients  Hypercholesterolemia Coronary Heart Disease , (1) ICD-10 Condition: E780||Pure hypercholesterolemia,  
 
Intervention / Comparator Agent  
Type  Name  Details 
Intervention  mipomersen sodium  200 mg subcutaneous injections administered once every other week for the first 8 weeks followed by once every week for 52 weeks. 
Intervention  mipomersen sodium  70mg subcutaneous injections administered three times a week every other week for the first 8 weeks followed by three times a week every week for 52 weeks. 
Comparator Agent  Placebo  Placebo subcutaneous injections administered once every other week for the first 8 weeks followed by once every week for 52 weeks. 
Comparator Agent  Placebo  Placebo subcutaneous injections administered three times a week every other week for the first 8 weeks followed by three times a week every week for 52 weeks. 
 
Inclusion Criteria  
Age From  18.00 Year(s)
Age To  99.00 Year(s)
Gender  Both 
Details  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 <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).
3. 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.
 
 
ExclusionCriteria 
Details  •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
 
 
Method of Generating Random Sequence   Computer generated randomization 
Method of Concealment   Centralized 
Blinding/Masking   Participant, Investigator and Outcome Assessor Blinded 
Primary Outcome  
Outcome  TimePoints 
Percent change from Baseline in low-density lipoprotein cholesterol (LDL-C) in Cohort 1   Baseline and Week 60  
 
Secondary Outcome  
Outcome  TimePoints 
• Percent Change from Baseline in Apolipoprotein B (Apo B)
• Percent Change from Baseline in Lipoprotein a
• Percent Change from Baseline in LDL-C in Cohort 2
• Number of Participants with Adverse Events
• Number of Participants with Injection Site Reactions
• Blood plasma concentration of Mipomersen  		 Week 60 
 
Target Sample Size   Total Sample Size="480"
Sample Size from India="40" 
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" 
Phase of Trial   Phase 3 
Date of First Enrollment (India)   01/09/2012 
Date of Study Completion (India) Applicable only for Completed/Terminated trials 
Date of First Enrollment (Global)  30/12/2011 
Date of Study Completion (Global) Applicable only for Completed/Terminated trials 
Estimated Duration of Trial   Years="3"
Months="0"
Days="0" 
Recruitment Status of Trial (Global)
Modification(s)  		 Closed to Recruitment of Participants 
Recruitment Status of Trial (India)  Closed to Recruitment of Participants 
Publication Details   None yet available.  
Individual Participant Data (IPD) Sharing Statement

Will individual participant data (IPD) be shared publicly (including data dictionaries)?  

Brief Summary  

This study is 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. 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.

The primary objective of this study is to 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 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.

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