Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT01475825
Registration number
NCT01475825
Ethics application status
Date submitted
17/11/2011
Date registered
21/11/2011
Date last updated
26/03/2019
Titles & IDs
Public title
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
Query!
Scientific title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study Followed by an Open-Label Continuation Period to Assess the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol
Query!
Secondary ID [1]
0
0
2011-001480-42
Query!
Secondary ID [2]
0
0
MIPO3801011
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
FOCUS FH
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Hypercholesterolemia
0
0
Query!
Heterozygous Familial
0
0
Query!
Condition category
Condition code
Metabolic and Endocrine
0
0
0
0
Query!
Other metabolic disorders
Query!
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - mipomersen sodium 200 mg
Treatment: Drugs - Placebo
Treatment: Drugs - mipomersen sodium 70 mg
Experimental: Regimen A: Mipomersen - Subcutaneous injection of mipomersen 200 mg once weekly
Placebo comparator: Regimen A: Placebo - Placebo matching subcutaneous injection once weekly.
Experimental: Regimen B: Mipomersen - Subcutaneous injection of mipomersen 70 mg thrice weekly.
Placebo comparator: Regimen B: Placebo - Placebo matching subcutaneous injection thrice weekly.
Treatment: Drugs: mipomersen sodium 200 mg
Subcutaneous mipomersen 200 mg once weekly
Treatment: Drugs: Placebo
Placebo vehicle for subcutaneous injection.
Treatment: Drugs: mipomersen sodium 70 mg
Subcutaneous mipomersen 70 mg thrice weekly
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Percent Change From Baseline To Primary Endpoint Visit (PET) In LDL-C In Cohort 1
Query!
Assessment method [1]
0
0
The percent change from baseline to PET in LDL-C was measured in Cohort 1, which included participants with severe heterozygous familial hypercholesterolemia (HeFH). Severe HeFH was defined as LDL-C levels =200 mg/deciliter (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.
Query!
Timepoint [1]
0
0
Baseline and Week 61
Query!
Secondary outcome [1]
0
0
Percent Change From Baseline To PET In LDL-C In Cohort 2
Query!
Assessment method [1]
0
0
The percent change from baseline to PET in LDL-C was measured in Cohort 2, which included participants with HeFH with LDL-C levels =160 mg/dL and \<200 mg/dL, plus the presence of CHD/risk equivalents.
Query!
Timepoint [1]
0
0
Baseline, PET (up to 60 weeks)
Query!
Secondary outcome [2]
0
0
Percent Change From Baseline To PET In Apolipoprotein B (Apo B) In Cohort 1
Query!
Assessment method [2]
0
0
The percent change from baseline to PET in Apo B was measured in participants in Cohort 1 with HeFH during the Blinded Treatment Period.
Query!
Timepoint [2]
0
0
Baseline and Week 61
Query!
Secondary outcome [3]
0
0
Percent Change From Baseline To PET In Apolipoprotein B (Apo B) In Cohort 2
Query!
Assessment method [3]
0
0
The percent change from baseline to PET in Apo B was measured in participants in Cohort 2 with HeFH during the Blinded Treatment Period.
Query!
Timepoint [3]
0
0
Baseline and Week 61
Query!
Secondary outcome [4]
0
0
Percent Change From Baseline To PET in Lipoprotein (a) In Cohort 1
Query!
Assessment method [4]
0
0
The percent change from baseline to PET in Lipoprotein A1 was measured in participants in Cohort 1 with HeFH during the Blinded Treatment Period.
Query!
Timepoint [4]
0
0
Baseline and Week 61
Query!
Secondary outcome [5]
0
0
Percent Change From Baseline To PET in Lipoprotein (a) In Cohort 2
Query!
Assessment method [5]
0
0
The percent change from baseline to PET in Lipoprotein A1 was measured in participants in Cohort 2 with HeFH during the Blinded Treatment Period.
Query!
Timepoint [5]
0
0
Baseline and Week 61
Query!
Eligibility
Key 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) 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))
* 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
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key 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
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
1/12/2011
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
29/12/2015
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
309
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment hospital [1]
0
0
- Perth
Query!
Recruitment hospital [2]
0
0
- South Brisbane
Query!
Recruitment postcode(s) [1]
0
0
- Perth
Query!
Recruitment postcode(s) [2]
0
0
- South Brisbane
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
California
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Colorado
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Florida
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Indiana
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Kansas
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Massachusetts
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Michigan
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Minnesota
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
Missouri
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
Nebraska
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
New Jersey
Query!
Country [12]
0
0
United States of America
Query!
State/province [12]
0
0
New York
Query!
Country [13]
0
0
United States of America
Query!
State/province [13]
0
0
Oregon
Query!
Country [14]
0
0
United States of America
Query!
State/province [14]
0
0
Pennsylvania
Query!
Country [15]
0
0
United States of America
Query!
State/province [15]
0
0
South Carolina
Query!
Country [16]
0
0
United States of America
Query!
State/province [16]
0
0
Tennessee
Query!
Country [17]
0
0
United States of America
Query!
State/province [17]
0
0
Texas
Query!
Country [18]
0
0
United States of America
Query!
State/province [18]
0
0
Virginia
Query!
Country [19]
0
0
Argentina
Query!
State/province [19]
0
0
Ciudad Autonoma de Buenos Aires
Query!
Country [20]
0
0
Argentina
Query!
State/province [20]
0
0
Cordoba
Query!
Country [21]
0
0
Belgium
Query!
State/province [21]
0
0
Edegem
Query!
Country [22]
0
0
Belgium
Query!
State/province [22]
0
0
Haine St. Paul
Query!
Country [23]
0
0
Belgium
Query!
State/province [23]
0
0
Leuven
Query!
Country [24]
0
0
Brazil
Query!
State/province [24]
0
0
Rio de Janeiro
Query!
Country [25]
0
0
Brazil
Query!
State/province [25]
0
0
Sao Paulo
Query!
Country [26]
0
0
Canada
Query!
State/province [26]
0
0
Quebec
Query!
Country [27]
0
0
Croatia
Query!
State/province [27]
0
0
Osijek
Query!
Country [28]
0
0
Croatia
Query!
State/province [28]
0
0
Zagreb
Query!
Country [29]
0
0
Czechia
Query!
State/province [29]
0
0
Hradec Kralove
Query!
Country [30]
0
0
Czechia
Query!
State/province [30]
0
0
Praha 1
Query!
Country [31]
0
0
Denmark
Query!
State/province [31]
0
0
Aarhus
Query!
Country [32]
0
0
Denmark
Query!
State/province [32]
0
0
Viborg
Query!
Country [33]
0
0
Germany
Query!
State/province [33]
0
0
Aachen
Query!
Country [34]
0
0
Germany
Query!
State/province [34]
0
0
Berlin
Query!
Country [35]
0
0
Germany
Query!
State/province [35]
0
0
Freiburg
Query!
Country [36]
0
0
Germany
Query!
State/province [36]
0
0
Hamburg
Query!
Country [37]
0
0
Germany
Query!
State/province [37]
0
0
Heidelberg
Query!
Country [38]
0
0
Germany
Query!
State/province [38]
0
0
Koeln
Query!
Country [39]
0
0
Germany
Query!
State/province [39]
0
0
Magdeburg
Query!
Country [40]
0
0
Greece
Query!
State/province [40]
0
0
Ioannina
Query!
Country [41]
0
0
Greece
Query!
State/province [41]
0
0
Kallithea
Query!
Country [42]
0
0
Hong Kong
Query!
State/province [42]
0
0
Hong Kong
Query!
Country [43]
0
0
Hungary
Query!
State/province [43]
0
0
Baja
Query!
Country [44]
0
0
Hungary
Query!
State/province [44]
0
0
Budapest
Query!
Country [45]
0
0
Hungary
Query!
State/province [45]
0
0
Debrecen
Query!
Country [46]
0
0
India
Query!
State/province [46]
0
0
New Delhi
Query!
Country [47]
0
0
Israel
Query!
State/province [47]
0
0
Holon
Query!
Country [48]
0
0
Israel
Query!
State/province [48]
0
0
Kfar Saba
Query!
Country [49]
0
0
Israel
Query!
State/province [49]
0
0
Ofakim
Query!
Country [50]
0
0
Italy
Query!
State/province [50]
0
0
Bologna
Query!
Country [51]
0
0
Italy
Query!
State/province [51]
0
0
Napoli
Query!
Country [52]
0
0
Italy
Query!
State/province [52]
0
0
Padova
Query!
Country [53]
0
0
Italy
Query!
State/province [53]
0
0
Palermo
Query!
Country [54]
0
0
Italy
Query!
State/province [54]
0
0
Pisa
Query!
Country [55]
0
0
Italy
Query!
State/province [55]
0
0
Roma
Query!
Country [56]
0
0
Korea, Republic of
Query!
State/province [56]
0
0
Seoul
Query!
Country [57]
0
0
Malaysia
Query!
State/province [57]
0
0
Kuala Lumpur
Query!
Country [58]
0
0
Malaysia
Query!
State/province [58]
0
0
Kubang Kerian
Query!
Country [59]
0
0
Netherlands
Query!
State/province [59]
0
0
Alkmaar
Query!
Country [60]
0
0
Netherlands
Query!
State/province [60]
0
0
Amsterdam
Query!
Country [61]
0
0
Netherlands
Query!
State/province [61]
0
0
Maastricht
Query!
Country [62]
0
0
Netherlands
Query!
State/province [62]
0
0
Nijmegen
Query!
Country [63]
0
0
Netherlands
Query!
State/province [63]
0
0
Utrecht
Query!
Country [64]
0
0
Netherlands
Query!
State/province [64]
0
0
Waalwijk
Query!
Country [65]
0
0
New Zealand
Query!
State/province [65]
0
0
Christchurch
Query!
Country [66]
0
0
Norway
Query!
State/province [66]
0
0
Bodo
Query!
Country [67]
0
0
Norway
Query!
State/province [67]
0
0
Oslo
Query!
Country [68]
0
0
Norway
Query!
State/province [68]
0
0
Sandefjord
Query!
Country [69]
0
0
Poland
Query!
State/province [69]
0
0
Bialystok
Query!
Country [70]
0
0
Poland
Query!
State/province [70]
0
0
Gdansk
Query!
Country [71]
0
0
Poland
Query!
State/province [71]
0
0
Katowice
Query!
Country [72]
0
0
Poland
Query!
State/province [72]
0
0
Krakow
Query!
Country [73]
0
0
Poland
Query!
State/province [73]
0
0
Naleczow
Query!
Country [74]
0
0
Poland
Query!
State/province [74]
0
0
Poznan
Query!
Country [75]
0
0
Poland
Query!
State/province [75]
0
0
Sopot
Query!
Country [76]
0
0
Poland
Query!
State/province [76]
0
0
Szczecin
Query!
Country [77]
0
0
Poland
Query!
State/province [77]
0
0
Warszawa
Query!
Country [78]
0
0
Poland
Query!
State/province [78]
0
0
Wroclaw
Query!
Country [79]
0
0
Russian Federation
Query!
State/province [79]
0
0
Barnaul
Query!
Country [80]
0
0
Russian Federation
Query!
State/province [80]
0
0
Kemerovo
Query!
Country [81]
0
0
Russian Federation
Query!
State/province [81]
0
0
Moscow
Query!
Country [82]
0
0
Russian Federation
Query!
State/province [82]
0
0
Novosibirsk
Query!
Country [83]
0
0
Russian Federation
Query!
State/province [83]
0
0
Petrozavodsk
Query!
Country [84]
0
0
Russian Federation
Query!
State/province [84]
0
0
Ryazan
Query!
Country [85]
0
0
Russian Federation
Query!
State/province [85]
0
0
Saint Petersburg
Query!
Country [86]
0
0
Russian Federation
Query!
State/province [86]
0
0
St-Petersburg
Query!
Country [87]
0
0
Russian Federation
Query!
State/province [87]
0
0
St. Petersburg
Query!
Country [88]
0
0
Russian Federation
Query!
State/province [88]
0
0
Tomsk
Query!
Country [89]
0
0
Russian Federation
Query!
State/province [89]
0
0
Yaroslavl
Query!
Country [90]
0
0
South Africa
Query!
State/province [90]
0
0
Cape Town
Query!
Country [91]
0
0
South Africa
Query!
State/province [91]
0
0
Pretoria
Query!
Country [92]
0
0
Spain
Query!
State/province [92]
0
0
Madrid
Query!
Country [93]
0
0
Sweden
Query!
State/province [93]
0
0
Stockholm
Query!
Country [94]
0
0
Taiwan
Query!
State/province [94]
0
0
New Taipei City
Query!
Country [95]
0
0
Taiwan
Query!
State/province [95]
0
0
Taipei
Query!
Country [96]
0
0
Turkey
Query!
State/province [96]
0
0
Ankara
Query!
Country [97]
0
0
Turkey
Query!
State/province [97]
0
0
Istanbul
Query!
Country [98]
0
0
Turkey
Query!
State/province [98]
0
0
Izmir
Query!
Country [99]
0
0
Turkey
Query!
State/province [99]
0
0
Sivas
Query!
Country [100]
0
0
Ukraine
Query!
State/province [100]
0
0
Ivano-Frankivsk
Query!
Country [101]
0
0
Ukraine
Query!
State/province [101]
0
0
Kiev
Query!
Country [102]
0
0
Ukraine
Query!
State/province [102]
0
0
Kyiv
Query!
Country [103]
0
0
Ukraine
Query!
State/province [103]
0
0
Odesa
Query!
Country [104]
0
0
Ukraine
Query!
State/province [104]
0
0
Odessa
Query!
Country [105]
0
0
United Kingdom
Query!
State/province [105]
0
0
Birmingham
Query!
Country [106]
0
0
United Kingdom
Query!
State/province [106]
0
0
Cardiff
Query!
Country [107]
0
0
United Kingdom
Query!
State/province [107]
0
0
Liverpool
Query!
Country [108]
0
0
United Kingdom
Query!
State/province [108]
0
0
London
Query!
Country [109]
0
0
United Kingdom
Query!
State/province [109]
0
0
Manchester
Query!
Country [110]
0
0
United Kingdom
Query!
State/province [110]
0
0
Oldham
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Kastle Therapeutics, LLC
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
Primary 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. Secondary Objectives: * 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 * 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 * Obtain additional data regarding ongoing safety and efficacy of mipomersen in patients with FH and inadequately controlled LDL-C who complete the primary efficacy assessment visit (PET) in the Blinded Treatment Period and continue treatment in Open-Label Continuation Period
Query!
Trial website
https://clinicaltrials.gov/study/NCT01475825
Query!
Trial related presentations / publications
Tragante V, Asselbergs FW, Swerdlow DI, Palmer TM, Moore JH, de Bakker PIW, Keating BJ, Holmes MV. Harnessing publicly available genetic data to prioritize lipid modifying therapeutic targets for prevention of coronary heart disease based on dysglycemic risk. Hum Genet. 2016 May;135(5):453-467. doi: 10.1007/s00439-016-1647-9. Epub 2016 Mar 5.
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Medical Monitor
Query!
Address
0
0
Genzyme, a Sanofi Company
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT01475825
Download to PDF