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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
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
Secondary ID [1] 0 0
2011-001480-42
Secondary ID [2] 0 0
MIPO3801011
Universal Trial Number (UTN)
Trial acronym
FOCUS FH
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hypercholesterolemia 0 0
Heterozygous Familial 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percent Change From Baseline To Primary Endpoint Visit (PET) In LDL-C In Cohort 1
Timepoint [1] 0 0
Baseline and Week 61
Secondary outcome [1] 0 0
Percent Change From Baseline To PET In LDL-C In Cohort 2
Timepoint [1] 0 0
Baseline, PET (up to 60 weeks)
Secondary outcome [2] 0 0
Percent Change From Baseline To PET In Apolipoprotein B (Apo B) In Cohort 1
Timepoint [2] 0 0
Baseline and Week 61
Secondary outcome [3] 0 0
Percent Change From Baseline To PET In Apolipoprotein B (Apo B) In Cohort 2
Timepoint [3] 0 0
Baseline and Week 61
Secondary outcome [4] 0 0
Percent Change From Baseline To PET in Lipoprotein (a) In Cohort 1
Timepoint [4] 0 0
Baseline and Week 61
Secondary outcome [5] 0 0
Percent Change From Baseline To PET in Lipoprotein (a) In Cohort 2
Timepoint [5] 0 0
Baseline and Week 61

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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
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
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/12/2011
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
29/12/2015
Sample size
Target
Accrual to date
Final
309
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
- Perth
Recruitment hospital [2] 0 0
- South Brisbane
Recruitment postcode(s) [1] 0 0
- Perth
Recruitment postcode(s) [2] 0 0
- South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Indiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Kansas
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
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United States of America
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Minnesota
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United States of America
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Missouri
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United States of America
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Nebraska
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United States of America
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New Jersey
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United States of America
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New York
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United States of America
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Oregon
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United States of America
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Pennsylvania
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United States of America
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South Carolina
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United States of America
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Tennessee
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United States of America
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Texas
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United States of America
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Virginia
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Argentina
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Ciudad Autonoma de Buenos Aires
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Argentina
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Cordoba
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Belgium
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Edegem
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Belgium
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Haine St. Paul
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Belgium
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Leuven
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Brazil
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Rio de Janeiro
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Brazil
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Sao Paulo
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Canada
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Quebec
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Croatia
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Osijek
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Croatia
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Zagreb
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Czechia
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Hradec Kralove
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Czechia
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Praha 1
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Denmark
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Aarhus
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Denmark
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Viborg
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Germany
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Aachen
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Germany
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Berlin
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Germany
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Freiburg
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Germany
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Hamburg
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Germany
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Heidelberg
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Germany
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Koeln
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Germany
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Magdeburg
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Greece
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Ioannina
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Greece
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Kallithea
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Hong Kong
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Hong Kong
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Hungary
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Baja
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Hungary
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Budapest
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Hungary
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Debrecen
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India
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New Delhi
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Israel
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Holon
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Israel
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Kfar Saba
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Israel
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Ofakim
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Italy
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Bologna
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Italy
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Napoli
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Italy
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Padova
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Italy
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Palermo
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Italy
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Pisa
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Italy
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Roma
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Korea, Republic of
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Seoul
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Malaysia
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Kuala Lumpur
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Malaysia
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Kubang Kerian
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Netherlands
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Alkmaar
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Netherlands
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Amsterdam
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Netherlands
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Maastricht
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Netherlands
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Nijmegen
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Utrecht
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Waalwijk
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New Zealand
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Christchurch
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Norway
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Bodo
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Norway
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Oslo
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Norway
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Sandefjord
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Poland
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Bialystok
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Gdansk
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Katowice
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Krakow
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Naleczow
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Poznan
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Sopot
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Szczecin
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Warszawa
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Poland
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Wroclaw
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Russian Federation
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Barnaul
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Russian Federation
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Kemerovo
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Russian Federation
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Moscow
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Russian Federation
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Novosibirsk
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Russian Federation
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Petrozavodsk
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Russian Federation
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Ryazan
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Russian Federation
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Saint Petersburg
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Russian Federation
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St-Petersburg
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Russian Federation
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St. Petersburg
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Russian Federation
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Tomsk
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Russian Federation
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Yaroslavl
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South Africa
State/province [90] 0 0
Cape Town
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South Africa
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Pretoria
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Spain
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Madrid
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Sweden
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Stockholm
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Taiwan
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New Taipei City
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Taiwan
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Taipei
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Turkey
State/province [96] 0 0
Ankara
Country [97] 0 0
Turkey
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Istanbul
Country [98] 0 0
Turkey
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Izmir
Country [99] 0 0
Turkey
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Sivas
Country [100] 0 0
Ukraine
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Ivano-Frankivsk
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Ukraine
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Kiev
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Ukraine
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Kyiv
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Ukraine
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Odesa
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Ukraine
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Odessa
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United Kingdom
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Birmingham
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United Kingdom
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Cardiff
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United Kingdom
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Liverpool
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United Kingdom
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London
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United Kingdom
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Manchester
Country [110] 0 0
United Kingdom
State/province [110] 0 0
Oldham

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Kastle Therapeutics, LLC
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Monitor
Address 0 0
Genzyme, a Sanofi Company
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
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