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
NCT ID: NCT01475825
Last Updated: 2019-03-26
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
309 participants
INTERVENTIONAL
2011-12-31
2015-12-29
Brief Summary
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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
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Detailed Description
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Study Design, masking - Study treatment was blinded (double-blinded) through the Primary Efficacy Assessment Visit in the Blinded Treatment Period. Study treatment was open-label in the Open-Label Continuation Period.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Regimen A: Mipomersen
Subcutaneous injection of mipomersen 200 mg once weekly
mipomersen sodium 200 mg
Subcutaneous mipomersen 200 mg once weekly
Regimen A: Placebo
Placebo matching subcutaneous injection once weekly.
Placebo
Placebo vehicle for subcutaneous injection.
Regimen B: Mipomersen
Subcutaneous injection of mipomersen 70 mg thrice weekly.
mipomersen sodium 70 mg
Subcutaneous mipomersen 70 mg thrice weekly
Regimen B: Placebo
Placebo matching subcutaneous injection thrice weekly.
Placebo
Placebo vehicle for subcutaneous injection.
Interventions
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mipomersen sodium 200 mg
Subcutaneous mipomersen 200 mg once weekly
Placebo
Placebo vehicle for subcutaneous injection.
mipomersen sodium 70 mg
Subcutaneous mipomersen 70 mg thrice weekly
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* Apheresis within 3 months prior to Screening or expected to start apheresis during the treatment phase
18 Years
ALL
No
Sponsors
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Kastle Therapeutics, LLC
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Monitor
Role: STUDY_DIRECTOR
Genzyme, a Sanofi Company
Locations
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Mission Viejo, California, United States
Aurora, Colorado, United States
Cooper City, Florida, United States
Winter Park, Florida, United States
Indianapolis, Indiana, United States
Kansas City, Kansas, United States
Boston, Massachusetts, United States
Ann Arbor, Michigan, United States
Grandville, Michigan, United States
Rochester, Minnesota, United States
St Louis, Missouri, United States
Omaha, Nebraska, United States
Summit, New Jersey, United States
North Massapequa, New York, United States
Portland, Oregon, United States
Lancaster, Pennsylvania, United States
Charleston, South Carolina, United States
Nashville, Tennessee, United States
Dallas, Texas, United States
Norfolk, Virginia, United States
Buenos Aires, , Argentina
Córdoba, , Argentina
Perth, , Australia
South Brisbane, , Australia
Edegem, , Belgium
Haine-Saint-Paul, , Belgium
Leuven, , Belgium
Rio de Janeiro, , Brazil
São Paulo, , Brazil
Chicoutimi, Quebec, Canada
Montreal, Quebec, Canada
Sainte-Foy, Quebec, Canada
Osijek, , Croatia
Zagreb, , Croatia
Hradec Králové, , Czechia
Prague, , Czechia
Aarhus, , Denmark
Viborg, , Denmark
Aachen, , Germany
Berlin, , Germany
Cologne, , Germany
Freiburg im Breisgau, , Germany
Hamburg, , Germany
Heidelberg, , Germany
Magdeburg, , Germany
Ioannina, , Greece
Kallithea, , Greece
Hong Kong, , Hong Kong
Baja, , Hungary
Budapest, , Hungary
Debrecen, , Hungary
New Delhi, , India
Holon, , Israel
Kfar Saba, , Israel
Ofakim, , Israel
Bologna, , Italy
Napoli, , Italy
Padua, , Italy
Palermo, , Italy
Pisa, , Italy
Roma, , Italy
Kuala Lumpur, , Malaysia
Kubang Kerian, , Malaysia
Alkmaar, , Netherlands
Amsterdam, , Netherlands
Maastricht, , Netherlands
Nijmegen, , Netherlands
Utrecht, , Netherlands
Waalwijk, , Netherlands
Christchurch, , New Zealand
Bodø, , Norway
Oslo, , Norway
Sandefjord, , Norway
Bialystok, , Poland
Gdansk, , Poland
Katowice, , Poland
Krakow, , Poland
Nałęczów, , Poland
Poznan, , Poland
Sopot, , Poland
Szczecin, , Poland
Warsaw, , Poland
Wroclaw, , Poland
Barnaul, , Russia
Kemerovo, , Russia
Moscow, , Russia
Novosibirsk, , Russia
Petrozavodsk, , Russia
Ryazan, , Russia
Saint Petersburg, , Russia
Tomsk, , Russia
Yaroslavl, , Russia
Cape Town, , South Africa
Pretoria, , South Africa
Seoul, , South Korea
Madrid, , Spain
Stockholm, , Sweden
New Taipei City, , Taiwan
Taipei, , Taiwan
Ankara, , Turkey (Türkiye)
Istanbul, , Turkey (Türkiye)
Izmir, , Turkey (Türkiye)
Sivas, , Turkey (Türkiye)
Ivano-Frankivsk, , Ukraine
Kiev, , Ukraine
Kyiv, , Ukraine
Odesa, , Ukraine
Birmingham, , United Kingdom
Cardiff, , United Kingdom
Liverpool, , United Kingdom
London, , United Kingdom
Manchester, , United Kingdom
Oldham, , United Kingdom
Countries
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References
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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.
Other Identifiers
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2011-001480-42
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
EFC12875
Identifier Type: OTHER
Identifier Source: secondary_id
MIPO3801011
Identifier Type: -
Identifier Source: org_study_id
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