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
Public ClinicalTrials.gov record NCT01475825. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official 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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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
Study identification
- NCT ID
- NCT01475825
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 309 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Dec 2011 (Actual)
- Primary completion
- Dec 29, 2015 (Actual)
- Completion
- Dec 29, 2015 (Actual)
- Last update posted
- Mar 26, 2019
2011 – 2015
United States locations
- U.S. sites
- 20
- U.S. states
- 18
- U.S. cities
- 20
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Not listed | Mission Viejo | California | — | — |
| Not listed | Aurora | Colorado | — | — |
| Not listed | Cooper City | Florida | — | — |
| Not listed | Winter Park | Florida | — | — |
| Not listed | Indianapolis | Indiana | — | — |
| Not listed | Kansas City | Kansas | — | — |
| Not listed | Boston | Massachusetts | — | — |
| Not listed | Ann Arbor | Michigan | — | — |
| Not listed | Grandville | Michigan | — | — |
| Not listed | Rochester | Minnesota | — | — |
| Not listed | St Louis | Missouri | — | — |
| Not listed | Omaha | Nebraska | — | — |
| Not listed | Summit | New Jersey | — | — |
| Not listed | North Massapequa | New York | — | — |
| Not listed | Portland | Oregon | — | — |
| Not listed | Lancaster | Pennsylvania | — | — |
| Not listed | Charleston | South Carolina | — | — |
| Not listed | Nashville | Tennessee | — | — |
| Not listed | Dallas | Texas | — | — |
| Not listed | Norfolk | Virginia | — | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 93 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01475825, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Mar 26, 2019 · Synced Oct 23, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT01475825 live on ClinicalTrials.gov.