Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
Public ClinicalTrials.gov record NCT00607373. 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 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of Mipomersen as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The purpose of this study is to evaluate the safety and efficacy of mipomersen (ISIS 301012) in subjects with homozygous familial hypercholesterolemia on lipid-lowering therapy. This study consisted of a 26-week treatment period and a 24-week post-treatment follow-up period. Following treatment and Week 28 evaluations, participants could elect to enroll in an open-label extension study (301012-CS6; NCT00694109). Participants who were not eligible or elected not to enroll in the open-label extension study or who discontinued during the 28-week treatment period were followed in this study for 24 weeks from administration of the last dose of study drug.
Study identification
- NCT ID
- NCT00607373
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 51 participants
Conditions and interventions
Conditions
- Lipid Metabolism, Inborn Errors
- Hypercholesterolemia, Autosomal Dominant
- Hyperlipidemias
- Metabolic Diseases
- Hyperlipoproteinemia Type II
- Metabolism, Inborn Errors
- Genetic Diseases, Inborn
- Infant, Newborn, Diseases
- Metabolic Disorder
- Congenital Abnormalities
- Hypercholesterolemia
- Hyperlipoproteinemias
- Dyslipidemias
- Lipid Metabolism Disorders
Eligibility (public fields only)
- Age range
- 12 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
- Jun 30, 2007
- Primary completion
- Feb 28, 2009
- Completion
- Feb 28, 2009
- Last update posted
- Sep 8, 2016
2007 – 2009
United States locations
- U.S. sites
- 2
- U.S. states
- 2
- U.S. cities
- 2
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Not listed | Charlotte | North Carolina | 28204 | — |
| Not listed | Cincinnati | Ohio | 45212 | — |
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 8 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00607373, 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 Sep 8, 2016 · Synced Sep 13, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT00607373 live on ClinicalTrials.gov.