Phase 3
N=51
Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
Lipid Metabolism, Inborn Errors · Hypercholesterolemia, Autosomal Dominant · Hyperlipidemias · Metabolic Diseases · Hyperlipoproteinemia Type II
Bottom Line
View on ClinicalTrials.gov: NCT00607373 ↗Enrolled (actual)
51
Serious AEs
5.9%
Results posted
Mar 2013
Primary outcome: Primary: Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Primary Efficacy Time Point — -3.31; -24.66 percentage of baseline — p=<0.001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- mipomersen (Drug); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Kastle Therapeutics, LLC
- Primary completion
- Mar 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Primary Efficacy Time Point |
-3.31; -24.66 | <0.001 sig |
| PRIMARY LDL-C at Baseline and the Primary Efficacy Time Point (PET) |
400.2; 438.9; 388.2; 326.2 | — |
| SECONDARY Percent Change From Baseline in Apolipoprotein B (Apo-B) at Primary Efficacy Time Point |
-2.54; -26.77 | <0.001 sig |
| SECONDARY Apo-B at Baseline and the Primary Efficacy Time Point (PET) |
259.2; 283.1; 252.6; 205.4 | — |
| SECONDARY Percentage Change From Baseline in Total Cholesterol at Primary Efficacy Time Point (PET) |
-1.98; -21.20 | <0.001 sig |
| SECONDARY Total Cholesterol at Baseline and the Primary Efficacy Time Point (PET) |
460.5; 502.4; 452.1; 389.7 | — |
| SECONDARY Percentage Change From Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Primary Efficacy Time Point (PET) |
-2.90; -24.50 | <0.001 sig |
| SECONDARY Non-HDL-C at Baseline and the Primary Efficacy Time Point (PET) |
418.9; 464.3; 409.1; 345.8 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- Diagnosis of Homozygous Familial Hypercholesterolemia (HoFH)
- Stable lipid-lowering therapy for 12 weeks
- Stable weight for 6 weeks
- Stable low fat diet for 8 weeks
Exclusion Criteria
- Significant health problems in the recent past including heart attack, stroke, blood disorders, cancer, or digestive problems
Data sourced from ClinicalTrials.gov (NCT00607373). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.