Phase 3
N=142
An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia
Lipid Metabolism, Inborn Errors · Hypercholesterolemia, Autosomal Dominant · Hyperlipidemias · Metabolic Diseases · Hyperlipoproteinemia Type II
Bottom Line
View on ClinicalTrials.gov: NCT00694109 ↗Enrolled (actual)
142
Serious AEs
25.5%
Results posted
Dec 2015
Primary outcome: Primary: Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) — -28.5; -27; -27.3; -27.9 percent change
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Mipomersen Sodium (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Kastle Therapeutics, LLC
- Primary completion
- Sep 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) |
-28.5; -27; -27.3; -27.9; -21.9; -21.4 | — |
| PRIMARY Percent Change From Baseline in Apolipoprotein B (Apo B) |
-28.9; -28.1; -30.3; -31.2; -29.1; -30.2 | — |
| PRIMARY Percent Change From Baseline in Total Cholesterol |
-21.7; -20.4; -20.1; -19.8; -14.9; -14.4 | — |
| PRIMARY Percent Change From Baseline in Non High Density Lipoprotein Cholesterol (Non-HDL-C) |
-27.2; -25.4; -25; -26.2; -20.7; -20 | — |
| SECONDARY Percent Change From Baseline in Triglycerides |
-20.1; -7.9; -10.2; -12.5; -10.9; -10.4 | — |
| SECONDARY Percent Change From Baseline in Lipoprotein (a) |
-20.5; -19; -17.9; -16.6; -15.8; -9.1 | — |
| SECONDARY Percent Change From Baseline in LDL Particles' Size (Total) |
-26.77; -27.77; -25.1; -32.65; -22.63; 6.11 | — |
| SECONDARY Percent Change From Baseline in LDL Particles' Size (Large) |
-5.01; -14.32; -27.04; -22.67; -2.94; 6.19 | — |
| SECONDARY Percent Change From Baseline in LDL Particles' Size (Medium) |
-9.50; 11.09; -19.62; -15.82; -5.65; 46.92 | — |
| SECONDARY Percent Change From Baseline in LDL Particles' Size (Small) |
-8.79; 1.72; -18.95; -27.95; -5.17; 51.94 | — |
| SECONDARY Percent Change From Baseline in LDL Particles' Size (Very Small) |
-5.05; -0.11; -18.7; -30.77; 0.75; 60.22 | — |
| SECONDARY Percent Change From Baseline in HDL Particles' Size (Large) |
160.8; 43.23; 58.26; 61.76; 121.16; 85.93 | — |
| SECONDARY Percent Change From Baseline in HDL Particles' Size (Medium) |
154.77; 176.14; 21.24; 838.32; 388.16; 233.78 | — |
| SECONDARY Percent Change From Baseline in HDL Particles' Size (Small) |
1.83; -9.81; -14.18; -11.47; 0.44; 8.31 | — |
| SECONDARY Percent Change From Baseline in Intermediate Density Lipoprotein Particles' Size |
-9.9; -27.35; 155.42; 32.88; 24.66; 57.46 | — |
| SECONDARY Percent Change From Baseline in Very Low Density Lipoprotein (VLDL) Particles' Size (Large) and Chylomicron Particles' Size |
109.23; 107.5; 123.42; 241.76; 86.75; 90.82 | — |
| SECONDARY Percent Change From Baseline in VLDL Particles' Size (Medium) |
70.81; 97.74; 172.46; 98.7; 63.25; 99.57 | — |
| SECONDARY Percent Change From Baseline in VLDL Particles' Size (Small) |
49.51; 30.48; 9.34; -30.36; 31.27; 32.14 | — |
| SECONDARY Percent Change From Baseline in Total VLDL Particles' Size and Chylomicron Particles' Size |
19.94; -14.25; 3.48; -18.66; 12.82; 19.69 | — |
| SECONDARY Change From Baseline in C-Reactive Protein |
0.67; -0.37; -1.05; 0.12; -0.18; 0.02 | — |
| SECONDARY Percent Change From Baseline in Apolipoprotein A-1 |
-1.01; -1.59; -3.73; -4.33; -1.37; -5.55 | — |
Summary
To evaluate the safety and efficacy of extended dosing with mipomersen (ISIS 301012) in participants with familial hypercholesterolemia or severe hypercholesterolemia on lipid-lowering therapy who had completed either the 301012-CS5 (NCT00607373), 301012-CS7 (NCT00706849), 301012-CS17 (NCT00477594) or MIPO3500108 (NCT00794664) clinical drug trials.
Eligibility Criteria
Inclusion Criteria
- Satisfactory completion of dosing in their initial study (Protocol 301012-CS5 [NCT00607373], 301012-CS7 [NCT00706849], 301012-CS17 [NCT00477594], or MIPO3500108 [NCT00794664])
Exclusion Criteria
- Had any new condition or worsening of existing condition which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study
Data sourced from ClinicalTrials.gov (NCT00694109). 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.