Phase 2
N=21
Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia
Lipid Metabolism, Inborn Errors · Hypercholesterolemia, Autosomal Dominant · Hyperlipidemias · Metabolic Diseases · Hyperlipoproteinemia Type II
Bottom Line
View on ClinicalTrials.gov: NCT00477594 ↗Enrolled (actual)
21
Serious AEs
33.3%
Results posted
Apr 2013
Primary outcome: Primary: Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) — -16.2; -2.6; -32.4; 30.6 percentage of baseline
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- mipomersen sodium (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Kastle Therapeutics, LLC
- Primary completion
- Mar 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) |
-16.2; -2.6; -32.4; 30.6; -13.8; 9.5 | — |
| PRIMARY Low-density Lipoprotein Cholesterol (LDL-C) Over Time |
177; 131; 145; 127; 121; 162 | — |
| SECONDARY Percent Change From Baseline in Apolipoprotein B |
-25.9; -7.3; -27.2; 20.8; -15.8; -3.7 | — |
| SECONDARY Apolipoprotein B Over Time |
148; 124; 136; 110; 110; 128 | — |
| SECONDARY Percent Change From Baseline in Total Cholesterol |
-10.5; -6.5; -25.3; -1.5; -11.2; -1.5 | — |
| SECONDARY Total Cholesterol Over Time |
251; 233; 216; 203; 209; 196 | — |
| SECONDARY Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol |
-24.7; -6.7; -34.3; 17.2; -16.5; 3.7 | — |
| SECONDARY Non-High-Density Lipoprotein Cholesterol Over Time |
206; 151; 167; 150; 161; 177 | — |
| SECONDARY Number of Participants With Treatment-Emergent Adverse Events (AEs) |
16; 5; 16; 5; 5; 2 | — |
| SECONDARY Percent Change From Baseline in Clinical Chemistry Parameters |
63.73; 29.96; 48.73; 3.53; 13.81; 12.50 | — |
| SECONDARY Percent Change From Baseline in Hematology Parameters |
-0.04; -5.24; 0.90; -4.24; -0.80; -5.99 | — |
| SECONDARY Percent Change From Baseline in Blood Pressure |
6.99; 2.35; -0.88; 10.67 | — |
| SECONDARY Percent Change From Baseline in Pulse Rate |
4.89; 6.28 | — |
| SECONDARY Percent Change From Baseline in Respiratory Rate |
0.87; -4.44 | — |
Summary
The purpose of this study is to evaluate the safety and efficacy of extended dosing of mipomersen in patients with familial hypercholesterolemia on lipid-lowering therapy who have completed either the 301012-CS8 (NCT00280995) or 301012-CS9 (NCT00281008) clinical drug trials.
Eligibility Criteria
Inclusion Criteria
- Satisfactory completion of dosing and Week 7 or Week 15 assessments (depending on the treatment and dose received) in their initial study (Protocol 301012-CS8 (NCT00280995) or 301012-CS9 (NCT00281008)).
Exclusion Criteria
- Have a new condition or worsening of existing condition which in the opinion of the Investigator would make the patient unsuitable for enrollment, or could interfere with patient's participation in or completion of the study.
Data sourced from ClinicalTrials.gov (NCT00477594). 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.