Phase 2
N=38
Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration
Lipid Metabolism, Inborn Errors · Hyperlipidemias · Metabolic Diseases · Hypolipoproteinemia · Hypolipoproteinemias
Bottom Line
View on ClinicalTrials.gov: NCT00362180 ↗Enrolled (actual)
38
Serious AEs
2.6%
Results posted
Jun 2015
Primary outcome: Primary: Change From Baseline in Liver Triglyceride (TG) Content As Measured by Magnetic Resonance Spectroscopy (MRS) — 0.2; 1.2; 6.4; 0.8 percentage of total liver content — p=0.7244
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- mipomersen (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Kastle Therapeutics, LLC
- Primary completion
- Mar 2010
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Liver Triglyceride (TG) Content As Measured by Magnetic Resonance Spectroscopy (MRS) |
0.2; 1.2; 6.4; 0.8; 1.1; 21.4 | 0.7244 |
| SECONDARY Baseline Apolipoprotein B |
86; 100.5; 155; 122.0; 126.5; 31.5 | — |
| SECONDARY Percent Change in Apolipoprotein B From Baseline to Day 99 |
-3.3; -20.8; -41.6149; -1.1; -1.8; 3.6 | — |
| SECONDARY Baseline Low-Density Lipoprotein Cholesterol |
103.5; 126.0; 156.0; 148.0; 151.5; 41.5 | — |
| SECONDARY Percent Change in Low-Density Lipoprotein Cholesterol From Baseline to Day 99 |
1.1; -14.3; -41.7143; -2.7; -5.4; 0.0 | — |
| SECONDARY Baseline Total Cholesterol |
201.0; 207.0; 263.0; 208.0; 220.5; 107.0 | — |
| SECONDARY Percent Change in Total Cholesterol From Baseline to Day 99 |
-3.5; -11.7; -29.5374; -3.1; 0.1; 1.4 | — |
Summary
This study will assess what, if any, effect that ISIS 301012 (mipomersen) has on liver triglyceride content in multiple groups of subjects with varying degrees of risk for hepatic steatosis. In order to enroll subject groups with varying degrees of risk, the study has included multiple cohorts (Cohorts A-G). Additions and removal of cohorts has been accomplished with protocol amendments.
Eligibility Criteria
Inclusion Criteria
- Group A - are healthy subjects
- Group D - has impaired fasting glucose and mixed dyslipidemia
- Group E - has a diagnosis of Heterozygous Familial Hypercholesterolemia (HeFH) and on stable lipid-lowering therapy for 3 months
- Group F - has a diagnosis of Familial Hypobetalipoproteinemia (FHBL)
- Group G - has a diagnosis of Diabetes and hypercholesterolemia
Exclusion Criteria
- Medical, surgical, laboratory or other conditions which in the judgment of the Physician Investigator would make the subject unsuitable for enrollment, or potentially interfere with subject participation or completion of the study.
Data sourced from ClinicalTrials.gov (NCT00362180). 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.