A Phase 3 interventional study of Mipomersen Sodium in Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant and Hyperlipidemias, sponsored by Kastle Therapeutics, LLC. Completed at 33 sites in 7 countries. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2016-09-09.
Sponsored by Kastle Therapeutics, LLC · Phase 3, Interventional, and Treatment
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.
All familial hypercholesterolemia (FH) or severe hypercholesterolemia participants who had tolerated the treatment regimen in Protocol 301012-CS5 (NCT00607373), 301012-CS7 (NCT00706849) or MIPO3500108 (NCT00794664) and satisfactorily completed the study through to Week 28 were eligible for participation in this open label treatment extension study for up to 4 years or until mipomersen was commercially available, whichever comes first. Consenting participants who had tolerated mipomersen and satisfactorily completed 301012-CS17 (NCT00477594) through Year 3 may also enroll for up to an additional 2 years of treatment in this study or until mipomersen was commercially available, whichever comes first. All participants, who entered the study, received 200 mg mipomersen (ISIS 301012) subcutaneously (s.c.) every week, including those who were randomized to placebo in their initial study. Participants who were originally enrolled in Protocol 301012-CS5 (NCT00607373) and weighed \<50 kg received 160 mg every week. Dose adjustments (70 mg injections administered three times per week, on separate days) were allowed for participants who were not tolerating or who had previous issues with tolerating the once a week injections due to injection site reactions (ISRs) or flu-like symptoms. Study visits and clinical lab assessments including hematology with differential, chemistry, serum lipid panel (total cholesterol, LDL-C, very low density lipoprotein cholesterol (VLDL-C), high density lipoprotein cholesterol (HDL-C), apolipoprotein B (apoB), apoA-1, triglycerides (TG) and Lp(a), and urinalysis was to be performed every 4-10 weeks during the treatment period. Plasma trough mipomersen (ISIS 301012) levels was to be measured to estimate exposure. Participants who completed dosing or who discontinued prematurely from the study for any reason was followed for safety for 24 weeks (safety follow-up period) after their last dose of mipomersen (ISIS 301012) or longer in the case of a significant adverse events (AE) or abnormal biochemical or clinical finding. Participants were required to return to the study center for clinical evaluation and clinical laboratory tests every 8 weeks during the safety follow-up period.
26 studies on the registry are indexed under Lipid Metabolism, Inborn Errors; 2 are open to participants now.
This study's enrollment of 144 is above the median of 21 across 21 interventional studies indexed under Lipid Metabolism, Inborn Errors.
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Exclusion Criteria:
Mipomersen Sodium once a week for up to 4 years (depending on participant's consent). Participants were followed for additional 24 week post-treatment.
Drug: Mipomersen Sodium
Subcutaneous injection as a single injection directly into the abdomen, thigh, or outer area of the upper arm.
Also known as: ISIS 301012, Kynamro®
Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C)
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in Apolipoprotein B (Apo B)
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in Total Cholesterol
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in Triglycerides
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in Lipoprotein (a)
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in LDL Particles' Size (Total)
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in LDL Particles' Size (Large)
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in LDL Particles' Size (Medium)
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in LDL Particles' Size (Small)
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in LDL Particles' Size (Very Small)
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in HDL Particles' Size (Large)
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in HDL Particles' Size (Medium)
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in HDL Particles' Size (Small)
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in Intermediate Density Lipoprotein Particles' Size
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in Very Low Density Lipoprotein (VLDL) Particles' Size (Large) and Chylomicron Particles' Size
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in VLDL Particles' Size (Medium)
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in VLDL Particles' Size (Small)
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in Total VLDL Particles' Size and Chylomicron Particles' Size
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years)
Change From Baseline in C-Reactive Protein
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to End of treatment; 24 weeks post treatment (up to 4.5 years)
Percent Change From Baseline in Apolipoprotein A-1
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
Time frame: Baseline up to Week 234; 24 weeks post treatment (up to 4.5 years)
The study was conducted at 33 centers in 7 countries. A total of 144 patients were enrolled in the study. 1 patient never received study drug. 2 of the enrolled patients came from a phase 2 study and its extension and consequently had very different treatment from the other treated patients, and thus were excluded from all summary tables.
| Milestone | Mipomersen |
|---|---|
| Started | 142 |
| Treated | 141 |
| Consented 2 years additional treatment | 42 |
| Completed consented length of treatment | 60 |
| Completed | 25 |
| Not completed | 117 |
| Withdrew: Physician decision | 3 |
| Withdrew: Adverse event | 74 |
| Withdrew: Not consent of more 2 years of treatment | 18 |
| Withdrew: Lack of efficacy | 2 |
| Withdrew: Withdrawal by subject | 13 |
| Withdrew: Pregnancy | 1 |
| Withdrew: Enrolled but not treated | 1 |
| Withdrew: Other | 2 |
| Withdrew: Consented but no additional treatment | 3 |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 26 (n = 130) | -28.5 (-31.9 to -25.1) |
| week 52 (n = 111) | -27 (-31.2 to -22.8) |
| week 76 (n = 66) | -27.3 (-33 to -21.6) |
| week 104 (n = 57) | -27.9 (-33.9 to -21.8) |
| week 130 (n = 42) | -21.9 (-31.1 to -12.7) |
| week 156 (n = 30) | -21.4 (-31.2 to -11.7) |
| week 182 (n = 26) | -23.6 (-36.6 to -10.6) |
| week 208 (n = 27) | -26.3 (-36.4 to -16.2) |
| week 234 (n = 17) | -22.5 (-34.3 to -10.6) |
| 24 weeks post last dose (n=117) | 1.6 (-2.6 to 5.9) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 26 (n = 130) | -28.9 (-32 to -25.8) |
| week 52 (n = 111) | -28.1 (-32 to -24.2) |
| week 76 (n = 66) | -30.3 (-34.7 to -26) |
| week 104 (n = 57) | -31.2 (-36.5 to -25.9) |
| week 130 (n = 43) | -29.1 (-35.7 to -22.5) |
| week 156 (n = 30) | -30.2 (-38.1 to -22.2) |
| week 182 (n = 26) | -31.1 (-39.9 to -22.2) |
| week 208 (n = 27) | -33.3 (-40.8 to -25.9) |
| week 234 (n = 17) | -31.4 (-38.7 to -24.1) |
| 24 weeks post last dose (n=117) | -3.46 (-6.9 to 0) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 26 (n = 130) | -21.7 (-24.4 to -18.9) |
| week 52 (n = 111) | -20.4 (-23.9 to -16.8) |
| week 76 (n = 66) | -20.1 (-24.6 to -15.5) |
| week 104 (n = 57) | -19.8 (-24.8 to -14.7) |
| week 130 (n = 43) | -14.9 (-22.1 to -7.8) |
| week 156 (n = 30) | -14.4 (-22.3 to -6.6) |
| week 182 (n = 26) | -14.3 (-25 to -3.5) |
| week 208 (n = 27) | -16.5 (-24.2 to -8.8) |
| week 234 (n = 17) | -12.5 (-21.5 to -3.4) |
| 24 weeks post last dose (n=117) | 1.94 (-1.5 to 5.4) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 26 (n = 130) | -27.2 (-30.4 to -24.1) |
| week 52 (n = 111) | -25.4 (-29.5 to -21.3) |
| week 76 (n = 66) | -25 (-30.4 to -19.7) |
| week 104 (n = 57) | -26.2 (-32 to -20.4) |
| week 130 (n = 43) | -20.7 (-29.1 to -12.3) |
| week 156 (n = 30) | -20 (-29.6 to -10.3) |
| week 182 (n = 26) | -21.7 (-34.7 to -8.7) |
| week 208 (n = 27) | -23.9 (-33.7 to -14.1) |
| week 234 (n = 17) | -19.9 (-31.5 to -8.2) |
| 24 weeks post last dose (n=117) | 2.5 (-1.8 to 6.7) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 26 (n = 130) | -20.1 (-33.1 to -1.2) |
| week 52 (n = 111) | -7.9 (-31.5 to 16.9) |
| week 76 (n = 66) | -10.2 (-27.7 to 13.8) |
| week 104 (n = 57) | -12.5 (-37.1 to 7.2) |
| week 130 (n = 43) | -10.9 (-36 to 10) |
| week 156 (n = 30) | -10.4 (-23.8 to 12.7) |
| week 182 (n = 26) | -12.9 (-27.4 to -1.6) |
| week 208 (n = 27) | -13.9 (-40 to 33) |
| week 234 (n = 17) | 1.3 (-15.4 to 15.7) |
| 24 weeks post last dose (n=117) | 2.1 (-17.2 to 27.7) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 26 (n = 130) | -20.5 (-39.3 to -3.6) |
| week 52 (n = 111) | -19 (-33.3 to 0) |
| week 76 (n = 66) | -17.9 (-33.3 to -0.5) |
| week 104 (n = 57) | -16.6 (-36.1 to 0) |
| week 130 (n = 43) | -15.8 (-31.3 to 0) |
| week 156 (n = 30) | -9.1 (-33.8 to 7.3) |
| week 182 (n = 26) | -9 (-27.2 to 7.6) |
| week 208 (n = 27) | -9.9 (-32.5 to 4.1) |
| week 234 (n = 17) | -18.3 (-31.6 to -4.8) |
| 24 weeks post last dose (n=117) | 0 (-6 to 5) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 52 (n = 91) | -26.77 (-32.7 to -20.8) |
| week 104 (n = 47) | -27.77 (-35.3 to -20.3) |
| week 156 (n = 20) | -25.1 (-40.3 to -9.9) |
| week 208 (n = 19) | -32.65 (-44.9 to -20.4) |
| End of treatment (n=139) | -22.63 (-27 to -18.3) |
| 24 weeks post last dose (n=115) | 6.11 (0.8 to 11.5) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 52 (n = 91) | -5.01 (-16.8 to 6.8) |
| week 104 (n = 47) | -14.32 (-27 to -1.6) |
| week 156 (n = 20) | -27.04 (-40.6 to -13.4) |
| week 208 (n = 19) | -22.67 (-41.6 to -3.8) |
| End of treatment (n=139) | -2.94 (-13.2 to 7.3) |
| 24 weeks post last dose (n=115) | 6.19 (-6.1 to 18.5) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 52 (n = 91) | -9.50 (-31.8 to 12.8) |
| week 104 (n = 47) | 11.09 (-42.2 to 64.4) |
| week 156 (n = 20) | -19.62 (-57.3 to 18) |
| week 208 (n = 19) | -15.82 (-62 to 30.4) |
| End of treatment (n=139) | -5.65 (-30.3 to 19) |
| 24 weeks post last dose (n=115) | 46.92 (5.6 to 88.2) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 52 (n = 91) | -8.79 (-33.7 to 16.2) |
| week 104 (n = 47) | 1.72 (-43.9 to 47.4) |
| week 156 (n = 20) | -18.95 (-58.8 to 20.9) |
| week 208 (n = 19) | -27.95 (-67.9 to 12) |
| End of treatment (n=139) | -5.17 (-29.2 to 18.8) |
| 24 weeks post last dose (n=115) | 51.94 (7.7 to 96.2) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 52 (n = 91) | -5.05 (-32.2 to 22.2) |
| week 104 (n = 47) | -0.11 (-44.4 to 44.2) |
| week 156 (n = 20) | -18.7 (-59.2 to 21.8) |
| week 208 (n = 19) | -30.77 (-69.3 to 7.8) |
| End of treatment (n=139) | 0.75 (-28 to 29.5) |
| 24 weeks post last dose (n=115) | 60.22 (7.5 to 112.9) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 52 (n = 89) | 160.8 (-38.3 to 359.9) |
| week 104 (n = 47) | 43.23 (-12.2 to 98.7) |
| week 156 (n = 20) | 58.26 (-38 to 154.6) |
| week 208 (n = 19) | 61.76 (-41.6 to 165.2) |
| End of treatment (n=134) | 121.16 (-17.3 to 259.6) |
| 24 weeks post last dose (n=110) | 85.93 (-7.3 to 179.1) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 52 (n = 44) | 154.77 (2.8 to 306.8) |
| week 104 (n = 28) | 176.14 (-68.6 to 420.9) |
| week 156 (n = 9) | 21.24 (-65.1 to 107.6) |
| week 208 (n = 8) | 838.32 (-1109.3 to 2785.9) |
| End of treatment (n= 68) | 388.16 (94.5 to 681.8) |
| 24 weeks post last dose (n=56) | 233.78 (7.9 to 459.7) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 52 (n = 91) | 1.83 (-7.3 to 10.9) |
| week 104 (n = 47) | -9.81 (-17.7 to -2) |
| week 156 (n = 20) | -14.18 (-25.1 to -3.2) |
| week 208 (n = 19) | -11.47 (-20 to -2.9) |
| End of treatment (n= 139) | 0.44 (-6.9 to 7.7) |
| 24 weeks post last dose (n=115) | 8.31 (0.6 to 16) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 52 (n = 79) | -9.9 (-45.6 to 25.8) |
| week 104 (n = 40) | -27.35 (-66.5 to 11.8) |
| week 156 (n = 16) | 155.42 (-90.1 to 401) |
| week 208 (n = 15) | 32.88 (-104 to 169.8) |
| End of treatment (n= 122) | 24.66 (-28.4 to 77.8) |
| 24 weeks post last dose (n=101) | 57.46 (15.2 to 99.8) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 52 (n = 86) | 109.23 (33.7 to 184.8) |
| week 104 (n = 46) | 107.5 (-10.2 to 225.2) |
| week 156 (n = 19) | 123.42 (-113 to 359.8) |
| week 208 (n = 18) | 241.76 (-241.5 to 725.1) |
| End of treatment (n= 132) | 86.75 (28.4 to 145.1) |
| 24 weeks post last dose (n=110) | 90.82 (21.8 to 159.9) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 52 (n = 88) | 70.81 (2.1 to 139.5) |
| week 104 (n = 47) | 97.74 (-21 to 216.5) |
| week 156 (n = 20) | 172.46 (5.3 to 339.7) |
| week 208 (n = 19) | 98.7 (-71.3 to 268.7) |
| End of treatment (n= 136) | 63.25 (12.1 to 114.4) |
| 24 weeks post last dose (n=113) | 99.57 (16.8 to 182.3) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 52 (n = 91) | 49.51 (-41.1 to 140.4) |
| week 104 (n = 47) | 30.48 (-75.1 to 136.1) |
| week 156 (n = 20) | 9.34 (-48.3 to 67) |
| week 208 (n = 19) | -30.36 (-49.8 to -10.9) |
| End of treatment (n= 139) | 31.27 (-27.3 to 89.8) |
| 24 weeks post last dose (n=115) | 32.14 (-5.3 to 69.6) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 52 (n = 91) | 19.94 (-36.7 to 74.6) |
| week 104 (n = 47) | -14.25 (-36.4 to 7.9) |
| week 156 (n = 20) | 3.48 (-27.5 to 34.5) |
| week 208 (n = 19) | -18.66 (-43.6 to 6.3) |
| End of treatment (n= 139) | 12.82 (-25.5 to 51.2) |
| 24 weeks post last dose (n=115) | 19.69 (3.2 to 36.1) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 26 (n=130) | 0.67 (-0.8 to 2.1) |
| week 52 (n=111) | -0.37 (-1.4 to 0.6) |
| week 76 (n=84) | -1.05 (-2 to -0.1) |
| week 104 (n=58) | 0.12 (-0.8 to 1) |
| week 130 (n=42) | -0.18 (-1.1 to 0.8) |
| week 156 (n=30) | 0.02 (-0.5 to 2.1) |
| week 182 (n=31) | 0.73 (0.1 to 1.4) |
| week 208 (n=27) | 0.2 (-0.5 to 0.9) |
| week 234 (n=18) | 0.53 (-0.5 to 1.5) |
| End of treatment (n=140) | 0.41 (-0.6 to 1.4) |
| 24 weeks post last dose (n=116) | 0.09 (-0.9 to 1.1) |
Baseline was defined as the last value prior to receiving the first dose of mipomersen in this study (for participants randomized to placebo in their index study and for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \>=6 months from their last dose of mipomersen in their index study), or the last value prior to receiving the first dose of mipomersen in their index study (for participants randomized to mipomersen in their index study and their first dose of mipomersen in this study was administered \<6 months from their last dose of mipomersen in their index study).
| percent change | Mipomersen |
|---|---|
| week 26 (n=130) | -1.01 (-3.8 to 1.7) |
| week 52 (n=111) | -1.59 (-4.7 to 1.6) |
| week 76 (n=66) | -3.73 (-7.9 to 0.5) |
| week 104 (n=57) | -4.33 (-9.1 to 0.4) |
| week 130 (n=43) | -1.37 (-6.1 to 3.4) |
| week 156 (n=30) | -5.55 (-11.2 to 0) |
| week 182 (n=26) | -3.17 (-9.4 to 3.1) |
| week 208 (n=27) | -2.19 (-7.2 to 2.8) |
| week 234 (n=17) | 3.68 (-3 to 10.3) |
| 24 weeks post last dose (n = 117) | -0.67 (-3.5 to 2.2) |
Collected over All Adverse Events (AE) were collected from signature of the informed consent form up to the final visit (59.7 months) regardless of seriousness or relationship to investigational product.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Mipomersen | — | 36/141 (25.5%) | 141/141 (100%) |
| Event | Mipomersen |
|---|---|
| Non-cardiac chest painGeneral disorders | 4/141 |
| Angina pectorisCardiac disorders | 3/141 |
| Atrial fibrillationCardiac disorders | 3/141 |
| Coronary artery diseaseCardiac disorders | 3/141 |
| Acute myocardial infarctionCardiac disorders | 2/141 |
| Aortic valve stenosisCardiac disorders | 2/141 |
| Ankle fractureInjury, poisoning and procedural complications | 2/141 |
| SyncopeNervous system disorders | 2/141 |
| Splenic haemorrhageBlood and lymphatic system disorders | 1/141 |
| Acute coronary syndromeCardiac disorders | 1/141 |
| Event | Mipomersen |
|---|---|
| Injection site erythemaGeneral disorders | 117/141 |
| Injection site painGeneral disorders | 102/141 |
| Injection site bruisingGeneral disorders | 72/141 |
| Influenza like illnessGeneral disorders | 70/141 |
| Injection site discolourationGeneral disorders | 55/141 |
| Injection site pruritusGeneral disorders | 46/141 |
| FatigueGeneral disorders | 38/141 |
| NauseaGastrointestinal disorders | 37/141 |
| HeadacheNervous system disorders | 35/141 |
| MyalgiaMusculoskeletal and connective tissue disorders | 33/141 |
Safety set: All enrolled participants who received at least 1 injection of study drug.
| Age, Continuous(years) | Mipomersen |
|---|---|
| Mean | 49.3 ± 15.3 |
| Sex: Female, Male(Participants) | Mipomersen |
|---|---|
| Female | 57 |
| Male | 84 |
This study is completed, as verified in Aug 2016. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Mipomersen
Lipid Metabolism, Inborn Errors →
Kastle Therapeutics, LLC