Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2008-006020-53 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Ionis Pharmaceuticals, Inc. | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The purpose of the study is to evaluate the safety and efficacy of dosing with mipomersen for 26 weeks in treating severely hypercholesterolemic patients who are on a maximally tolerated lipid-lowering regimen and who are not on apheresis.
Hypercholesterolemia is characterized by markedly elevated low density lipoproteins (LDL). Elevated LDL is a major risk factor for coronary heart disease (CHD).
Mipomersen is an antisense drug that reduces a protein in the liver cells called apolipoprotein B (Apo-B). Apo-B plays a role in producing low density lipoprotein cholesterol (LDL-C) (the "bad" cholesterol) and moving it from the liver to one's bloodstream. High LDL-C is an independent risk factor for the development of coronary heart disease (CHD) or other diseases of blood vessels. It has been shown that lowering LDL-C reduces the risk of heart attacks and other major adverse cardiovascular events. The purpose of this study is to determine whether mipomersen safely and effectively lowers LDL-C in severely hypercholesterolemic patients who are on a maximally tolerated lipid-lowering regimen and who are not on apheresis.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Weekly subcutaneous injections for 26 weeks |
|
| Mipomersen | Experimental | 200 mg weekly subcutaneous injections for 26 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Mipomersen | Drug | 200 mg (1 mL), weekly subcutaneous injections for 26 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Primary Efficacy Time Point | LDL-C was measured in mg/dL. Samples were taken following an overnight fast. For patients with triglycerides <400 mg/dL, LDL-C was obtained using Friedewald's calculation; and for patients with triglycerides >=400 mg/dL, LDL-C was directly measured by the central laboratory using ultracentrifugation. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| LDL-C at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in Apolipoprotein B (Apo-B) at Primary Efficacy Time Point | Apo-B was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage Change From Baseline in Triglycerides at Primary Efficacy Time Point (PET) | Triglycerides were measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. |
Inclusion Criteria:
Fasting LDL-C ≥200 mg/dL (5.1 mmol/L) at screening and the presence of at least 1 of the following criteria:
On stable, maximally tolerated statin therapy for 8 weeks
On stable, medication from an additional class of hypolipidemic agents for 8 weeks.
On stable, low fat diet for 12 weeks
Stable weight for 6 weeks
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | Genzyme, a Sanofi Company | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Jupiter | Florida | 33458 | United States | |||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 23152839 | Result | McGowan MP, Tardif JC, Ceska R, Burgess LJ, Soran H, Gouni-Berthold I, Wagener G, Chasan-Taber S. Randomized, placebo-controlled trial of mipomersen in patients with severe hypercholesterolemia receiving maximally tolerated lipid-lowering therapy. PLoS One. 2012;7(11):e49006. doi: 10.1371/journal.pone.0049006. Epub 2012 Nov 13. | |
| 27578134 |
Not provided
Not provided
One hundred and four patients were screened and 58 randomized in a 2:1 treatment arm ratio.
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Weekly subcutaneous injections for 26 weeks |
| FG001 | Mipomersen | 200 mg weekly subcutaneous injections for 26 weeks |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Treatment Period |
|
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Placebo | Drug | 1 mL weekly subcutaneous injections for 26 weeks |
|
| Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Apo-B at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Percentage Change From Baseline in Total Cholesterol at Primary Efficacy Time Point (PET) | Total cholesterol was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Total Cholesterol at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Percentage Change From Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Primary Efficacy Time Point (PET) | Non-HDL-C was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Non-HDL-C at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Triglycerides at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Percentage Change From Baseline in Lipoprotein(a) at Primary Efficacy Time Point (PET) | Lipoprotein(a) was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Lipoprotein(a) at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Percentage Change From Baseline in Very-Low-Density Lipoprotein Cholesterol (VLDL-C) at Primary Efficacy Time Point (PET) | VLDL-C was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| VLDL-C at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Change From Baseline in Ratio of Low-density Lipoprotein Cholesterol (LDL-C) to High-density Lipoprotein Cholesterol (HDL-C) at Primary Efficacy Time Point (PET) | LDL-C and HDL-C were measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Ratio of LDL-C to HDL-C at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Percent Change From Baseline in Apolipoprotein A1 (Apo-A1) at Primary Efficacy Time Point (PET) | Apo-A1 was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Apo-A1 at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Percentage Change From Baseline in High-Density Lipoprotein Cholesterol (HDL-C) at Primary Efficacy Time Point (PET) | HDL-C was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| HDL-C at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
| Winter Park |
| Florida |
| 32792 |
| United States |
| Atlanta | Georgia | 30338 | United States |
| Kansas City | Kansas | 66160 | United States |
| Boston | Massachusetts | 02114 | United States |
| St Louis | Missouri | 63104 | United States |
| Concord | New Hampshire | 03301 | United States |
| Cincinnati | Ohio | 45212 | United States |
| Aiken | South Carolina | 29801 | United States |
| Winnipeg | Manitoba | R3A 1M5 | Canada |
| Chicoutimi | Quebec | G7H 5H6 | Canada |
| Montreal | Quebec | H1T 1C8 | Canada |
| Hardec Kralove | 500 05 | Czechia |
| Pilsen | 305 99 | Czechia |
| Prague | 128 08 | Czechia |
| Uherské Hradiště | 686 68 | Czechia |
| Berlin | 13353 | Germany |
| Freiburg im Breisgau | 79106 | Germany |
| Heidelberg | 69120 | Germany |
| Koln (Lindenthal) | 50937 | Germany |
| Cape Town | 7500 | South Africa |
| Cape Town | 7925 | South Africa |
| Gauteng | 0157 | South Africa |
| Pretoria | 0002 | South Africa |
| Guildford | Surrey | GU2 7XX | United Kingdom |
| London | SE1 7EH | United Kingdom |
| Manchester | MI3 9WL | United Kingdom |
| Duell PB, Santos RD, Kirwan BA, Witztum JL, Tsimikas S, Kastelein JJP. Long-term mipomersen treatment is associated with a reduction in cardiovascular events in patients with familial hypercholesterolemia. J Clin Lipidol. 2016 Jul-Aug;10(4):1011-1021. doi: 10.1016/j.jacl.2016.04.013. Epub 2016 May 9. |
| 25614280 | Derived | Santos RD, Raal FJ, Catapano AL, Witztum JL, Steinhagen-Thiessen E, Tsimikas S. Mipomersen, an antisense oligonucleotide to apolipoprotein B-100, reduces lipoprotein(a) in various populations with hypercholesterolemia: results of 4 phase III trials. Arterioscler Thromb Vasc Biol. 2015 Mar;35(3):689-99. doi: 10.1161/ATVBAHA.114.304549. Epub 2015 Jan 22. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Follow-up Period |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Weekly subcutaneous injections for 26 weeks |
| BG001 | Mipomersen | 200 mg weekly subcutaneous injections for 26 weeks |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Number | participants |
| ||||||||||||||||
| Ethnicity | Number | participants |
| ||||||||||||||||
| Body Mass Index | Mean | Standard Deviation | kg/m^2 |
| |||||||||||||||
| Waist/hip ratio | Mean | Standard Deviation | ratio |
| |||||||||||||||
| Metabolic syndrome | Yes if 3 or more risk factors are present: 1) Abdominal obesity 2) Triglycerides >=150 mg/dl * 3) High density lipoprotein cholesterol (men <40 mg/dl) (women <50 mg/dl) * 4) Systolic blood pressure >=130 or diastolic >=85 mmHg * 5) Fasting glucose >=100 mg/dl * * = or on medication for condition | Number | participants |
| |||||||||||||||
| Tobacco Use | Number | participants |
| ||||||||||||||||
| Alcohol Use | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Primary Efficacy Time Point | LDL-C was measured in mg/dL. Samples were taken following an overnight fast. For patients with triglycerides <400 mg/dL, LDL-C was obtained using Friedewald's calculation; and for patients with triglycerides >=400 mg/dL, LDL-C was directly measured by the central laboratory using ultracentrifugation. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set (FAS). The FAS, which represents the practically-feasible intent-to-treat (ITT) population as delineated in ICH Guideline E9, consists of treated participants with a valid baseline and at least one post-baseline LDL-C measure. | Posted | Mean | Standard Deviation | percentage of baseline | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Primary | LDL-C at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | mg/dL | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change From Baseline in Apolipoprotein B (Apo-B) at Primary Efficacy Time Point | Apo-B was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | percentage of baseline | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Apo-B at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | mg/dL | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage Change From Baseline in Total Cholesterol at Primary Efficacy Time Point (PET) | Total cholesterol was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | percentage of baseline | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Total Cholesterol at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | mg/dL | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage Change From Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Primary Efficacy Time Point (PET) | Non-HDL-C was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | percentage of baseline | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Non-HDL-C at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | mg/dL | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Percentage Change From Baseline in Triglycerides at Primary Efficacy Time Point (PET) | Triglycerides were measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | percentage of baseline | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Triglycerides at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | mg/dL | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Percentage Change From Baseline in Lipoprotein(a) at Primary Efficacy Time Point (PET) | Lipoprotein(a) was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | percentage of baseline | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Lipoprotein(a) at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | mg/dL | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Percentage Change From Baseline in Very-Low-Density Lipoprotein Cholesterol (VLDL-C) at Primary Efficacy Time Point (PET) | VLDL-C was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | percentage of baseline | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | VLDL-C at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | mg/dL | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Change From Baseline in Ratio of Low-density Lipoprotein Cholesterol (LDL-C) to High-density Lipoprotein Cholesterol (HDL-C) at Primary Efficacy Time Point (PET) | LDL-C and HDL-C were measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Median | Inter-Quartile Range | percentage of baseline | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Ratio of LDL-C to HDL-C at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Median | Inter-Quartile Range | ratio | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Percent Change From Baseline in Apolipoprotein A1 (Apo-A1) at Primary Efficacy Time Point (PET) | Apo-A1 was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | percentage of baseline | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Apo-A1 at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | mg/dL | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Percentage Change From Baseline in High-Density Lipoprotein Cholesterol (HDL-C) at Primary Efficacy Time Point (PET) | HDL-C was measured in mg/dL. Samples were taken following an overnight fast. Baseline was defined as the average of the screening and Study Day 1 (pre-treatment) assessments. An assessment was not included in this calculation if it was associated with a non-fasting blood draw or was drawn more than 4 weeks prior to Study Day 1. The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | percentage of baseline | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | HDL-C at Baseline and the Primary Efficacy Time Point (PET) | The PET was the post-baseline visit, for which LDL-C was assessed, closest to 14 days after the last dose of study drug. | Full analysis set | Posted | Mean | Standard Deviation | mg/dL | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET) up to week 28 |
|
|
Day 1 to week 28. On-treatment AEs started on/after the first study drug dose and on/before the end of the treatment period. The treatment period was the time study drug was administered until the later of the PET or 14 days after last study drug dose.
The Safety Set includes all randomized patients who receive at least 1 injection of the study treatment. In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | Weekly subcutaneous injections for 26 weeks | 0 | 19 | 16 | 19 | ||
| EG001 | Mipomersen | 200 mg weekly subcutaneous injections for 26 weeks | 6 | 39 | 39 | 39 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute myocardial infarction | Cardiac disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Angina pectoris | Cardiac disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Angina unstable | Cardiac disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Prinzmetal angina | Cardiac disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Device malfunction | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Hepatic steatosis | Hepatobiliary disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 13.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Angina pectoris | Cardiac disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Coronary artery disease | Cardiac disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Supraventricular extrasystoles | Cardiac disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Tinnitus | Ear and labyrinth disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Eye swelling | Eye disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Vitreous floaters | Eye disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Chills | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site discolouration | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site erythema | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site haematoma | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site induration | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site inflammation | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site mass | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site nodule | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site oedema | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site papule | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site pruritus | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site rash | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site reaction | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site recall reaction | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site swelling | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site urticaria | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site vesicles | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Hepatic function abnormal | Hepatobiliary disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Hepatic steatosis | Hepatobiliary disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Liver tenderness | Hepatobiliary disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Allergy to plants | Immune system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Impetigo | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Onychomycosis | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Pleurisy viral | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Animal bite | Injury, poisoning and procedural complications | MedDRA 13.0 | Systematic Assessment |
| |
| Animal scratch | Injury, poisoning and procedural complications | MedDRA 13.0 | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 13.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 13.0 | Systematic Assessment |
| |
| Epicondylitis | Injury, poisoning and procedural complications | MedDRA 13.0 | Systematic Assessment |
| |
| Post procedural complication | Injury, poisoning and procedural complications | MedDRA 13.0 | Systematic Assessment |
| |
| Post procedural haematoma | Injury, poisoning and procedural complications | MedDRA 13.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Cardiac murmur | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Electrocardiogram PR prolongation | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Epstein-Barr virus antibody positive | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Liver function test abnormal | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Prothrombin time prolonged | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Weight increased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Glucose tolerance impaired | Metabolism and nutrition disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Type 2 diabetes mellitus | Metabolism and nutrition disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Groin pain | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Osteochondrosis | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Pain in jaw | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Benign breast neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 13.0 | Systematic Assessment |
| |
| Cerebrovascular accident | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Restless legs syndrome | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Bipolar disorder | Psychiatric disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Renal cyst | Renal and urinary disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Bronchitis chronic | Respiratory, thoracic and mediastinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Blister | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Hyperkeratosis | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Xanthoma | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Flushing | Vascular disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Hot flush | Vascular disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Orthostatic hypotension | Vascular disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Peripheral arterial occlusive disease | Vascular disorders | MedDRA 13.0 | Systematic Assessment |
|
In multi-site studies, PI can publish site data after a multi-centered publication or 12 months after study completion. PI gives Genzyme a draft 60 days before publication. Genzyme can ask that confidential information be removed, and can defer publication another 6 months (contracts have variable timeframes) upon notifying PI that it will file a patent application on inventions contained in the draft.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Genzyme Medical Information | Genzyme Corporation | 617-252-7832 |
| ID | Term |
|---|---|
| D006937 | Hypercholesterolemia |
| D003327 | Coronary Disease |
| ID | Term |
|---|---|
| D006949 | Hyperlipidemias |
| D050171 | Dyslipidemias |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D017202 | Myocardial Ischemia |
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
| D014652 | Vascular Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C524142 | mipomersen |
Not provided
Not provided
Not provided
| Protocol non-compliance |
|
| Male |
|
| Black |
|
| Asian |
|
| Multiple |
|
| Other |
|
| Hispanic or Latino |
|
| Yes |
|
| Non-current |
|
| Never |
|
| Non-current |
|
| Never |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|