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| ID | Type | Description | Link |
|---|---|---|---|
| 2007-005140-24 | EudraCT Number |
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| Name | Class |
|---|---|
| Ionis Pharmaceuticals, Inc. | INDUSTRY |
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The purpose of this study is to determine safety and efficacy of mipomersen (ISIS 301012) in the reduction of total cholesterol, low density lipoprotein cholesterol (LDL-C), and apolipoprotein B (apoB) in high risk subjects intolerant to statins.
In humans, apoB is the principal apolipoprotein of the atherogenic lipoproteins, comprising very low-density lipoprotein (VLDL), intermediate density lipoprotein (IDL) and low density lipoprotein (LDL). ApoB messenger ribonucleic acid (mRNA) is abundantly present in the liver. Within the endoplasmatic reticulum, apoB requires lipidation by microsomal triglyceride transfer protein, which allows apoB to be incorporated in the VLDL particle within the lumen of the endoplasmatic reticulum. Non-lipidated apoB is readily degraded via ubiquitination. Notably, apoB within the VLDL particle is obligatory for hepatic secretion of VLDL. ApoB remains present within the VLDL-metabolism pathway, from secretion to clearance of the end product LDL by the liver LDL receptor. As a consequence, apoB reliably reflects the total burden of atherogenic lipoproteins. Thus, apoB carries strong prognostic value for cardiovascular events, which exceeds the predictive value of LDL-C. Conversely, decreased levels of apoB (e.g. in familial hypobetalipoproteinemia) have been associated with reduced levels of atherosclerosis. These genetic observations have prompted interest in pharmacologic inhibition of apoB synthesis.
Mipomersen (ISIS 301012) is an antisense drug targeted to human apoB, the principal apolipoprotein of LDL and its metabolic precursor, VLDL. Mipomersen (ISIS 301012) is complementary to the coding region of the mRNA for apoB, binding by Watson and Crick base pairing. The hybridization (binding) of mipomersen (ISIS 301012) to the cognate mRNA results in Ribonuclease (RNase) H-mediated degradation of the cognate mRNA, thus inhibiting translation of the apoB protein.
This was a randomized, double-blind, placebo-controlled Phase 2 study to assess the safety and efficacy of mipomersen administration in high-risk statin-intolerant patients with hypercholesterolemia. This study consisted of a ≤3-week screening period, 26 weeks of treatment, and a 24-week post-treatment follow-up period.
Eligible patients were randomized in a 2:1 ratio to receive mipomersen 200 mg or matching volume placebo subcutaneous (SC) injections weekly.
Following the screening visit, eligible patients returned to the study center for clinical evaluation every week for study drug administration and assessments.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | 1 mL placebo saline, weekly subcutaneous injections for 26 weeks |
|
| Mipomersen | Experimental | 200 mg (1 mL), weekly subcutaneous injections for 26 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| mipomersen | Drug |
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| |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the 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. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Low-density Lipoprotein Cholesterol at Baseline and the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Summary of Participants With Adverse Events | The on-treatment time frame spanned the time during which the study drug was administered until the later of the primary efficacy time point and 14 days beyond the last study drug date. Adverse events (AEs) were considered related if assessed by the Investigator as possibly, probably or definitely related to study drug. Severity was assessed as:
Serious AEs (SAEs) are those that resulted in death, were life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly, or resulted in an important medical event that may have jeopardized the patient or required medical or surgical intervention. | Pre-treatment (prior to first dose), On-treatment (Day 1 to week 28), Post-treatment (Week 28-52) |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in Apolipoprotein B at the Primary Efficacy Time Point | Apolipoprotein B was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in Triglycerides at the Primary Efficacy Time Point | Triglycerides were measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor | Genzyme, a Sanofi Company | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Amsterdam | 1105AZ | Netherlands |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 22507979 | Derived | Visser ME, Wagener G, Baker BF, Geary RS, Donovan JM, Beuers UH, Nederveen AJ, Verheij J, Trip MD, Basart DC, Kastelein JJ, Stroes ES. Mipomersen, an apolipoprotein B synthesis inhibitor, lowers low-density lipoprotein cholesterol in high-risk statin-intolerant patients: a randomized, double-blind, placebo-controlled trial. Eur Heart J. 2012 May;33(9):1142-9. doi: 10.1093/eurheartj/ehs023. Epub 2012 Apr 16. | |
| 20707601 |
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42 patients were screened, 34 participants were randomized, and 33 participants were treated: 21 participants to mipomersen and 12 participants to placebo.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | 1 mL placebo saline, weekly subcutaneous injections for 26 weeks |
| FG001 | Mipomersen | 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Treatment Period |
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| placebo |
| Drug |
|
|
| Apolipoprotein B at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Percent Change From Baseline in Total Cholesterol at the Primary Efficacy Time Point | Total cholesterol was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Total Cholesterol at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol at the Primary Efficacy Time Point | Non-high-density lipoprotein cholesterol was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Non-High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Triglycerides at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Percent Change From Baseline in Lipoprotein (a) at the Primary Efficacy Time Point | Lipoprotein (a) was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Lipoprotein (a) at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Percent Change From Baseline in Very Low Density Lipoprotein Cholesterol at the Primary Efficacy Time Point | Very low density lipoprotein cholesterol was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Very Low Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Percent Change From Baseline in the Ratio of Low-Density Lipoprotein Cholesterol to High-Density Lipoprotein Cholesterol at the Primary Efficacy Time Point | The ratio of low-density lipoprotein (LDL) cholesterol to high-density lipoprotein (HDL) cholesterol was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Ratio of Low-Density Lipoprotein Cholesterol to High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Percent Change From Baseline in Apolipoprotein A1 at the Primary Efficacy Time Point | Apolipoprotein A1 was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Apolipoprotein A1 at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Percent Change From Baseline in High-Density Lipoprotein Cholesterol at the Primary Efficacy Time Point | High-density lipoprotein cholesterol (HDL-C) was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
| Derived |
| Patel N, Hegele RA. Mipomersen as a potential adjunctive therapy for hypercholesterolemia. Expert Opin Pharmacother. 2010 Oct;11(15):2569-72. doi: 10.1517/14656566.2010.512006. |
| Full Analysis Set (FAS) and Safety Set |
|
| COMPLETED |
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| NOT COMPLETED |
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| Follow-up Period |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | 1 mL placebo saline, weekly subcutaneous injections for 26 weeks |
| BG001 | Mipomersen | 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Number | participants |
| ||||||||||||||||
| Body Mass Index (BMI) | Mean | Standard Deviation | kg/m^2 |
| |||||||||||||||
| Waist/hip Ratio | Mean | Standard Deviation | ratio |
| |||||||||||||||
| Metabolic syndrome | A participant was classified as having metabolic syndrome if any 3 of the following risk factors existed: 1) Waist circumference ≥102 cm (men) or ≥88 cm (women); 2) Triglycerides ≥150 mg/dl *; 3) High density lipoprotein cholesterol <40 mg/dl (men) or <50 mg/dl (women)*; 4) Systolic blood pressure ≥130 or diastolic ≥85 mmHg *; 5) Fasting glucose ≥100 mg/dl *. * = or on medication for the specified risk factor. | Number | participants |
| |||||||||||||||
| Tobacco Use | Number | participants |
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| Alcohol use | Number | participants |
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| 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 the 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. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | The Full Analysis Set, which consisted of all randomized patients who received at least 1 injection of study drug (mipomersen or placebo) and with a valid baseline and at least 1 post-baseline LDL-C measurement. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Primary | Low-density Lipoprotein Cholesterol at Baseline and the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Primary | Summary of Participants With Adverse Events | The on-treatment time frame spanned the time during which the study drug was administered until the later of the primary efficacy time point and 14 days beyond the last study drug date. Adverse events (AEs) were considered related if assessed by the Investigator as possibly, probably or definitely related to study drug. Severity was assessed as:
Serious AEs (SAEs) are those that resulted in death, were life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly, or resulted in an important medical event that may have jeopardized the patient or required medical or surgical intervention. | Safety set of all randomized participants who received at least one injection of study drug. | Posted | Number | participants | Pre-treatment (prior to first dose), On-treatment (Day 1 to week 28), Post-treatment (Week 28-52) |
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| Secondary | Percent Change From Baseline in Apolipoprotein B at the Primary Efficacy Time Point | Apolipoprotein B was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | The Full Analysis Set, which consisted of all randomized patients who received at least 1 injection of study drug (mipomersen or placebo) and with a valid baseline and at least 1 post-baseline LDL-C measurement. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Secondary | Apolipoprotein B at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | Full analysis set | Posted | Median | Inter-Quartile Range | mg/dL | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Secondary | Percent Change From Baseline in Total Cholesterol at the Primary Efficacy Time Point | Total cholesterol was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | The Full Analysis Set, which consisted of all randomized patients who received at least 1 injection of study drug (mipomersen or placebo) and with a valid baseline and at least 1 post-baseline LDL-C measurement. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Secondary | Total Cholesterol at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Secondary | Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol at the Primary Efficacy Time Point | Non-high-density lipoprotein cholesterol was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | The Full Analysis Set, which consisted of all randomized patients who received at least 1 injection of study drug (mipomersen or placebo) and with a valid baseline and at least 1 post-baseline LDL-C measurement. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Secondary | Non-High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Other Pre-specified | Percent Change From Baseline in Triglycerides at the Primary Efficacy Time Point | Triglycerides were measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | The Full Analysis Set, which consisted of all randomized patients who received at least 1 injection of study drug (mipomersen or placebo) and with a valid baseline and at least 1 post-baseline LDL-C measurement. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Other Pre-specified | Triglycerides at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Other Pre-specified | Percent Change From Baseline in Lipoprotein (a) at the Primary Efficacy Time Point | Lipoprotein (a) was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | The Full Analysis Set, which consisted of all randomized patients who received at least 1 injection of study drug (mipomersen or placebo) and with a valid baseline and at least 1 post-baseline LDL-C measurement. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Other Pre-specified | Lipoprotein (a) at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Other Pre-specified | Percent Change From Baseline in Very Low Density Lipoprotein Cholesterol at the Primary Efficacy Time Point | Very low density lipoprotein cholesterol was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | The Full Analysis Set, which consisted of all randomized patients who received at least 1 injection of study drug (mipomersen or placebo) and with a valid baseline and at least 1 post-baseline LDL-C measurement. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Other Pre-specified | Very Low Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Other Pre-specified | Percent Change From Baseline in the Ratio of Low-Density Lipoprotein Cholesterol to High-Density Lipoprotein Cholesterol at the Primary Efficacy Time Point | The ratio of low-density lipoprotein (LDL) cholesterol to high-density lipoprotein (HDL) cholesterol was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | The Full Analysis Set, which consisted of all randomized patients who received at least 1 injection of study drug (mipomersen or placebo) and with a valid baseline and at least 1 post-baseline LDL-C measurement. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Other Pre-specified | Ratio of Low-Density Lipoprotein Cholesterol to High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | Full analysis set | Posted | Mean | Standard Deviation | ratio | Baseline (the average of the screening and Day 1 pre-treatment assessments) and the Primary Efficacy Time point (PET; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Other Pre-specified | Percent Change From Baseline in Apolipoprotein A1 at the Primary Efficacy Time Point | Apolipoprotein A1 was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | The Full Analysis Set, which consisted of all randomized patients who received at least 1 injection of study drug (mipomersen or placebo) and with a valid baseline and at least 1 post-baseline LDL-C measurement. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Other Pre-specified | Apolipoprotein A1 at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Other Pre-specified | Percent Change From Baseline in High-Density Lipoprotein Cholesterol at the Primary Efficacy Time Point | High-density lipoprotein cholesterol (HDL-C) was measured in mg/dL. Samples were taken following an overnight fast. The primary efficacy time point was the post-baseline visit closest to 14 days after the last dose of study treatment. | The Full Analysis Set, which consisted of all randomized patients who received at least 1 injection of study drug (mipomersen or placebo) and with a valid baseline and at least 1 post-baseline LDL-C measurement. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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| Other Pre-specified | High-Density Lipoprotein Cholesterol at Baseline and at the Primary Efficacy Time Point | The primary efficacy time point (PET) was the post-baseline visit closest to 14 days after the last dose of study treatment. | 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; Week 28 or the post-baseline visit closest to 14 days after the last dose). |
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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 | 1 mL placebo saline, weekly subcutaneous injections for 26 weeks | 1 | 12 | 12 | 12 | ||
| EG001 | Mipomersen | 200 mg (1 mL) of mipomersen, weekly subcutaneous injections for 26 weeks | 0 | 21 | 21 | 21 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute myocardial infarction | Cardiac disorders | MedDRA 13.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Haemorrhagic diathesis | Blood and lymphatic system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Angina pectoris | Cardiac disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Left ventricular hypertrophy | Cardiac disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Deafness unilateral | Ear and labyrinth disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Eye allergy | Eye disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Eye inflammation | Eye disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Eye pruritus | Eye disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Panophthalmitis | Eye disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Abdominal pain lower | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Abnormal faeces | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Change of bowel habit | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Faeces discoloured | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Faeces hard | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Food poisoning | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Frequent bowel movements | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Gastrointestinal pain | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Gingivitis | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Intestinal functional disorder | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Tooth disorder | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Toothache | 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 |
| |
| Chest pain | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Feeling abnormal | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Feeling cold | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Impaired healing | 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 discomfort | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site erythema | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site exfoliation | 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 nodule | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site pallor | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site pruritus | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site reaction | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site swelling | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site vesicles | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site warmth | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Sensation of pressure | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Tenderness | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Vessel puncture site haematoma | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Vessel puncture site reaction | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Hepatic steatosis | Hepatobiliary disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Eye infection | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Injection site cellulitis | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 13.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 13.0 | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA 13.0 | Systematic Assessment |
| |
| Post procedural complication | Injury, poisoning and procedural complications | MedDRA 13.0 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 13.0 | Systematic Assessment |
| |
| Activated partial thromboplastin time | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Activated partial thromboplastin time prolonged | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Albumin urine present | 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 calcium decreased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Blood phosphorus decreased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Blood potassium increased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Body temperature decreased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Body temperature increased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Coagulation test abnormal | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Electrocardiogram poor R-wave progression | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Gastric pH decreased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Globulins decreased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| International normalised ratio 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 |
| |
| Protein urine present | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Prothrombin time prolonged | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Sputum abnormal | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Urine albumin/creatinine ratio increased | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Bone disorder | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Bursitis | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Groin pain | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Musculoskeletal disorder | 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 |
| |
| Myosclerosis | 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 |
| |
| Sensation of heaviness | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Tendon pain | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Aphonia | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Dizziness postural | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Migraine with aura | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Poor quality sleep | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Depressed mood | Psychiatric disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Initial insomnia | Psychiatric disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Listless | Psychiatric disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Tension | Psychiatric disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Menstruation irregular | Reproductive system and breast disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Postmenopausal haemorrhage | Reproductive system and breast disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Hyperkeratosis | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Nail disorder | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Nail growth abnormal | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Pruritus generalised | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Skin burning sensation | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Hot flush | Vascular disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Vasodilatation | Vascular disorders | MedDRA 13.0 | Systematic Assessment |
|
Institution will submit for review a proposed publication or presentation at least 90 days prior to submission date. Sponsor has the right to delay publication or presentation for not more than 6 months to address patent applications. Sponsor also has the right to demand in writing the deletion of confidential information. Investigator will not make public raw study data, detailed subject cases, or information identifying any subject.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Genzyme Medical Information | Genzyme Corporation | 617-252-7832 |
| ID | Term |
|---|---|
| D008659 | Metabolic Diseases |
| D006949 | Hyperlipidemias |
| D006937 | Hypercholesterolemia |
| D050171 | Dyslipidemias |
| D052439 | Lipid Metabolism Disorders |
| ID | Term |
|---|---|
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C524142 | mipomersen |
Not provided
Not provided
Not provided
| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Black |
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| Yes |
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| Non-current |
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| Never |
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| Non-current |
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| Never |
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