Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2011-001480-42 | EudraCT Number | ||
| EFC12875 | Other Identifier | Sanofi |
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Primary objective:
Determine whether mipomersen (ISIS 301012) significantly reduces atherogenic lipid levels in patients with severe heterozygous familial hypercholesterolemia (severe HeFH), defined as low-density lipoprotein cholesterol (LDL-C) levels ≥200 mg/dL plus the presence of coronary heart disease (CHD)/risk equivalents or LDL-C levels ≥300 mg/dL regardless of the presence of CHD/risk equivalents (referred to as Cohort 1) compared to placebo. Two different mipomersen dosing regimens will be studied: subcutaneous (SC) mipomersen 200 mg once weekly versus placebo, and SC mipomersen 70 mg thrice weekly versus placebo.
Secondary Objectives:
The study consisted of a Screening period of up to 4 weeks, Blinded Treatment Phase of 60 weeks, Open-Label Continuation Period of 26 weeks, and Post-Treatment Phase of 24 weeks.
Study Design, masking - Study treatment was blinded (double-blinded) through the Primary Efficacy Assessment Visit in the Blinded Treatment Period. Study treatment was open-label in the Open-Label Continuation Period.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Regimen A: Mipomersen | Experimental | Subcutaneous injection of mipomersen 200 mg once weekly |
|
| Regimen A: Placebo | Placebo Comparator | Placebo matching subcutaneous injection once weekly. |
|
| Regimen B: Mipomersen | Experimental | Subcutaneous injection of mipomersen 70 mg thrice weekly. |
|
| Regimen B: Placebo | Placebo Comparator | Placebo matching subcutaneous injection thrice weekly. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| mipomersen sodium 200 mg | Drug | Subcutaneous mipomersen 200 mg once weekly |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline To Primary Endpoint Visit (PET) In LDL-C In Cohort 1 | The percent change from baseline to PET in LDL-C was measured in Cohort 1, which included participants with severe heterozygous familial hypercholesterolemia (HeFH). Severe HeFH was defined as LDL-C levels ≥200 mg/deciliter (dL) plus the presence of coronary heart disease (CHD)/risk equivalents or LDL-C levels ≥300 mg/dL regardless of the presence of CHD/risk equivalents. | Baseline and Week 61 |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline To PET In LDL-C In Cohort 2 | The percent change from baseline to PET in LDL-C was measured in Cohort 2, which included participants with HeFH with LDL-C levels ≥160 mg/dL and <200 mg/dL, plus the presence of CHD/risk equivalents. | Baseline, PET (up to 60 weeks) |
| Percent Change From Baseline To PET In Apolipoprotein B (Apo B) In Cohort 1 |
Not provided
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 |
|---|---|---|---|---|---|---|
| Mission Viejo | California | United States | ||||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 26946290 | Derived | Tragante V, Asselbergs FW, Swerdlow DI, Palmer TM, Moore JH, de Bakker PIW, Keating BJ, Holmes MV. Harnessing publicly available genetic data to prioritize lipid modifying therapeutic targets for prevention of coronary heart disease based on dysglycemic risk. Hum Genet. 2016 May;135(5):453-467. doi: 10.1007/s00439-016-1647-9. Epub 2016 Mar 5. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Regimen A: Mipomersen, 200 mg, Once Weekly | Participants received once weekly subcutaneous (SC) injections of mipomersen sodium 200 milligrams (mg) during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
Not provided
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Not provided
Not provided
| Placebo | Drug | Placebo vehicle for subcutaneous injection. |
|
| mipomersen sodium 70 mg | Drug | Subcutaneous mipomersen 70 mg thrice weekly |
|
|
The percent change from baseline to PET in Apo B was measured in participants in Cohort 1 with HeFH during the Blinded Treatment Period. |
| Baseline and Week 61 |
| Percent Change From Baseline To PET In Apolipoprotein B (Apo B) In Cohort 2 | The percent change from baseline to PET in Apo B was measured in participants in Cohort 2 with HeFH during the Blinded Treatment Period. | Baseline and Week 61 |
| Percent Change From Baseline To PET in Lipoprotein (a) In Cohort 1 | The percent change from baseline to PET in Lipoprotein A1 was measured in participants in Cohort 1 with HeFH during the Blinded Treatment Period. | Baseline and Week 61 |
| Percent Change From Baseline To PET in Lipoprotein (a) In Cohort 2 | The percent change from baseline to PET in Lipoprotein A1 was measured in participants in Cohort 2 with HeFH during the Blinded Treatment Period. | Baseline and Week 61 |
| Aurora |
| Colorado |
| United States |
| Cooper City | Florida | United States |
| Winter Park | Florida | United States |
| Indianapolis | Indiana | United States |
| Kansas City | Kansas | United States |
| Boston | Massachusetts | United States |
| Ann Arbor | Michigan | United States |
| Grandville | Michigan | United States |
| Rochester | Minnesota | United States |
| St Louis | Missouri | United States |
| Omaha | Nebraska | United States |
| Summit | New Jersey | United States |
| North Massapequa | New York | United States |
| Portland | Oregon | United States |
| Lancaster | Pennsylvania | United States |
| Charleston | South Carolina | United States |
| Nashville | Tennessee | United States |
| Dallas | Texas | United States |
| Norfolk | Virginia | United States |
| Buenos Aires | Argentina |
| Córdoba | Argentina |
| Perth | Australia |
| South Brisbane | Australia |
| Edegem | Belgium |
| Haine-Saint-Paul | Belgium |
| Leuven | Belgium |
| Rio de Janeiro | Brazil |
| São Paulo | Brazil |
| Chicoutimi | Quebec | Canada |
| Montreal | Quebec | Canada |
| Sainte-Foy | Quebec | Canada |
| Osijek | Croatia |
| Zagreb | Croatia |
| Hradec Králové | Czechia |
| Prague | Czechia |
| Aarhus | Denmark |
| Viborg | Denmark |
| Aachen | Germany |
| Berlin | Germany |
| Cologne | Germany |
| Freiburg im Breisgau | Germany |
| Hamburg | Germany |
| Heidelberg | Germany |
| Magdeburg | Germany |
| Ioannina | Greece |
| Kallithea | Greece |
| Hong Kong | Hong Kong |
| Baja | Hungary |
| Budapest | Hungary |
| Debrecen | Hungary |
| New Delhi | India |
| Holon | Israel |
| Kfar Saba | Israel |
| Ofakim | Israel |
| Bologna | Italy |
| Naples | Italy |
| Padova | Italy |
| Palermo | Italy |
| Pisa | Italy |
| Roma | Italy |
| Kuala Lumpur | Malaysia |
| Kubang Kerian | Malaysia |
| Alkmaar | Netherlands |
| Amsterdam | Netherlands |
| Maastricht | Netherlands |
| Nijmegen | Netherlands |
| Utrecht | Netherlands |
| Waalwijk | Netherlands |
| Christchurch | New Zealand |
| Bodø | Norway |
| Oslo | Norway |
| Sandefjord | Norway |
| Bialystok | Poland |
| Gdansk | Poland |
| Katowice | Poland |
| Krakow | Poland |
| Nałęczów | Poland |
| Poznan | Poland |
| Sopot | Poland |
| Szczecin | Poland |
| Warsaw | Poland |
| Wroclaw | Poland |
| Barnaul | Russia |
| Kemerovo | Russia |
| Moscow | Russia |
| Novosibirsk | Russia |
| Petrozavodsk | Russia |
| Ryazan | Russia |
| Saint Petersburg | Russia |
| Tomsk | Russia |
| Yaroslavl | Russia |
| Cape Town | South Africa |
| Pretoria | South Africa |
| Seoul | South Korea |
| Madrid | Spain |
| Stockholm | Sweden |
| New Taipei City | Taiwan |
| Taipei | Taiwan |
| Ankara | Turkey (Türkiye) |
| Istanbul | Turkey (Türkiye) |
| Izmir | Turkey (Türkiye) |
| Sivas | Turkey (Türkiye) |
| Ivano-Frankivsk | Ukraine |
| Kiev | Ukraine |
| Kyiv | Ukraine |
| Odesa | Ukraine |
| Birmingham | United Kingdom |
| Cardiff | United Kingdom |
| Liverpool | United Kingdom |
| London | United Kingdom |
| Manchester | United Kingdom |
| Oldham | United Kingdom |
| Regimen A: Placebo, Once Weekly |
Participants received once weekly SC injections of Placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. |
| FG002 | Regimen B: Mipomersen, 70 mg, Thrice Weekly | Participants received thrice weekly SC injections of mipomersen sodium 70 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. |
| FG003 | Regimen B: Placebo, Thrice Weekly | Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. |
| Randomized and Treated |
|
| Completed Blinded Treatment Period |
|
| Entered Open-Label Continuation |
|
| Completed Open-Label Continuation |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline low-density lipoprotein cholesterol (LDL-C) measurement, and had at least 1 post-baseline LDL-C measurement.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Regimen A: Mipomersen, 200 mg, Once Weekly | Participants received once weekly SC injections of mipomersen sodium 200 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. |
| BG001 | Regimen A: Placebo, Once Weekly | Participants received once weekly SC injections of Placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. |
| BG002 | Regimen B: Mipomersen, 70 mg, Thrice Weekly | Participants received thrice weekly SC injections of mipomersen sodium 70 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. |
| BG003 | Regimen B: Placebo, Thrice Weekly | Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. |
| BG004 | 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 |
| |||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| |||||||||||
| LDL-C Baseline Values | The full analysis set was analyzed in two cohorts, shown below. | Mean | Standard Deviation | mg/dL |
| |||||||||
| Apolipoprotein B Baseline Values | The full analysis set was analyzed in two cohorts, shown below. | Mean | Standard Deviation | mg/dL |
| |||||||||
| Lipoprotein (a) Baseline Values | The full analysis set was analyzed in two cohorts, shown below. | Mean | Standard Deviation | mg/dL |
|
| 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 To Primary Endpoint Visit (PET) In LDL-C In Cohort 1 | The percent change from baseline to PET in LDL-C was measured in Cohort 1, which included participants with severe heterozygous familial hypercholesterolemia (HeFH). Severe HeFH was defined as LDL-C levels ≥200 mg/deciliter (dL) plus the presence of coronary heart disease (CHD)/risk equivalents or LDL-C levels ≥300 mg/dL regardless of the presence of CHD/risk equivalents. | The full analysis set for Cohort 1 included all randomized participants who took at least 1 dose of study drug in Cohort 1 (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement. | Posted | Least Squares Mean | Standard Error | Percent | Baseline and Week 61 |
|
|
| ||||||||||||||||||||||||||||||||||
| Secondary | Percent Change From Baseline To PET In LDL-C In Cohort 2 | The percent change from baseline to PET in LDL-C was measured in Cohort 2, which included participants with HeFH with LDL-C levels ≥160 mg/dL and <200 mg/dL, plus the presence of CHD/risk equivalents. | The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement. | Posted | Least Squares Mean | Standard Error | Percent | Baseline, PET (up to 60 weeks) |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change From Baseline To PET In Apolipoprotein B (Apo B) In Cohort 1 | The percent change from baseline to PET in Apo B was measured in participants in Cohort 1 with HeFH during the Blinded Treatment Period. | The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement. | Posted | Least Squares Mean | Standard Error | Percent | Baseline and Week 61 |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change From Baseline To PET In Apolipoprotein B (Apo B) In Cohort 2 | The percent change from baseline to PET in Apo B was measured in participants in Cohort 2 with HeFH during the Blinded Treatment Period. | The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement. | Posted | Least Squares Mean | Standard Error | Percent | Baseline and Week 61 |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change From Baseline To PET in Lipoprotein (a) In Cohort 1 | The percent change from baseline to PET in Lipoprotein A1 was measured in participants in Cohort 1 with HeFH during the Blinded Treatment Period. | The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement. | Posted | Least Squares Mean | Standard Error | Percent | Baseline and Week 61 |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change From Baseline To PET in Lipoprotein (a) In Cohort 2 | The percent change from baseline to PET in Lipoprotein A1 was measured in participants in Cohort 2 with HeFH during the Blinded Treatment Period. | The full analysis set included all randomized participants who took at least 1 dose of study drug (mipomersen or placebo), had a valid baseline LDL-C measurement, and had at least 1 post-baseline LDL-C measurement. | Posted | Least Squares Mean | Standard Error | Percent | Baseline and Week 61 |
|
Adverse events were monitored from the time of the administration of the participant's first dose of study drug (mipomersen or placebo) through the participant's last visit of the Post-Treatment Period, up to Week 110.
Adverse events were collected for all randomized participants who received at least 1 dose of study drug (mipomersen or placebo).
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 | Regimen A: Mipomersen, 200 mg, Once Weekly | Participants received once weekly SC injections of mipomersen sodium 200 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. | 1 | 104 | 17 | 104 | 82 | 104 |
| EG001 | Regimen A: Placebo, Once Weekly | Participants received once weekly SC injections of Placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. | 0 | 51 | 13 | 51 | 27 | 51 |
| EG002 | Regimen B: Mipomersen, 70 mg, Thrice Weekly | Participants received thrice weekly SC injections of mipomersen sodium 70 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. | 1 | 102 | 23 | 102 | 78 | 102 |
| EG003 | Regimen B: Placebo, Thrice Weekly | Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. | 0 | 52 | 11 | 52 | 33 | 52 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Angina pectoris | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Angina unstable | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Acute myocardial infarction | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Coronary artery disease | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Myocardial infarction | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Acute coronary syndrome | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Atrial flutter | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Coronary artery stenosis | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Myocardial ischaemia | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Supraventricular tachycardia | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Death | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Drug intolerance | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Transient ischaemic attack | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Aphasia | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Carotid artery stenosis | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cerebral infarction | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Hemiparesis | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Ischaemic stroke | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Abdominal hernia | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Gastric ulcer | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Gastritis erosive | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Large intestine perforation | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Obstruction gastric | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pancreatic cyst | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Diverticulitis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Device related infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Escherichia pyelonephritis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Joint contracture | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Spinal osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pleuritic pain | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Embolism venous | Vascular disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Hypertensive crisis | Vascular disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Peripheral arterial occlusive disease | Vascular disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Peripheral artery stenosis | Vascular disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Renal cyst | Renal and urinary disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Benign prostatic hyperplasia | Reproductive system and breast disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cervical polyp | Reproductive system and breast disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Fibrocystic breast disease | Reproductive system and breast disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Ovarian cyst torsion | Reproductive system and breast disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Coronary artery restenosis | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Helicobacter test negative | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Low density lipoprotein increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Eye haemorrhage | Eye disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Lymphoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 18.1 | Systematic Assessment |
| |
| Abortion spontaneous | Pregnancy, puerperium and perinatal conditions | MedDRA 18.1 | Systematic Assessment |
| |
| Major depression | Psychiatric disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Suicide attempt | Psychiatric disorders | MedDRA 18.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Influenza like illness | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Injection site erythema | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Injection site swelling | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Injection site pruritus | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Angina pectoris | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Abdominal pain lower | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Hepatic steatosis | Hepatobiliary disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Kastle Therapeutics, LLC | 1-800-745-4447 | medinfo@genzyme.com |
| ID | Term |
|---|---|
| D006937 | Hypercholesterolemia |
| ID | Term |
|---|---|
| D006949 | Hyperlipidemias |
| D050171 | Dyslipidemias |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
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| C524142 | mipomersen |
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| Cohort 2 |
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| Cohort 2 |
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| Cohort 2 |
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Participants received thrice weekly SC injections of mipomersen sodium 70 mg during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period.
| OG003 | Regimen B: Placebo, Thrice Weekly | Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. |
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| OG003 | Regimen B: Placebo, Thrice Weekly | Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. |
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| OG003 | Regimen B: Placebo, Thrice Weekly | Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. |
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| OG003 | Regimen B: Placebo, Thrice Weekly | Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. |
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| OG003 | Regimen B: Placebo, Thrice Weekly | Participants received thrice weekly SC injections of placebo during the 60-week Blinded Treatment Period, continued the dosing regimen during the 26-week Open-Label Continuation Period, and then entered the 24-week Post-Treatment Period. |
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