Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2014-002421-35 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Akcea Therapeutics | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
The purpose of this study is to evaluate the efficacy and safety of volanesorsen given for 52 weeks in participants with Familial Chylomicronemia Syndrome
Not provided
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Volanesorsen-matching placebo administered subcutaneously once-weekly for 52 weeks. |
|
| Volanesorsen | Experimental | Volanesorsen 300 mg administered subcutaneously once-weekly for 52 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Volanesorsen | Drug |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change in Fasting Triglycerides (TG) From Baseline to Month 3 | The Month 3 endpoint was defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments. | Baseline to 3 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Postprandial TG Area Under the Curve (AUC)(0-9h) | Participants had 2 postprandial assessments - one at Baseline (completed at least 48 hours prior to first dose) and one at any time between Week 13 and 19, inclusive. Assessment timepoints include from 1-hr before to up to 9 hrs after ingestion of the meal at 1-hour interval. Postprandial AUC results were calculated using a linear trapezoidal rule for each postprandial measure in the subset of participants who had postprandial assessments 0-9 hour results at baseline and the postbaseline between Week 13 to 19. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| IONIS Investigative Site | Encinitas | California | 92024 | United States | ||
| IONIS Investigative Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31390500 | Result | Witztum JL, Gaudet D, Freedman SD, Alexander VJ, Digenio A, Williams KR, Yang Q, Hughes SG, Geary RS, Arca M, Stroes ESG, Bergeron J, Soran H, Civeira F, Hemphill L, Tsimikas S, Blom DJ, O'Dea L, Bruckert E. Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome. N Engl J Med. 2019 Aug 8;381(6):531-542. doi: 10.1056/NEJMoa1715944. | |
| 37164837 |
Not provided
Not provided
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67 participants were randomized, and 66 received study drug. The study included an 8-week screening period (including a diet-stabilization period), a 52-week treatment period, and a 13-week post-treatment evaluation period.
67 participants were randomized at 40 study centers in the United States, Canada, Brazil, France, Germany, Israel, Italy, Netherlands, South Africa, Spain, and the United Kingdom.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Volanesorsen-matching placebo administered subcutaneously once-weekly for 52 weeks. |
| FG001 | Volanesorsen | Volanesorsen 300 mg administered subcutaneously once-weekly for 52 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Placebo | Drug |
|
| Baseline to an on-treatment assessment between Week 13 and Week 19 |
| Absolute Change From Baseline in Fasting TG at Month 3 | The Month 3 endpoint was defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments. | Baseline to 3 months |
| Treatment Response Rate Defined as Participants With Fasting Plasma TG < 750 mg/dL at Month 3 | The Month 3 endpoint was defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments. mg/dL = milligrams per deciliter | Baseline to 3 months |
| Treatment Response Rate Defined as Participants With Fasting TG ≥ 40% Reduction From Baseline at Month 3 | The Month 3 endpoint was defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments. | Baseline to 3 months |
| Frequency and Severity of Participant-reported Abdominal Pain During the Treatment Period | Abdominal pain was measured according to the Bracket electronic patient-reported outcomes (ePRO) assessment. Scores were categorized as follows: no pain (pain score: 0), mild (pain score: 1-3), moderate (pain score: 4-6), or severe (pain score: 7-10). The yearly frequency was calculated as the number of episodes during the on-treatment period / (last dose date - first dose date + 28) * 365.25. Missing data were imputed by using next observation carried back (NOCB) if there was a subsequent score available. | Baseline to 12 months |
| Frequency of the Composite of Episodes of Acute Pancreatitis and Participant-reported Moderate/Severe Abdominal Pain During the Treatment Period | Moderate/severe abdominal pain was defined as having a pain score of 4-10 on the Bracket electronic patient-reported outcomes (ePRO) assessment. Scores were categorized as follows: no pain (pain score: 0), mild (pain score: 1-3), moderate (pain score: 4-6), or severe (pain score: 7-10). The yearly frequency was calculated as the number of episodes during the on-treatment period / (last dose date - first dose date + 28) * 365.25. | 12 months |
| Change From Baseline in Hepatosplenomegaly as Assessed by MRI at Week 52 | The Week 52 endpoint was defined as the average of Week 50 (Day 344)/Week 51 (Day 351) and Week 52 (Day 358) fasting assessments. | Baseline to Week 52 |
| San Francisco |
| California |
| 94143 |
| United States |
| IONIS Investigative Site | Kansas City | Kansas | 66214 | United States |
| IONIS Investigative Site | Boston | Massachusetts | 02114 | United States |
| IONIS Investigative Site | New York | New York | 10016 | United States |
| IONIS Investigative Site | Oklahoma City | Oklahoma | 73103 | United States |
| IONIS Investigative Site | Portland | Oregon | 97239 | United States |
| IONIS Investigative Site | Philadelphia | Pennsylvania | 19104 | United States |
| IONIS Investigative Site | Houston | Texas | 77030 | United States |
| IONIS Investigative Site | Norfolk | Virginia | 23510 | United States |
| IONIS Investigative Site | Seattle | Washington | 98104 | United States |
| IONIS Investigative Site | Campinas | 13059-740 | Brazil |
| IONIS Investigative Site | São Paulo | 04039-030 | Brazil |
| IONIS Investigative Site | São Paulo | 05403-000 | Brazil |
| IONIS Investigative Site | Vancouver | British Columbia | V6Z1Y6 | Canada |
| IONIS Investigative Site | Chicoutimi | Quebec | G7H 5H6 | Canada |
| IONIS Investigative Site | Sainte-Foy | Quebec | G1V 4M6 | Canada |
| IONIS Investigative Site | Marseille | 13385 | France |
| IONIS Investigative Site | Nantes | 44093 | France |
| IONIS Investigative Site | Paris | 75013 | France |
| IONIS Investigative Site | Berlin | 13353 | Germany |
| IONIS Investigative Site | Dresden | 01307 | Germany |
| IONIS Investigative Site | Szikszó | 3800 | Hungary |
| IONIS Investigative Site | Safed | 13110 | Israel |
| IONIS Investigative Site | Milan | 20162 | Italy |
| IONIS Investigative Site | Palermo | 90127 | Italy |
| IONIS Investigative Site | Rome | 00161 | Italy |
| IONIS Investigative Site | Amsterdam | 1105 AZ | Netherlands |
| IONIS Investigative Site | Rotterdam | 3000 | Netherlands |
| IONIS Investigative Site | Cape Town | 7925 | South Africa |
| IONIS Investigative Site | Zaragoza | Aragon | 50009 | Spain |
| IONIS Investigative Site | A Coruña | Galicia | 15001 | Spain |
| IONIS Investigative Site | Barcelona | 08036 | Spain |
| IONIS Investigative Site | Madrid | 28007 | Spain |
| IONIS Investigative Site | Málaga | 29010 | Spain |
| IONIS Investigative Site | Seville | 41013 | Spain |
| IONIS Investigative Site | Birmingham | B9 5SS | United Kingdom |
| IONIS Investigative Site | Manchester | M13 9WL | United Kingdom |
| IONIS Investigative Site | Manchester | M23 9LT | United Kingdom |
| IONIS Investigative Site | Peterborough | PE3 9GZ | United Kingdom |
| Prohaska TA, Alexander VJ, Karwatowska-Prokopczuk E, Tami J, Xia S, Witztum JL, Tsimikas S. APOC3 inhibition with volanesorsen reduces hepatic steatosis in patients with severe hypertriglyceridemia. J Clin Lipidol. 2023 May-Jun;17(3):406-411. doi: 10.1016/j.jacl.2023.04.007. Epub 2023 Apr 27. |
| 29748148 | Derived | Hegele RA, Berberich AJ, Ban MR, Wang J, Digenio A, Alexander VJ, D'Erasmo L, Arca M, Jones A, Bruckert E, Stroes ES, Bergeron J, Civeira F, Witztum JL, Gaudet D. Clinical and biochemical features of different molecular etiologies of familial chylomicronemia. J Clin Lipidol. 2018 Jul-Aug;12(4):920-927.e4. doi: 10.1016/j.jacl.2018.03.093. Epub 2018 Apr 4. |
| 27271183 | Derived | Digenio A, Dunbar RL, Alexander VJ, Hompesch M, Morrow L, Lee RG, Graham MJ, Hughes SG, Yu R, Singleton W, Baker BF, Bhanot S, Crooke RM. Antisense-Mediated Lowering of Plasma Apolipoprotein C-III by Volanesorsen Improves Dyslipidemia and Insulin Sensitivity in Type 2 Diabetes. Diabetes Care. 2016 Aug;39(8):1408-15. doi: 10.2337/dc16-0126. Epub 2016 Jun 6. |
| Dosed |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
The safety set included all randomized participants who received at least one dose of study drug.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Volanesorsen-matching placebo administered subcutaneously once-weekly for 52 weeks. |
| BG001 | Volanesorsen | Volanesorsen 300 mg administered subcutaneously once-weekly for 52 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 | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Region of Enrollment | Number | participants |
| ||||||||||||||||
| Fasting Triglycerides | Mean | Standard Deviation | milligrams per deciliter (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 in Fasting Triglycerides (TG) From Baseline to Month 3 | The Month 3 endpoint was defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments. | The full analysis set included all participants who were randomized, received at least one dose of study drug, and had a baseline TG assessment. | Posted | Least Squares Mean | 95% Confidence Interval | percent change | Baseline to 3 months |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Postprandial TG Area Under the Curve (AUC)(0-9h) | Participants had 2 postprandial assessments - one at Baseline (completed at least 48 hours prior to first dose) and one at any time between Week 13 and 19, inclusive. Assessment timepoints include from 1-hr before to up to 9 hrs after ingestion of the meal at 1-hour interval. Postprandial AUC results were calculated using a linear trapezoidal rule for each postprandial measure in the subset of participants who had postprandial assessments 0-9 hour results at baseline and the postbaseline between Week 13 to 19. | The full analysis set included all participants who were randomized, received at least one dose of study drug, and had a baseline TG assessment. Data were reported for evaluable participants. | Posted | Mean | Standard Deviation | millimole hours per liter (mmol*h/L) | Baseline to an on-treatment assessment between Week 13 and Week 19 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline in Fasting TG at Month 3 | The Month 3 endpoint was defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments. | The full analysis set included all participants who were randomized, received at least one dose of study drug, and had a baseline TG assessment. | Posted | Least Squares Mean | 95% Confidence Interval | mg/dL | Baseline to 3 months |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Treatment Response Rate Defined as Participants With Fasting Plasma TG < 750 mg/dL at Month 3 | The Month 3 endpoint was defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments. mg/dL = milligrams per deciliter | The full analysis set included all participants who were randomized, received at least one dose of study drug, and had a baseline TG assessment. Data were reported for evaluable participants. | Posted | Number | participants | Baseline to 3 months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Treatment Response Rate Defined as Participants With Fasting TG ≥ 40% Reduction From Baseline at Month 3 | The Month 3 endpoint was defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments. | The full analysis set included all participants who were randomized, received at least one dose of study drug, and had a baseline TG assessment. | Posted | Number | participants | Baseline to 3 months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Frequency and Severity of Participant-reported Abdominal Pain During the Treatment Period | Abdominal pain was measured according to the Bracket electronic patient-reported outcomes (ePRO) assessment. Scores were categorized as follows: no pain (pain score: 0), mild (pain score: 1-3), moderate (pain score: 4-6), or severe (pain score: 7-10). The yearly frequency was calculated as the number of episodes during the on-treatment period / (last dose date - first dose date + 28) * 365.25. Missing data were imputed by using next observation carried back (NOCB) if there was a subsequent score available. | The full analysis set included all participants who were randomized, received at least one dose of study drug, and had a baseline TG assessment. | Posted | Number | participants | Baseline to 12 months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Frequency of the Composite of Episodes of Acute Pancreatitis and Participant-reported Moderate/Severe Abdominal Pain During the Treatment Period | Moderate/severe abdominal pain was defined as having a pain score of 4-10 on the Bracket electronic patient-reported outcomes (ePRO) assessment. Scores were categorized as follows: no pain (pain score: 0), mild (pain score: 1-3), moderate (pain score: 4-6), or severe (pain score: 7-10). The yearly frequency was calculated as the number of episodes during the on-treatment period / (last dose date - first dose date + 28) * 365.25. | The full analysis set included all participants who were randomized, received at least one dose of study drug, and had a baseline TG assessment. | Posted | Mean | Standard Deviation | events per participant per year | 12 months |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Hepatosplenomegaly as Assessed by MRI at Week 52 | The Week 52 endpoint was defined as the average of Week 50 (Day 344)/Week 51 (Day 351) and Week 52 (Day 358) fasting assessments. | The full analysis set included all participants who were randomized, received at least one dose of study drug, and had a baseline TG assessment. | Posted | Least Squares Mean | 95% Confidence Interval | cubic centimeters (cm^3) | Baseline to Week 52 |
|
|
Up to approximately 65 weeks.
The safety set included all randomized participants who received at least one dose of study drug.
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 | Volanesorsen-matching placebo administered subcutaneously once-weekly for 52 weeks. | 0 | 33 | 5 | 33 | 27 | 33 |
| EG001 | Volanesorsen | Volanesorsen 300 mg administered subcutaneously once-weekly for 52 weeks. | 0 | 33 | 7 | 33 | 32 | 33 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Colitis ischaemic | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pancreatitis acute | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cyst | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cholangitis | Hepatobiliary disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Drug-induced liver injury | Hepatobiliary disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Ankle fracture | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Liver function test abnormal | Investigations | MedDRA (19.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Injection site erythema | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site swelling | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site pruritus | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site discolouration | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site induration | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site bruising | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site oedema | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site reaction | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site hypoaesthesia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site pallor | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site warmth | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Oedema | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Chills | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site dryness | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site haematoma | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site urticaria | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Creatinine renal clearance decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hot flush | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Diabetes mellitus | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Vaginal haemorrhage | Reproductive system and breast disorders | MedDRA (19.1) | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Ionis Pharmaceuticals, Inc. | Ionis Pharmaceuticals, Inc. | 800-679-4747 | patients@ionisph.com |
| ID | Term |
|---|---|
| C538489 | Familial hyperchylomicronemia syndrome |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| C000593612 | ISIS 304801 |
| D053305 | Apolipoprotein C-III |
| ID | Term |
|---|---|
| D001056 | Apolipoproteins C |
| D001053 | Apolipoproteins |
| D008074 | Lipoproteins |
| D008055 | Lipids |
| D001059 | Apoproteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
Not provided
Not provided
| Male |
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| Not Hispanic or Latino |
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| Asian |
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| Other Race |
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| Netherlands |
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| United States |
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| Brazil |
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| Italy |
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| United Kingdom |
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| South Africa |
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| Israel |
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| France |
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| Germany |
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| Spain |
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| Participants |
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