Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2014-003434-93 | 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 26 weeks in participants with Hypertriglyceridemia.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Volanesorsen-matching placebo administered subcutaneously once-weekly for 26 weeks. |
|
| Volanesorsen 300 mg weekly | Experimental | Volanesorsen 300 mg administered subcutaneously once-weekly for 26 weeks. |
|
| Volanesorsen 300 mg biweekly, post Week 13 | Experimental | Volanesorsen 300 mg administered subcutaneously once-weekly for 13 weeks, then bi-weekly for 13 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Volanesorsen | Drug | 300 mg volanesorsen administered subcutaneously once-weekly for 26 weeks. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change in Fasting Triglycerides (TG) From Baseline to Month 3 | Baseline to 3 months |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change in Fasting TG From Baseline to Month 3 | Baseline to 3 months | |
| Treatment Response Rate Defined as Participants With Fasting TG ≥ 40% Reduction From Baseline at Month 3 | Baseline to 3 months |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| IONIS Investigative Site | Encinitas | California | 92024 | United States | ||
| IONIS Investigative Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37164837 | Derived | 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. | |
| 33798466 | Derived |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
114 participants were randomized, and 113 received study drug. One patient was randomized, but discontinued before dosing, and thus included only in the volanesorsen Total (Not public) column. The study included a ≤ 8-week screening period (including a diet-stabilization period), a 26-week treatment period, and a 13-week post-treatment evaluation period.
A total of 114 participants were randomized at multiple study centers worldwide.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Volanesorsen-matching placebo administered subcutaneously once-weekly for 26 weeks. |
| FG001 | Volanesorsen 300 mg Weekly | Volanesorsen 300 mg administered subcutaneously once-weekly for 26 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
| Placebo | Drug | Volanesorsen-matching placebo administered subcutaneously once-weekly for 26 weeks. |
|
| Percent Change in High-density Lipoprotein-cholesterol (HDL-C) From Baseline | Baseline to 3 months |
| Treatment Response Rate Defined as Participants With Fasting TG < 150 mg/dL Reduction From Baseline at Month 3 | mg/dL = milligrams per deciliter | Baseline to 3 months |
| Change From Baseline in Homeostasis Model Assessment-estimated Insulin Resistance (HOMA-IR) | HOMA-IR was calculated using the following formula: fasting insulin micro-international units per millimeter (μIU/mL) x fasting glucose mg/dL]/405. A negative change from baseline indicates improvement; a positive change from baseline indicates worsening. | Baseline to 3 and 6 months |
| Change From Baseline in Glycated Hemoglobin (HbA1c) in Type 2 Diabetes Mellitus (T2DM) Participants | Baseline to 3 and 6 months |
| San Francisco |
| California |
| 94143 |
| United States |
| IONIS Investigative Site | Boca Raton | Florida | 33434 | United States |
| IONIS Investigative Site | Miami | Florida | 33147 | United States |
| IONIS Investigative Site | Sterling | Illinois | 61081 | United States |
| IONIS Investigative Site | Kansas City | Kansas | 66160 | United States |
| IONIS Investigative Site | Salisbury | Maryland | 73103 | United States |
| IONIS Investigative Site | Towson | Maryland | 21204 | United States |
| IONIS Investigative Site | Boston | Massachusetts | 02114 | United States |
| IONIS Investigative Site | Grandville | Michigan | 49418 | United States |
| IONIS Investigative Site | North Massapequa | New York | 11758-1802 | United States |
| IONIS Investigative Site | Benson | North Carolina | 27504 | United States |
| IONIS Investigative Site | Chapel Hill | North Carolina | 27609 | United States |
| IONIS Investigative Site | Farmville | North Carolina | 27828 | United States |
| IONIS Investigative Site | Greenville | North Carolina | 27834 | United States |
| IONIS Investigative Site | Morrisville | North Carolina | 27560 | United States |
| IONIS Investigative Site | Raleigh | North Carolina | 27612 | United States |
| IONIS Investigative Site | Wilson | North Carolina | 27609 | United States |
| IONIS Investigative Site | Wilson | North Carolina | 27893 | United States |
| IONIS Investigative Site | Cincinnati | Ohio | 45227 | United States |
| IONIS Investigative Site | Kettering | Ohio | 45429 | United States |
| IONIS Investigative Site | Marion | Ohio | 43302 | United States |
| IONIS Investigative Site | Oklahoma City | Oklahoma | 73103 | United States |
| IONIS Investigative Site | Portland | Oregon | 97239 | United States |
| IONIS Investigative Site | Providence | Rhode Island | 02906 | United States |
| IONIS Investigative Site | Houston | Texas | 77030 | United States |
| IONIS Investigative Site | Salt Lake City | Utah | 84108 | United States |
| IONIS Investigative Site | Norfolk | Virginia | 23510 | United States |
| IONIS Investigative Site | Seattle | Washington | 98104 | United States |
| IONIS Investigative Site | Vancouver | British Columbia | V6Z 1Y6 | Canada |
| IONIS Investigative Site | London | Ontario | N6A 5B7 | Canada |
| IONIS Investigative Site | Chicoutimi | Quebec | G7H 7K9 | Canada |
| IONIS Investigative Site | Sainte-Foy | Quebec | G1V 4M6 | Canada |
| IONIS Investigative Site | Dijon | 21079 | France |
| IONIS Investigative Site | Marseille | 13385 | France |
| IONIS Investigative Site | Paris | 75013 | France |
| IONIS Investigative Site | Saint-Herblain | France |
| IONIS Investigative Site | Cologne | North Rhine-Westphalia | 50937 | Germany |
| IONIS Investigative Site | Berlin | 13353 | Germany |
| IONIS Investigative Site | Dresden | 01307 | Germany |
| IONIS Investigative Site | Amsterdam | North Holland | 1105 AZ | Netherlands |
| IONIS Investigative Site | Rotterdam | South Holland | 3045 PM | Netherlands |
| IONIS Investigative Site | Utrecht | 3584 CX | Netherlands |
| IONIS Investigative Site | Manchester | M23 9LT | United Kingdom |
| IONIS Investigative Site | Peterborough | PE3 6DA | United Kingdom |
| Gouni-Berthold I, Alexander VJ, Yang Q, Hurh E, Steinhagen-Thiessen E, Moriarty PM, Hughes SG, Gaudet D, Hegele RA, O'Dea LSL, Stroes ESG, Tsimikas S, Witztum JL; COMPASS study group. Efficacy and safety of volanesorsen in patients with multifactorial chylomicronaemia (COMPASS): a multicentre, double-blind, randomised, placebo-controlled, phase 3 trial. Lancet Diabetes Endocrinol. 2021 May;9(5):264-275. doi: 10.1016/S2213-8587(21)00046-2. Epub 2021 Mar 30. |
| FG002 | Volanesorsen 300 mg Biweekly, Post Week 13 | Volanesorsen 300 mg administered subcutaneously once-weekly for 13 weeks, then bi-weekly for 13 weeks. |
| COMPLETED | Completed here refers to participants who completed the study treatment. |
|
| NOT COMPLETED |
|
|
The full analysis set (FAS) included all participants who were randomized, received at least one dose of study drug, and had a baseline triglycerides (TG) assessment.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Volanesorsen-matching placebo administered subcutaneously once-weekly for 26 weeks. |
| BG001 | Volanesorsen 300 mg Weekly | Volanesorsen 300 mg administered subcutaneously once-weekly for 26 weeks. |
| BG002 | Volanesorsen 300 mg Biweekly, Post Week 13 | Volanesorsen 300 mg administered subcutaneously once-weekly for 13 weeks, then bi-weekly for 13 weeks. |
| BG003 | 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 |
| ||||||||||||||||
| 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 full analysis set (FAS) 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 | Absolute Change in Fasting TG From Baseline to Month 3 | The FAS included all participants who were randomized, received at least one dose of study drug, and had a baseline TG assessment. Volanesorsen 300 mg biweekly group includes patients who received weekly dosing in first 13 weeks, and then bi-weekly for 13 weeks. For month 3 assessments, the results were combined since all patients were on weekly dosing. And for month 6 assessments, the results were split to show the results in each dosing group. | Posted | Least Squares Mean | 95% Confidence Interval | mg/dL | Baseline to 3 months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Treatment Response Rate Defined as Participants With Fasting TG ≥ 40% Reduction From Baseline at Month 3 | The FAS included all participants who were randomized, received at least one dose of study drug, and had a baseline TG assessment. | Posted | Count of Participants | Participants | Baseline to 3 months |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change in High-density Lipoprotein-cholesterol (HDL-C) From Baseline | The FAS 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 | Treatment Response Rate Defined as Participants With Fasting TG < 150 mg/dL Reduction From Baseline at Month 3 | mg/dL = milligrams per deciliter | The FAS included all participants who were randomized, received at least one dose of study drug, and had a baseline TG assessment. | Posted | Count of Participants | Participants | Baseline to 3 months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Homeostasis Model Assessment-estimated Insulin Resistance (HOMA-IR) | HOMA-IR was calculated using the following formula: fasting insulin micro-international units per millimeter (μIU/mL) x fasting glucose mg/dL]/405. A negative change from baseline indicates improvement; a positive change from baseline indicates worsening. | The FAS included all participants who were randomized, received at least one dose of study drug, and had a baseline TG assessment. Number analyzed were the participants evaluated at the given time point. | Posted | Mean | Standard Deviation | score | Baseline to 3 and 6 months |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Glycated Hemoglobin (HbA1c) in Type 2 Diabetes Mellitus (T2DM) Participants | The FAS included all participants who were randomized, received at least one dose of study drug, and had a baseline TG assessment. Number analyzed were the T2DM participants evaluated at the given time point. | Posted | Mean | Standard Deviation | percentage | Baseline to 3 and 6 months |
|
|
Up to approximately 39 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 26 weeks. | 0 | 38 | 4 | 38 | 31 | 38 |
| EG001 | Volanesorsen 300 mg Weekly | Volanesorsen 300 mg administered subcutaneously once-weekly for 26 weeks. | 0 | 25 | 2 | 25 | 24 | 25 |
| EG002 | Volanesorsen 300 mg Biweekly, Post Week 13 | Volanesorsen 300 mg administered subcutaneously once-weekly for 13 weeks, then bi-weekly for 13 weeks. | 0 | 50 | 6 | 50 | 49 | 50 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vertigo | Ear and labyrinth disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Ileus paralytic | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pancreatitis acute | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pancreatitis relapsing | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Serum sickness | Immune system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pancreas infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Ulna fracture | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Carotid artery stenosis | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hemiparesis | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hypertensive crisis | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Peripheral arterial occlusive disease | Vascular disorders | 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 |
| |
| Injection site swelling | 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 discomfort | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site bruising | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site rash | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site reaction | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site warmth | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site haemorrhage | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site hypoaesthesia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site inflammation | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site mass | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site oedema | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | 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 |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dry mouth | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Red blood cell sedimentation rate increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Low density lipoprotein increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Diabetes mellitus | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Gout | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Type 2 diabetes mellitus | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Actinic keratosis | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Albuminuria | Renal and urinary disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hot flush | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hepatic pain | Hepatobiliary 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 |
|---|---|
| D015228 | Hypertriglyceridemia |
| ID | Term |
|---|---|
| D006949 | Hyperlipidemias |
| D050171 | Dyslipidemias |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000593612 | ISIS 304801 |
Not provided
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Asian |
|
| American Indian or Alaskan Native |
|
| Other Race |
|
| Multiple |
|
|
|
|
|
|
|
|
|
| Units | Counts |
|---|
| Participants |
|
|
|