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| Name | Class |
|---|---|
| Ionis Pharmaceuticals, Inc. | INDUSTRY |
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This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of triglyceride (TG) levels in participants with FCS.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| AKCEA-ANGPTL3-LRx 20 mg | Experimental | Participants received a subcutaneous (SC) injection of AKCEA-ANGPTL3-LRx, 20 milligrams (mg), weekly (QW) for 13-weeks of treatment period. Participants were followed up to Week 26. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AKCEA-ANGPTL3-LRx | Drug | AKCEA-ANGPTL3-LRx at dose 20 mg, administered via SC injection QW. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG) | Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments. | Baseline to Month 3 |
| Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG) | Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments. | Baseline to Month 3 |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change From Baseline to Month 3 in Fasting Angiopoietin-Like 3 (ANGPTL3) | Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments. | Baseline to Month 3 |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigative Site | Montreal | Quebec | H2W 1R7 | Canada |
A total of 4 participants were screened, 3 of whom were enrolled and treated with at least one dose of study drug and were included in the analysis. This study consisted of up to an 8-week screening period, a 13-week treatment period and a 13-week follow-up (post-treatment evaluation) period.
Participants took part in the study at 1 study site in Canada from 21-December-2017 to 4-September-2018.
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| ID | Title | Description |
|---|---|---|
| FG000 | AKCEA-ANGPTL3-LRx 20 mg | Participants received a subcutaneous (SC) injection of AKCEA-ANGPTL3-LRx, 20 milligrams (mg), weekly (QW) for 13-weeks of treatment period. Participants were followed up to Week 26. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | AKCEA-ANGPTL3-LRx 20 mg | Participants received a SC injection of AKCEA-ANGPTL3-LRx, 20 mg, QW for 13-weeks of treatment period. Participants were followed up to Week 26. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG) | Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | mg/dL | Baseline to Month 3 |
|
From time of informed consent to end of follow-up period (Up to Week 26).
Safety set included all participants who were enrolled and received at least 1 dose of study drug.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | AKCEA-ANGPTL3-LRx 20 mg | Participants received a SC injection of AKCEA-ANGPTL3-LRx, 20 mg, QW for 13-weeks of treatment period. Participants were followed up to Week 26. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Asthenia | General disorders | MedDRA 20.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Akcea Therapeutics | 617-207-0289 | clinicalstudies@akceatx.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Oct 26, 2017 | Dec 11, 2020 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| C538489 | Familial hyperchylomicronemia syndrome |
| D008072 | Hyperlipoproteinemia Type I |
| D006951 | Hyperlipoproteinemias |
| D006949 | Hyperlipidemias |
| D050171 | Dyslipidemias |
| D052439 | Lipid Metabolism Disorders |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| ID | Term |
|---|---|
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| C000621591 | IONIS-ANGPTL3-LRx |
| C000723171 | vupanorsen |
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| Percent Change From Baseline to Month 3 in Fasting Angiopoietin-like 3 (ANGPTL3) |
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments. |
| Baseline to Month 3 |
| Fasting Lipid and Lipoprotein Measurements at Month 3 | Fasting lipid and lipoprotein measurements included non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C and LDL-C. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments. | Month 3 |
| Absolute Change From Baseline to Month 3 in Other Fasting Lipid Parameters | Other fasting lipid measurements included total cholesterol (TC), non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C, and LDL-C. Baseline was defined as average of Day 1 predose fasting assessment and last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments. | Baseline to Month 3 |
| Percent (%) Change From Baseline to Month 3 in Other Fasting Lipid Parameters | Other fasting lipid measurements included TC, non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C, and LDL-C. Baseline was defined as average of Day 1 predose fasting assessment and last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments. | Baseline to Month 3 |
| Change From Baseline to Day 92 in Maximum Postprandial Triglycerides (TG) | Participants consumed standardized pre-cooked meals (lunches and dinners and instructions for breakfasts and snacks) for 2 days prior to the postprandial assessments. Change from Baseline to Day 92 in maximum postprandial TG was assessed. | Baseline to Day 92 |
| Number of Participants Who Experienced Abdominal Pain During the Treatment Period | Days 1, 29, 57 and 92 |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | An adverse event (AE) was defined as any unfavorable and unintended sign (including a clinically-significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE is considered related to the investigational drug product. A TEAE was defined as any AE starting on or after the first dose of the study drug. | From time of informed consent to end of follow-up period (Up to Week 26) |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Fasting Triglycerides (TG) | Mean | Standard Deviation | milligrams per deciliter (mg/dL) |
|
| Fasting Angiopoietin-Like 3 (ANGPTL3) | Mean | Standard Deviation | nanograms per milliliter (ng/mL) |
|
| Fasting Total Cholesterol (TC) | Mean | Standard Deviation | mg/dL |
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| Fasting Non-High Density Lipoprotein Cholesterol (Non-HDL-C) | Mean | Standard Deviation | mg/dL |
|
| Fasting Apolipoprotein B (ApoB) | Mean | Standard Deviation | mg/dL |
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| Fasting High Density Lipoprotein Cholesterol (HDL-C) | Mean | Standard Deviation | mg/dL |
|
| Fasting Apolipoprotein A-1 (ApoA-1) | Mean | Standard Deviation | mg/dL |
|
| Fasting Very Low Density Lipoprotein Cholesterol (VLDL-C) | Mean | Standard Deviation | mg/dL |
|
| Fasting Low Density Lipoprotein Cholesterol (LDL-C) | Mean | Standard Deviation | mg/dL |
|
| Maximum Postprandial Triglycerides | Mean | Standard Deviation | mg/dL |
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
|
| Primary | Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG) | Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | percent change | Baseline to Month 3 |
|
|
|
| Secondary | Absolute Change From Baseline to Month 3 in Fasting Angiopoietin-Like 3 (ANGPTL3) | Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | ng/mL | Baseline to Month 3 |
|
|
|
| Secondary | Percent Change From Baseline to Month 3 in Fasting Angiopoietin-like 3 (ANGPTL3) | Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | percent change | Baseline to Month 3 |
|
|
|
| Secondary | Fasting Lipid and Lipoprotein Measurements at Month 3 | Fasting lipid and lipoprotein measurements included non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C and LDL-C. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | mg/dL | Month 3 |
|
|
|
| Secondary | Absolute Change From Baseline to Month 3 in Other Fasting Lipid Parameters | Other fasting lipid measurements included total cholesterol (TC), non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C, and LDL-C. Baseline was defined as average of Day 1 predose fasting assessment and last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | mg/dL | Baseline to Month 3 |
|
|
|
| Secondary | Percent (%) Change From Baseline to Month 3 in Other Fasting Lipid Parameters | Other fasting lipid measurements included TC, non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C, and LDL-C. Baseline was defined as average of Day 1 predose fasting assessment and last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | percent change | Baseline to Month 3 |
|
|
|
| Secondary | Change From Baseline to Day 92 in Maximum Postprandial Triglycerides (TG) | Participants consumed standardized pre-cooked meals (lunches and dinners and instructions for breakfasts and snacks) for 2 days prior to the postprandial assessments. Change from Baseline to Day 92 in maximum postprandial TG was assessed. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | mg/dL | Baseline to Day 92 |
|
|
|
| Secondary | Number of Participants Who Experienced Abdominal Pain During the Treatment Period | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Count of Participants | Participants | Days 1, 29, 57 and 92 |
|
|
|
| Secondary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | An adverse event (AE) was defined as any unfavorable and unintended sign (including a clinically-significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE is considered related to the investigational drug product. A TEAE was defined as any AE starting on or after the first dose of the study drug. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Count of Participants | Participants | From time of informed consent to end of follow-up period (Up to Week 26) |
|
|
|
| 0 |
| 3 |
| 0 |
| 3 |
| 3 |
| 3 |
| Influenza like illness | General disorders | MedDRA 20.1 | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 20.1 | Systematic Assessment |
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| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 20.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 20.1 | Systematic Assessment |
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| Vertigo positional | Ear and labyrinth disorders | MedDRA 20.1 | Systematic Assessment |
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| Influenza | Infections and infestations | MedDRA 20.1 | Systematic Assessment |
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| Hyperlipidaemia | Metabolism and nutrition disorders | MedDRA 20.1 | Systematic Assessment |
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| Loss of libido | Psychiatric disorders | MedDRA 20.1 | Systematic Assessment |
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| Mood swings | Psychiatric disorders | MedDRA 20.1 | Systematic Assessment |
|
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| Title | Measurements |
|---|---|
|
| ApoA-1 |
|
| VLDL-C |
|
| LDL-C |
|
|
| Absolute Change From Baseline to Month 3 in HDL-C |
|
| Absolute Change From Baseline to Month 3 in ApoA-1 |
|
| Absolute Change From Baseline to Month 3 in VLDL-C |
|
| Absolute Change From Baseline to Month 3 in LDL-C |
|
|
| % Change From Baseline to Month 3 in HDL-C |
|
| % Change From Baseline to Month 3 in ApoA-1 |
|
| % Change From Baseline to Month 3 in VLDL-C |
|
| % Change From Baseline to Month 3 in LDL-C |
|