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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 fasting triglycerides in participants with familial partial lipodystrophy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| AKCEA-ANGPTL3-LRx 20 mg | Experimental | Participants received AKCEA-ANGPTL3-LRx 20 milligrams (mg) administered every week for 26 weeks by subcutaneous (SC) injection. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AKCEA-ANGPTL3-LRx | Drug | AKCEA-ANGPTL3-LRx solution for SC injection. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in Fasting Triglycerides Levels at End of the Treatment (Week 27) | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Area Under the Curve (AUC) of Plasma Glucose as Assessed by Mixed Meal Test (MMT) at End of the Treatment | Change from Baseline to Week 27 in the area under the curve (AUC) of Plasma Glucose was assessed. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in AUC of Serum Insulin as Assessed by MMT at End of the Treatment |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Site | Ann Arbor | Michigan | 48105 | United States |
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A total of 8 participants diagnosed with familial partial lipodystrophy (FPL) were screened, out of which 4 participants were treated with AKCEA-ANGPTL3-LRx 20 milligrams (mg). All 4 participants completed the study.
This study was conducted at a single center in the United States of America from 15 June 2018 to 21 August 2019.
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| ID | Title | Description |
|---|---|---|
| FG000 | AKCEA-ANGPTL3-LRx 20 mg | Participants received AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by subcutaneous (SC) injection. |
| 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 AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by SC injection. |
| 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 | Percent Change From Baseline in Fasting Triglycerides Levels at End of the Treatment (Week 27) | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | percent change | Baseline and End of the Treatment (Week 27) |
|
|
From signing of informed consent to end of follow up period (Up to week 40)
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 AKCEA-ANGPTL3-LRx 20 mg administered every week for 26 weeks by subcutaneous (SC) injection. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute coronary syndrome | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hot flush | Vascular 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 | Feb 16, 2018 | Jan 6, 2021 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D052496 | Lipodystrophy, Familial Partial |
| D008060 | Lipodystrophy |
| D052439 | Lipid Metabolism Disorders |
| D050171 | Dyslipidemias |
| D003923 | Diabetes Mellitus, Lipoatrophic |
| ID | Term |
|---|---|
| D000083083 | Laminopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012875 | Skin Diseases, Metabolic |
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Change from Baseline to Week 27 in the AUC of Serum Insulin was assessed. |
| Baseline and End of the Treatment (Week 27) |
| Change From Baseline in AUC of Serum C-peptide as Assessed by MMT at End of the Treatment | Change from Baseline to Week 27 in the AUC of Serum C-peptide was assessed. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in AUC of Free Fatty Acid (FFA) as Assessed by MMT at End of the Treatment | Change from Baseline to Week 27 in the AUC of FFA was assessed. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in AUC of Serum Ghrelin as Assessed by MMT at End of the Treatment | Change from Baseline to Week 27 in the AUC of Serum Ghrelin was assessed. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in AUC of Incretin Hormone (Gastric Inhibitory Polypeptide [GIP]) as Assessed by MMT at End of the Treatment | Change from Baseline to Week 27 in the AUC of Incretin Hormone: GIP was assessed. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in AUC of Incretin Hormone (Glucagon-like Peptide -1 [GLP-1]) as Assessed by MMT at End of the Treatment | Change from Baseline to Week 27 in the AUC of Incretin Hormone: GLP-1 was assessed. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in AUC of Peptide Tyrosine Tyrosine (PYY) as Assessed by MMT at End of the Treatment | Change from Baseline to Week 27 in the AUC of PYY was assessed. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in HDL-C at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in LDL-C at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. LDL-C calculated using ultracentrifugation method. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Total Cholesterol (TC) at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in VLDL-C at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. VLDL-C was calculated using direct test method. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Non-HDL-C at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in ApoB at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in ApoB-48 at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Apolipoprotein B 100 (ApoB-100) at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in ApoA-1 at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in ApoC-III at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in ApoC-III: Chylomicron at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in ApoC-III: VLDL at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in ApoC-III: LDL at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in ApoC-III: HDL at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Lipoprotein a (Lp[a]) at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Free Fatty Acid (FFA) at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Glycerol Levels at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Lipoprotein Particle Size at End of the Treatment | The baseline was defined as the Day 1 pre-dose fasting assessment. Lipoprotein Particle size included: HDL size, LDL size and VLDL size. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Hemoglobin A1c (HbA1c) at End of the Treatment | The baseline was defined as the last non-missing assessment prior to the first dose of study drug. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Homeostasis Model Assessment-Estimated Insulin Resistance (HOMA-IR) | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Adiponectin at End of the Treatment | The baseline was defined as the Day 1 pre-dose fasting assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in and Leptin at End of the Treatment | The baseline was defined as the Day 1 pre-dose fasting assessment | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Hepatic Fat Fraction (HFF) as Assessed by Magnetic Resonance Imaging (MRI) at End of the Treatment | The baseline was defined as the last non-missing assessment prior to the first dose of study drug. | Baseline and End of the Treatment (Week 27) |
| Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Skinfold Thickness at End of the Treatment | The baseline was defined as the last assessment prior to the first dose of study drug. Change in body fat distribution was measured as right anterior thigh skinfold thickness and right tricep skinfold thickness by Skinfold Thickness. | Baseline and End of the Treatment (Week 27) |
| Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment | The baseline was defined as the Screening assessment. Change in body fat distribution (arm bone mass, arm fat mass, arm lean mass, arm total mass, leg bone mass, leg fat mass, leg lean mass, leg total mass, total bone mass, total fat mass, total lean mass, total total mass , trunk bone mass, trunk fat mass, trunk lean mass and trunk total mass) was measures obtained from DEXA. | Baseline and End of the Treatment (Week 27) |
| Changes From Baseline in Body Fat Distribution for Total Bone Mineral Density in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment | The baseline was defined as Screening assessment. Change in body fat distribution for total Bone mineral density was measures obtained from DEXA. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Visceral Adipose Tissue (VAT) as Measured by Magnetic Resonance Imaging (MRI) at End of the Treatment | The baseline was defined as the last assessment prior to the first dose of study drug. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Subcutaneous Adipose Tissue (SAT) as MRI at End of the Treatment | The baseline was defined as the last assessment prior to the first dose of study drug. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Body Weight at End of the Treatment | The baseline was defined as the Day 1 pre-dose assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Waist Circumference at End of the Treatment | The baseline was defined as the Screening assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Waist/Hip Ratio at End of the Treatment | The baseline was defined as screening assessment. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Quality of Life (QoL) | The baseline was defined as the screening assessment. Quality of life measures the severity of fatigue, severity of trouble thinking or remembering and severity of waking up tired in participants, on a scale ranging from 0 to 3, where, 0= No problem, 1= Mild, 2= Moderate and 3= severe. Higher scores indicates more severity or more impact on quality of life. | Baseline and End of the Treatment (Week 27) |
| Change From Baseline in Pain Score at End of the Treatment | The baseline was defined as a Screening assessment. Pain score is used to determine disease activity in participants, on a scale ranging from 0 to 5 where 0= never, 1= hardly noticed, 2= slightly, 3= moderately, 4= strongly, and 5= very strongly where higher scores indicated higher degree of pain. | Baseline and End of the Treatment (Week 27) |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | An adverse event (AE) is 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 treatment-emergent adverse event (TEAE) is defined as any AE starting on or after the first dose of the study drug | From signing of informed consent to end of follow up period (Up to week 40) |
| 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 | milligram per deciliter (mg/dL) |
|
| Fasting Apolipoprotein B-48 (Apo B-48) | Mean | Standard Deviation | mg/dL |
|
| Fasting Apolipoprotein C-III (Apo CIII) | Mean | Standard Deviation | mg/dL |
|
| Fasting Apolipoprotein B (ApoB) | Mean | Standard Deviation | mg/dL |
|
| Fasting Non-High Density Lipoprotein- Cholesterol (Non-HDL-C) | Mean | Standard Deviation | mg/dL |
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| Fasting High Density Lipoprotein- Cholesterol (HDL-C) | Mean | Standard Deviation | mg/dL |
|
| Fasting Apolipoprotein A-I (Apo A-1) | Mean | Standard Deviation | mg/dL |
|
| Fasting Very low Density Lipoprotein-Cholesterol (VLDL-C) | VLDL-C calculated using direct test method. | Number analyzed signifies the number of participants with data available for fasting VLDL-C. | Mean | Standard Deviation | mg/dL |
|
| Fasting Low Density Lipoprotein Cholesterol (LDL-C) | LDL-C calculated using ultracentrifugation method. | Number analyzed signifies the number of participants with data available for fasting LDL-C. | Mean | Standard Deviation | mg/dL |
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| Area Under Curve (AUC) of Plasma Glucose | Mean | Standard Deviation | milligram/deciliter*minute (mg/dL*min) |
|
| AUC of Serum Insulin | Mean | Standard Deviation | milli international units/ liter*minute |
|
| AUC of Serum C-peptide | Number analyzed signifies the number of participants with data available for AUC of Serum C-peptide. | Mean | Standard Deviation | nanogram/milliliter*minute (ng/mL*min) |
|
| AUC of Free Fatty Acid | Number analyzed signifies the number of participants with data available for AUC of Free fatty acid. | Mean | Standard Deviation | milliequivalents/liter*min (mEq/L*min) |
|
| AUC of Gastric Inhibitory Polypeptide (GIP) | Number analyzed signifies the number of participants with data available for AUC of GIP. | Mean | Standard Deviation | picograms/milliliter*minute (pg/mL*min) |
|
| AUC of Glucagon-like Peptide 1 (GLP-1) | Number analyzed signifies the number of participants with data available for AUC of GLP-1. | Mean | Standard Deviation | picomoles/liter*minute (pmol/L*min) |
|
| AUC of Peptide Tyrosine Tyrosine (PYY) | Mean | Standard Deviation | pg/mL*min |
|
| AUC of Serum Ghrelin | Mean | Standard Deviation | pg/mL*min |
|
| Participants |
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| Secondary | Change From Baseline in Area Under the Curve (AUC) of Plasma Glucose as Assessed by Mixed Meal Test (MMT) at End of the Treatment | Change from Baseline to Week 27 in the area under the curve (AUC) of Plasma Glucose 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*min | Baseline and End of the Treatment (Week 27) |
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|
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| Secondary | Change From Baseline in AUC of Serum Insulin as Assessed by MMT at End of the Treatment | Change from Baseline to Week 27 in the AUC of Serum Insulin 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 | milli international units per liter*min | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in AUC of Serum C-peptide as Assessed by MMT at End of the Treatment | Change from Baseline to Week 27 in the AUC of Serum C-peptide 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 | ng/mL*min | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in AUC of Free Fatty Acid (FFA) as Assessed by MMT at End of the Treatment | Change from Baseline to Week 27 in the AUC of FFA 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 | mEq/L*min | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in AUC of Serum Ghrelin as Assessed by MMT at End of the Treatment | Change from Baseline to Week 27 in the AUC of Serum Ghrelin 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 | pg/mL*min | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in AUC of Incretin Hormone (Gastric Inhibitory Polypeptide [GIP]) as Assessed by MMT at End of the Treatment | Change from Baseline to Week 27 in the AUC of Incretin Hormone: GIP 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 | pg/mL*min | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in AUC of Incretin Hormone (Glucagon-like Peptide -1 [GLP-1]) as Assessed by MMT at End of the Treatment | Change from Baseline to Week 27 in the AUC of Incretin Hormone: GLP-1 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 | pmol/L*min | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in AUC of Peptide Tyrosine Tyrosine (PYY) as Assessed by MMT at End of the Treatment | Change from Baseline to Week 27 in the AUC of PYY 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 | pg/mL*min | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in HDL-C at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | mg/dL | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in LDL-C at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. LDL-C calculated using ultracentrifugation method. | 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 and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in Total Cholesterol (TC) at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | mg/dL | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in VLDL-C at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. VLDL-C was calculated using direct test method. | 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 and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in Non-HDL-C at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | mg/dL | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in ApoB at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | mg/dL | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in ApoB-48 at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | mg/dL | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in Apolipoprotein B 100 (ApoB-100) at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | mg/dL | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in ApoA-1 at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | mg/dL | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in ApoC-III at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | mg/dL | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in ApoC-III: Chylomicron at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Data is not available as the collected Baseline samples were not sufficient to assess this outcome measure. | Posted | Baseline and End of the Treatment (Week 27) |
|
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| Secondary | Change From Baseline in ApoC-III: VLDL at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Data is not available as the collected Baseline samples were not sufficient to assess this outcome measure. | Posted | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in ApoC-III: LDL at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | 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 and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in ApoC-III: HDL at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | mg/dL | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in Lipoprotein a (Lp[a]) at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | nanomoles per liter (nmol/L) | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in Free Fatty Acid (FFA) at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | millimoles per liter (mmol/L) | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in Glycerol Levels at End of the Treatment | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | micromoles per liter (μmol/L) | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in Lipoprotein Particle Size at End of the Treatment | The baseline was defined as the Day 1 pre-dose fasting assessment. Lipoprotein Particle size included: HDL size, LDL size and VLDL size. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | nanometer (nm) | Baseline and End of the Treatment (Week 27) |
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|
| Secondary | Change From Baseline in Hemoglobin A1c (HbA1c) at End of the Treatment | The baseline was defined as the last non-missing assessment prior to the first dose of study drug. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | percentage of HbA1c | Baseline and End of the Treatment (Week 27) |
|
|
|
| Secondary | Change From Baseline in Homeostasis Model Assessment-Estimated Insulin Resistance (HOMA-IR) | The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. | Due to the low number of participants, data for this outcome measure was not collected. | Posted | Baseline and End of the Treatment (Week 27) |
|
|
| Secondary | Change From Baseline in Adiponectin at End of the Treatment | The baseline was defined as the Day 1 pre-dose fasting assessment. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | microgram per milliliter (µg/mL) | Baseline and End of the Treatment (Week 27) |
|
|
|
| Secondary | Change From Baseline in and Leptin at End of the Treatment | The baseline was defined as the Day 1 pre-dose fasting assessment | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | nanogram per milliliter (ng/mL) | Baseline and End of the Treatment (Week 27) |
|
|
|
| Secondary | Change From Baseline in Hepatic Fat Fraction (HFF) as Assessed by Magnetic Resonance Imaging (MRI) at End of the Treatment | The baseline was defined as the last non-missing assessment prior to the first dose of study drug. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | percentage of hepatic fat | Baseline and End of the Treatment (Week 27) |
|
|
|
| Secondary | Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Skinfold Thickness at End of the Treatment | The baseline was defined as the last assessment prior to the first dose of study drug. Change in body fat distribution was measured as right anterior thigh skinfold thickness and right tricep skinfold thickness by Skinfold Thickness. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | millimeter (mm) | Baseline and End of the Treatment (Week 27) |
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|
|
| Secondary | Changes From Baseline in Body Fat Distribution for Various Areas in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment | The baseline was defined as the Screening assessment. Change in body fat distribution (arm bone mass, arm fat mass, arm lean mass, arm total mass, leg bone mass, leg fat mass, leg lean mass, leg total mass, total bone mass, total fat mass, total lean mass, total total mass , trunk bone mass, trunk fat mass, trunk lean mass and trunk total mass) was measures obtained from DEXA. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | gram (g) | Baseline and End of the Treatment (Week 27) |
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|
|
| Secondary | Changes From Baseline in Body Fat Distribution for Total Bone Mineral Density in the Body as Measured by Dual-Energy X-ray Absorptiometry (DEXA) at End of the Treatment | The baseline was defined as Screening assessment. Change in body fat distribution for total Bone mineral density was measures obtained from DEXA. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | gram per centimeter square (g/cm^2) | Baseline and End of the Treatment (Week 27) |
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|
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| Secondary | Change From Baseline in Visceral Adipose Tissue (VAT) as Measured by Magnetic Resonance Imaging (MRI) at End of the Treatment | The baseline was defined as the last assessment prior to the first dose of study drug. | Due to the low number of participants, data for this outcome measure was not collected. | Posted | Baseline and End of the Treatment (Week 27) |
|
|
| Secondary | Change From Baseline in Subcutaneous Adipose Tissue (SAT) as MRI at End of the Treatment | The baseline was defined as the last assessment prior to the first dose of study drug. | Due to the low number of participants, data for this outcome measure was not collected. | Posted | Baseline and End of the Treatment (Week 27) |
|
|
| Secondary | Change From Baseline in Body Weight at End of the Treatment | The baseline was defined as the Day 1 pre-dose assessment. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | kg | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in Waist Circumference at End of the Treatment | The baseline was defined as the Screening assessment. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | cm | Baseline and End of the Treatment (Week 27) |
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| Secondary | Change From Baseline in Waist/Hip Ratio at End of the Treatment | The baseline was defined as screening assessment. | Due to the low number of participants, data for this outcome measure was not collected. | Posted | Baseline and End of the Treatment (Week 27) |
|
|
| Secondary | Change From Baseline in Quality of Life (QoL) | The baseline was defined as the screening assessment. Quality of life measures the severity of fatigue, severity of trouble thinking or remembering and severity of waking up tired in participants, on a scale ranging from 0 to 3, where, 0= No problem, 1= Mild, 2= Moderate and 3= severe. Higher scores indicates more severity or more impact on quality of life. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Count of Participants | Participants | Baseline and End of the Treatment (Week 27) |
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|
|
| Secondary | Change From Baseline in Pain Score at End of the Treatment | The baseline was defined as a Screening assessment. Pain score is used to determine disease activity in participants, on a scale ranging from 0 to 5 where 0= never, 1= hardly noticed, 2= slightly, 3= moderately, 4= strongly, and 5= very strongly where higher scores indicated higher degree of pain. | Safety set included all participants who were enrolled and received at least 1 dose of study drug. | Posted | Mean | Standard Deviation | score on a scale | Baseline and End of the Treatment (Week 27) |
|
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| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | An adverse event (AE) is 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 treatment-emergent adverse event (TEAE) is 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 signing of informed consent to end of follow up period (Up to week 40) |
|
|
|
| 0 |
| 4 |
| 3 |
| 4 |
| 4 |
| 4 |
| Angina pectoris | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
|
| Atrial fibrillation | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
|
| Atrial flutter | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
|
| Ventricular tachycardia | Cardiac disorders | MedDRA (20.1) | Systematic Assessment |
|
| Pancreatitis acute | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
|
| Troponin increased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
|
| Albumin urine present | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Antinuclear antibody positive | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Gamma-glutamyltransferase increased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Hepatic steatosis | Hepatobiliary disorders | MedDRA (20.1) | Systematic Assessment |
|
| Blood magnesium decreased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Creatinine urine increased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (20.1) | Systematic Assessment |
|
| Blood bilirubin increased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA (20.1) | Systematic Assessment |
|
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA (20.1) | Systematic Assessment |
|
| Abdominal discomfort | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
|
| International normalised ratio increased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Blood glucose increased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Glycosylated haemoglobin increased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (20.1) | Systematic Assessment |
|
| Conjunctivitis | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
|
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA (20.1) | Systematic Assessment |
|
| Oedema peripheral | General disorders | MedDRA (20.1) | Systematic Assessment |
|
| Insulin C-peptide decreased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Urine albumin/creatinine ratio increased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Depression | Psychiatric disorders | MedDRA (20.1) | Systematic Assessment |
|
| Gastroenteritis viral | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
|
| urinary casts | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
|
| Blood creatinine increased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA (20.1) | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA (20.1) | Systematic Assessment |
|
| Presyncope | Nervous system disorders | MedDRA (20.1) | Systematic Assessment |
|
| Mean cell haemoglobin decreased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Haemoglobin decreased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Mean cell volume decreased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Glomerular filtration rate decreased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Injection site dryness | General disorders | MedDRA (20.1) | Systematic Assessment |
|
| Hypersensitivity | Immune system disorders | MedDRA (20.1) | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (20.1) | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Blood alkaline phosphatase increased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Blood calcium decreased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Blood pressure increased | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Crystal urine present | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Liver function test | Investigations | MedDRA (20.1) | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (20.1) | Systematic Assessment |
|
The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D003924 | Diabetes Mellitus, Type 2 |
| D003920 | Diabetes Mellitus |
| D044882 | Glucose Metabolism Disorders |
| D004700 | Endocrine System Diseases |
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Arm Total Mass |
|
| Leg Bone Mass |
|
| Leg Fat Mass |
|
| Leg Lean Mass |
|
| Leg Total Mass |
|
| Total Bone Mass |
|
| Total Fat Mass |
|
| Total Lean Mass |
|
| Total Total Mass |
|
| Trunk Bone Mass |
|
| Trunk Fat Mass |
|
| Trunk Lean Mass |
|
| Trunk Total Mass |
|
| Severity of trouble thinking or remembering |
|
| Severity of waking up tired |
|