Hypertriglyceridemia
Conditions
Keywords
Severe Hypertriglyceridemia (HTG), Recurrent Acute Pancreatitis
Brief summary
The primary objective of the study is to determine the proportion of patients with elevated triglycerides (TG), without familial chylomicronemia syndrome (FCS) due to loss of function (LoF) mutations in lipoprotein lipase (LPL), and a history of hypertriglyceridemia (HTG)-associated acute pancreatitis (AP) who experience a recurrent episode of AP after treatment with evinacumab versus placebo. The secondary objectives of the study are: * To determine the change in the standard lipid profile after therapy with evinacumab versus placebo * To determine the changes in specialty lipoprotein parameters (ApoC3, ApoB48, ApoB100, and nuclear magnetic resonance \[NMR\] lipid profile) after therapy with evinacumab versus placebo * To measure the number of AP episodes per patient * To assess the safety and tolerability of evinacumab * To assess the potential immunogenicity of evinacumab * To assess the concentrations of total evinacumab and total angiopoietin-like 3 (ANGPTL3)
Interventions
DRUGevinacumab
Intravenous infusion every 4 weeks (Q4W)
OTHERPlacebo
Intravenous infusion Q4W
Sponsors
Regeneron Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. Adults without FCS due to LPL loss of function mutations 2. Documented history of 1 HTG-associated AP episode within 24 months of screening 3. Fasting serum TG value \>880 mg/dL (10 mmol/L) or \>500 mg/dL (5.6mmol/L) determined during the screening period as described in the protocol 4. Stable dose of lipid-lowering therapy (≥8 weeks) and willingness to maintain a stable regimen throughout the study 5. Body mass index ≥18.0 and ≤45.0 kg/m2 6. Compliance with a stable diet and exercise regimen at screening and willingness to continue the diet through the end of the study Key
Exclusion criteria
1. Hospitalization for AP within 4 weeks of screening 2. Known genetic FCS defined as homozygous or compound heterozygous LoF mutations in LPL as defined in the protocol 3. Symptomatic gallstone disease within 6 months prior to screening as defined in the protocol 4. Use of any medication or nutraceutical known to alter serum lipids which has not been part of a stable therapeutic regimen for at least 8 weeks, and there are no plans to change the regimen during the study 5. Presence of any clinically significant, uncontrolled endocrine disease known to influence serum lipids as defined in the protocol 6. Has received a COVID-19 vaccination within 1-week of planned start medication or for which the planned COVID-19 vaccination would not be completed 1-week prior to start of the study Note: Other protocol-defined Inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With at Least One Positively Adjudicated Acute Pancreatitis (AP) Episode | Baseline to 52 weeks | All AP (Acute Pancreatitis) episodes occurred post-study drug treatment. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change in Fasting Triglycerides (TGs) - (From Baseline to Week 52) | Baseline to week 52 | — |
| Percent Change in Total Cholesterol (TC) - (Baseline to Week 52) | Baseline to week 52 | — |
| Percent Change in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) - (From Baseline to Week 52) | Baseline to week 52 | — |
| Percent Change in Apolipoprotein B48 (ApoB48) From Baseline to Week 52 | Baseline to week 52 | — |
| Percent Change in Apolipoprotein B100 (ApoB100) Levels From Baseline to Week 52 | Baseline to week 52 | — |
| Percent Change in Nuclear Magnetic Resonance (NMR)-Determined Particle Size and Number From Baseline to Week 52 | Baseline to week 52 | — |
| Number of Independently Adjudicated Positive Episodes of Acute Pancreatitis (AP) Per Participant | Up to 52 weeks | — |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs | From start of study drug administration up to off drug follow-up (up to Week 72) | — |
| Percent Change in Apolipoprotein C3 (ApoC3) From Baseline to Week 52 | Baseline to week 52 | Apolipoproteins transport lipids through the body by binding with fat and cholesterol to form lipoproteins. ApoC3 was a component of very-low-density lipoproteins (VLDL), high-density lipoprotein (HDL), and triglyceride-rich chylomicrons and regulates lipid metabolism. Percent change in ApoC3 from Baseline to Week 52 was reported. |
| Number of Participants With Clinically Significant Changes From Baseline in Laboratory Parameters | From start of study drug administration up to off drug follow-up (up to Week 72) | Clinical laboratory parameters included biochemistry, hematology and urinalysis. The number of participants with clinically significant changes from baseline in laboratory parameters were reported. Clinical significance was determined by the investigator. |
| Number of Participants With Positive Treatment-emergent Anti-Drug Antibodies (ADA) | Baseline to Week 52 | Treatment-Emergent ADA was defined as any positive post baseline assay response when baseline results were negative or missing. Treatment-Emergent ADA responses were further classified as: Persistent (a positive result in the ADA assay detected in at least 2 consecutive post baseline samples separated by at least a 16-week post baseline period \[based on\] nominal sampling time\], with no ADA-negative results in-between, regardless of any missing samples); Indeterminate (a positive result in the ADA assay at the last collection time point only, regardless of any missing samples); Transient (not persistent/indeterminate, regardless of any missing samples). Number of participants with positive treatment-emergent ADA response during Week 52 were reported. |
| Number of Participants With Positive Neutralizing Antibodies (NAb) | Baseline to Week 52 | NAb positive was defined as presence of at least one positive nAb sample. |
| Percent Change in Fasting High-density Lipoprotein Cholesterol (HDL-C) From Baseline to Week 52 | Baseline to Week 52 | Percent Change in fasting HDL-C from Baseline to Week 52 was reported. |
| Percent Change in Fasting Low Density Lipoprotein (LDL-C) From Baseline to Week 52 | Baseline to Week 52 | LDL-C levels were determined in beta-quantification with ultracentrifugation method. Percent change in fasting LDL-C from Baseline to Week 52 was reported. |
| Concentration of Total Evinacumab in Serum | Pre-dose and End of Infusion (EOI) at Weeks 0, 4, 8, 12, 16, 20, 24, 32, 36, 40, 44, and 48; Pre-dose at Weeks 28 and 52 | Concentration of total evinacumab in serum by time at Pre-dose and End of Infusion were analyzed and reported. |
| Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Pre-dose and End of Infusion (EOI) at Weeks 0, 4, 8, 12, 16, 20, 24, 32, 36, 40, 44, and 48; Pre-dose at Weeks 28 and 52 | Concentration of total ANGPTL3 in serum by time were analyzed and reported. |
| Number of Participants With TEAEs Based on Severity | From start of study drug administration up to off drug follow-up (up to Week 72) | AE was defined any untoward medical occurrence in a participant administered with a study drug, which does not necessarily had a causal relationship with this treatment. TEAEs are defined as AEs that developed or worsened during the treatment period. Severity of TEAEs was graded according to the following scale: Mild: Does not interfere in a significant manner with the participants normal functioning level, Moderate: Produces some impairment of functioning but is not hazardous to health and Severe: Produces significant impairment of functioning or incapacitation and is a definite hazard to the participants health. |
Countries
Canada, Germany, Netherlands, United States
Participant flow
Pre-assignment details
A total of 40 participants were screened, of which 21 participants met the eligibility criteria and were randomized in 1:1 to receive either evinacumab or matched placebo. The sponsor terminated the study early due to enrollment issues.
Participants by arm
| Arm | Count |
|---|---|
| Placebo Randomized 1:1 | 10 |
| Evinacumab Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks. | 11 |
| Total | 21 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Lost to Follow-up | 3 | 2 |
| Overall Study | Subject Decision | 0 | 4 |
Baseline characteristics
| Characteristic | Evinacumab | Total | Placebo |
|---|---|---|---|
| Age, Continuous | 45.3 Years STANDARD_DEVIATION 9.46 | 45.6 Years STANDARD_DEVIATION 12.21 | 46.0 Years STANDARD_DEVIATION 15.22 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 3 Participants | 5 Participants | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 8 Participants | 16 Participants | 8 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 2 Participants | 1 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 9 Participants | 18 Participants | 9 Participants |
| Sex: Female, Male Female | 2 Participants | 5 Participants | 3 Participants |
| Sex: Female, Male Male | 9 Participants | 16 Participants | 7 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 10 | 0 / 11 |
| other Total, other adverse events | 8 / 10 | 8 / 11 |
| serious Total, serious adverse events | 4 / 10 | 5 / 11 |
Outcome results
Primary
Percentage of Participants With at Least One Positively Adjudicated Acute Pancreatitis (AP) Episode
All AP (Acute Pancreatitis) episodes occurred post-study drug treatment.
Time frame: Baseline to 52 weeks
Population: The Intent-to-treat Analysis Set (ITT) population was defined as all randomized participants who received 1 dose or part of a dose of study drug.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Placebo | Percentage of Participants With at Least One Positively Adjudicated Acute Pancreatitis (AP) Episode | 10.0 Percentage of Participants |
| Evinacumab | Percentage of Participants With at Least One Positively Adjudicated Acute Pancreatitis (AP) Episode | 27.3 Percentage of Participants |
Secondary
Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum
Concentration of total ANGPTL3 in serum by time were analyzed and reported.
Time frame: Pre-dose and End of Infusion (EOI) at Weeks 0, 4, 8, 12, 16, 20, 24, 32, 36, 40, 44, and 48; Pre-dose at Weeks 28 and 52
Population: The total target analysis set included all treated participants who received any study drug and who had at least 1 non-missing post-dose total ANGPTL3 measurement following the first dose of study drug. Here, number analyzed signifies to participants evaluable for this outcome at given timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 0: Pre-Dose | 0.0930 mg/L | Standard Deviation 0.0225 |
| Placebo | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 0: EOI (End of Infusion) | 0.0826 mg/L | Standard Deviation 0.018 |
| Placebo | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 4: Pre-Dose | 0.0876 mg/L | Standard Deviation 0.0257 |
| Placebo | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 4: EOI (End of Infusion) | 0.0828 mg/L | Standard Deviation 0.026 |
| Placebo | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 8: Pre-Dose | 0.0914 mg/L | Standard Deviation 0.0287 |
| Placebo | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 8: EOI (End of Infusion) | 0.0765 mg/L | Standard Deviation 0.028 |
| Placebo | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 12: Pre-Dose | 0.0767 mg/L | Standard Deviation 0.0121 |
| Placebo | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 12: EOI (End of Infusion) | 0.0699 mg/L | Standard Deviation 0.0106 |
| Placebo | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 16: Pre-Dose | 0.0812 mg/L | Standard Deviation 0.0277 |
| Placebo | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 16: EOI (End of Infusion) | 0.0730 mg/L | Standard Deviation 0.0164 |
| Placebo | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 28: Pre-Dose | 0.0807 mg/L | Standard Deviation 0.0103 |
| Evinacumab | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 0: Pre-Dose | 0.0907 mg/L | Standard Deviation 0.0272 |
| Evinacumab | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 8: EOI (End of Infusion) | 0.298 mg/L | Standard Deviation 0.0831 |
| Evinacumab | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 0: EOI (End of Infusion) | 0.211 mg/L | Standard Deviation 0.0362 |
| Evinacumab | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 36: Pre-Dose | 0.162 mg/L | Standard Deviation 0.107 |
| Evinacumab | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 4: Pre-Dose | 0.248 mg/L | Standard Deviation 0.105 |
| Evinacumab | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 12: Pre-Dose | 0.281 mg/L | Standard Deviation 0.0851 |
| Evinacumab | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 4: EOI (End of Infusion) | 0.306 mg/L | Standard Deviation 0.0992 |
| Evinacumab | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 16: Pre-Dose | 0.230 mg/L | Standard Deviation 0.056 |
| Evinacumab | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 8: Pre-Dose | 0.243 mg/L | Standard Deviation 0.0811 |
| Evinacumab | Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum | Week 12: EOI (End of Infusion) | 0.322 mg/L | Standard Deviation 0.0672 |
Secondary
Concentration of Total Evinacumab in Serum
Concentration of total evinacumab in serum by time at Pre-dose and End of Infusion were analyzed and reported.
Time frame: Pre-dose and End of Infusion (EOI) at Weeks 0, 4, 8, 12, 16, 20, 24, 32, 36, 40, 44, and 48; Pre-dose at Weeks 28 and 52
Population: The PK analysis set included all randomized participants who received any study drug and for each participant who has at least 1 non-missing post-baseline measurement of evinacumab concentration. Here, number analyzed signifies to participants evaluable for this outcome at given timepoints. Data was planned to be reported only for evinacumab arm.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Concentration of Total Evinacumab in Serum | Week 0: Pre-dose | 1.39 milligrams per liter (mg/L) | Standard Deviation 4.4 |
| Placebo | Concentration of Total Evinacumab in Serum | Week 0: EOI (End of Infusion) | 573 milligrams per liter (mg/L) | Standard Deviation 95.7 |
| Placebo | Concentration of Total Evinacumab in Serum | Week 4: Pre-dose | 97.6 milligrams per liter (mg/L) | Standard Deviation 37.4 |
| Placebo | Concentration of Total Evinacumab in Serum | Week 4: EOI (End of Infusion) | 638 milligrams per liter (mg/L) | Standard Deviation 286 |
| Placebo | Concentration of Total Evinacumab in Serum | Week 8: Pre-dose | 133 milligrams per liter (mg/L) | Standard Deviation 88.6 |
| Placebo | Concentration of Total Evinacumab in Serum | Week 8: EOI (End of Infusion) | 724 milligrams per liter (mg/L) | Standard Deviation 123 |
| Placebo | Concentration of Total Evinacumab in Serum | Week 12: Pre-dose | 158 milligrams per liter (mg/L) | Standard Deviation 81.1 |
| Placebo | Concentration of Total Evinacumab in Serum | Week 12: EOI (End of Infusion) | 749 milligrams per liter (mg/L) | Standard Deviation 215 |
| Placebo | Concentration of Total Evinacumab in Serum | Week 16: Pre-dose | 201 milligrams per liter (mg/L) | Standard Deviation 68.7 |
| Placebo | Concentration of Total Evinacumab in Serum | Week 36: Pre-dose | 99.1 milligrams per liter (mg/L) | Standard Deviation 171 |
Secondary
Number of Independently Adjudicated Positive Episodes of Acute Pancreatitis (AP) Per Participant
Time frame: Up to 52 weeks
Population: The ITT population is defined as all randomized participants who received 1 dose or part of a dose of study drug. Participants in the ITT population will be analyzed according to the treatment group allocated by randomization
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Number of Independently Adjudicated Positive Episodes of Acute Pancreatitis (AP) Per Participant | 0.1 Number of Episodes | Standard Deviation 0.32 |
| Evinacumab | Number of Independently Adjudicated Positive Episodes of Acute Pancreatitis (AP) Per Participant | 0.4 Number of Episodes | Standard Deviation 0.67 |
Secondary
Number of Participants With Clinically Significant Changes From Baseline in Laboratory Parameters
Clinical laboratory parameters included biochemistry, hematology and urinalysis. The number of participants with clinically significant changes from baseline in laboratory parameters were reported. Clinical significance was determined by the investigator.
Time frame: From start of study drug administration up to off drug follow-up (up to Week 72)
Population: The SAF included all participants who received at least 1 dose or part of a dose of double-blind study drug.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Placebo | Number of Participants With Clinically Significant Changes From Baseline in Laboratory Parameters | 0 Participants |
| Evinacumab | Number of Participants With Clinically Significant Changes From Baseline in Laboratory Parameters | 0 Participants |
Secondary
Number of Participants With Positive Neutralizing Antibodies (NAb)
NAb positive was defined as presence of at least one positive nAb sample.
Time frame: Baseline to Week 52
Population: Data was not collected and analyzed for this outcome measure as the sponsor terminated the study early due to enrollment issues.
Secondary
Number of Participants With Positive Treatment-emergent Anti-Drug Antibodies (ADA)
Treatment-Emergent ADA was defined as any positive post baseline assay response when baseline results were negative or missing. Treatment-Emergent ADA responses were further classified as: Persistent (a positive result in the ADA assay detected in at least 2 consecutive post baseline samples separated by at least a 16-week post baseline period \[based on\] nominal sampling time\], with no ADA-negative results in-between, regardless of any missing samples); Indeterminate (a positive result in the ADA assay at the last collection time point only, regardless of any missing samples); Transient (not persistent/indeterminate, regardless of any missing samples). Number of participants with positive treatment-emergent ADA response during Week 52 were reported.
Time frame: Baseline to Week 52
Population: The ADA analysis set included all treated participants who received any amount of study drug (active or placebo) and had at least one non-missing ADA result following the first dose of the study drug or placebo. Here, Overall number of participants analyzed refers to participants who were evaluable for this outcome measure.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Placebo | Number of Participants With Positive Treatment-emergent Anti-Drug Antibodies (ADA) | Participants with Treatment-Emergent Response: Persistent | 0 Participants |
| Placebo | Number of Participants With Positive Treatment-emergent Anti-Drug Antibodies (ADA) | Participants with Treatment-Emergent Response: Transient | 0 Participants |
| Placebo | Number of Participants With Positive Treatment-emergent Anti-Drug Antibodies (ADA) | Participants with Treatment-Emergent Response: Indeterminate | 1 Participants |
| Evinacumab | Number of Participants With Positive Treatment-emergent Anti-Drug Antibodies (ADA) | Participants with Treatment-Emergent Response: Persistent | 0 Participants |
| Evinacumab | Number of Participants With Positive Treatment-emergent Anti-Drug Antibodies (ADA) | Participants with Treatment-Emergent Response: Transient | 0 Participants |
| Evinacumab | Number of Participants With Positive Treatment-emergent Anti-Drug Antibodies (ADA) | Participants with Treatment-Emergent Response: Indeterminate | 0 Participants |
Secondary
Number of Participants With TEAEs Based on Severity
AE was defined any untoward medical occurrence in a participant administered with a study drug, which does not necessarily had a causal relationship with this treatment. TEAEs are defined as AEs that developed or worsened during the treatment period. Severity of TEAEs was graded according to the following scale: Mild: Does not interfere in a significant manner with the participants normal functioning level, Moderate: Produces some impairment of functioning but is not hazardous to health and Severe: Produces significant impairment of functioning or incapacitation and is a definite hazard to the participants health.
Time frame: From start of study drug administration up to off drug follow-up (up to Week 72)
Population: The SAF included all participants who received at least 1 dose or part of a dose of double-blind study drug. Here, Overall number of participants analyzed refers to participants who were evaluable for this outcome measure.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Placebo | Number of Participants With TEAEs Based on Severity | Participants with Mild TEAEs | 3 Participants |
| Placebo | Number of Participants With TEAEs Based on Severity | Participants with Moderate TEAEs | 3 Participants |
| Placebo | Number of Participants With TEAEs Based on Severity | Participants with Severe TEAEs | 4 Participants |
| Evinacumab | Number of Participants With TEAEs Based on Severity | Participants with Mild TEAEs | 3 Participants |
| Evinacumab | Number of Participants With TEAEs Based on Severity | Participants with Moderate TEAEs | 1 Participants |
| Evinacumab | Number of Participants With TEAEs Based on Severity | Participants with Severe TEAEs | 3 Participants |
Secondary
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
Time frame: From start of study drug administration up to off drug follow-up (up to Week 72)
Population: The SAF included all participants who received at least 1 dose or part of a dose of double-blind study drug.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Placebo | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs | Participants with TEAEs | 10 Participants |
| Placebo | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs | Participants with Serious TEAEs | 4 Participants |
| Evinacumab | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs | Participants with TEAEs | 7 Participants |
| Evinacumab | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs | Participants with Serious TEAEs | 5 Participants |
Secondary
Percent Change in Apolipoprotein B100 (ApoB100) Levels From Baseline to Week 52
Time frame: Baseline to week 52
Population: The ITT population is defined as all randomized participants who received 1 dose or part of a dose of study drug. Participants in the ITT population will be analyzed according to the treatment group allocated by randomization. Participants who were evaluable for this outcome measure and 0 in the overall analyzed field signifies that none of the participants were available for the assessment at the specified timepoint for placebo arm.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Evinacumab | Percent Change in Apolipoprotein B100 (ApoB100) Levels From Baseline to Week 52 | 225.14 Percent Change |
Secondary
Percent Change in Apolipoprotein B48 (ApoB48) From Baseline to Week 52
Time frame: Baseline to week 52
Population: The ITT population is defined as all randomized participants who received 1 dose or part of a dose of study drug. Participants in the ITT population will be analyzed according to the treatment group allocated by randomization. Participants who were evaluable for this outcome measure and 0 in the overall analyzed field signifies that none of the participants were available for the assessment at the specified timepoint for placebo arm.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Evinacumab | Percent Change in Apolipoprotein B48 (ApoB48) From Baseline to Week 52 | -92.09 Percent Change |
Secondary
Percent Change in Apolipoprotein C3 (ApoC3) From Baseline to Week 52
Apolipoproteins transport lipids through the body by binding with fat and cholesterol to form lipoproteins. ApoC3 was a component of very-low-density lipoproteins (VLDL), high-density lipoprotein (HDL), and triglyceride-rich chylomicrons and regulates lipid metabolism. Percent change in ApoC3 from Baseline to Week 52 was reported.
Time frame: Baseline to week 52
Population: ITT population was defined as all randomized participants who received 1 dose or part of a dose of study drug. Here, Overall number of participants analyzed refers to participants who were evaluable for this outcome measure and 0 in the overall analyzed field signifies that none of the participants were available for the assessment at the specified timepoint for placebo arm.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Evinacumab | Percent Change in Apolipoprotein C3 (ApoC3) From Baseline to Week 52 | -73.78 Percent Change |
Secondary
Percent Change in Fasting High-density Lipoprotein Cholesterol (HDL-C) From Baseline to Week 52
Percent Change in fasting HDL-C from Baseline to Week 52 was reported.
Time frame: Baseline to Week 52
Population: The SAF included all participants who received at least 1 dose or part of a dose of double-blind study drug. Here, Overall number of participants analyzed refers to participants who were evaluable for this outcome measure and 0 in the overall analyzed field signifies that none of the participants were available for the assessment at the specified timepoint for placebo arm.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Evinacumab | Percent Change in Fasting High-density Lipoprotein Cholesterol (HDL-C) From Baseline to Week 52 | 52.94 Percent change |
Secondary
Percent Change in Fasting Low Density Lipoprotein (LDL-C) From Baseline to Week 52
LDL-C levels were determined in beta-quantification with ultracentrifugation method. Percent change in fasting LDL-C from Baseline to Week 52 was reported.
Time frame: Baseline to Week 52
Population: The SAF included all participants who received at least 1 dose or part of a dose of double-blind study drug. Here, Overall number of participants analyzed refers to participants who were evaluable for this outcome measure and 0 in the overall analyzed field signifies that none of the participants were available for the assessment at the specified timepoint for placebo arm.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Evinacumab | Percent Change in Fasting Low Density Lipoprotein (LDL-C) From Baseline to Week 52 | 695.00 Percent Change |
Secondary
Percent Change in Fasting Triglycerides (TGs) - (From Baseline to Week 52)
Time frame: Baseline to week 52
Population: The ITT population is defined as all randomized participants who received 1 dose or part of a dose of study drug. Participants in the ITT population will be analyzed according to the treatment group allocated by randomization. Participants who were evaluable for this outcome measure and 0 in the overall analyzed field signifies that none of the participants were available for the assessment at the specified timepoint for placebo arm.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Evinacumab | Percent Change in Fasting Triglycerides (TGs) - (From Baseline to Week 52) | -92.88 Percent Change |
Secondary
Percent Change in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) - (From Baseline to Week 52)
Time frame: Baseline to week 52
Population: ITT population was defined as all randomized participants who received 1 dose or part of a dose of study drug. Here, Overall number of participants analyzed refers to participants who were evaluable for this outcome measure and 0 in the overall analyzed field signifies that none of the participants were available for the assessment at the specified timepoint for placebo arm.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Evinacumab | Percent Change in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) - (From Baseline to Week 52) | -60.12 Percent Change |
Secondary
Percent Change in Nuclear Magnetic Resonance (NMR)-Determined Particle Size and Number From Baseline to Week 52
Time frame: Baseline to week 52
Population: Data was not collected and analyzed for this outcome measure as the sponsor terminated the study early due to enrollment issues.
Secondary
Percent Change in Total Cholesterol (TC) - (Baseline to Week 52)
Time frame: Baseline to week 52
Population: ITT population was defined as all randomized participants who received 1 dose or part of a dose of study drug. Here, Overall number of participants analyzed refers to participants who were evaluable for this outcome measure and 0 in the overall analyzed field signifies that none of the participants were available for the assessment at the specified timepoint for placebo arm.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Evinacumab | Percent Change in Total Cholesterol (TC) - (Baseline to Week 52) | -57.62 Percent Change |