Trial Outcomes & Findings for Efficacy and Safety of Evinacumab in Adult Patients With Severe Hypertriglyceridemia for the Prevention of Recurrent Acute Pancreatitis (NCT NCT04863014)

NCT ID: NCT04863014

Last Updated: 2024-05-22

Results Overview

All AP (Acute Pancreatitis) episodes occurred post-study drug treatment.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 21 participants
• Primary outcome timeframe: Baseline to 52 weeks
• Results posted on: 2024-05-22

Participant Flow

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.

Participant milestones

Measure	Placebo Randomized 1:1	Evinacumab Participants received evinacumab 20 milligrams per kilogram (mg/kg) IV infusion Q4W starting from Day 1 up to 52 weeks.
Overall Study STARTED	10	11
Overall Study COMPLETED	7	5
Overall Study NOT COMPLETED	3	6

Reasons for withdrawal

Measure	Placebo Randomized 1:1	Evinacumab Participants received evinacumab 20 milligrams per kilogram (mg/kg) IV infusion Q4W starting from Day 1 up to 52 weeks.
Overall Study Lost to Follow-up	3	2
Overall Study Subject Decision	0	4

Baseline Characteristics

Efficacy and Safety of Evinacumab in Adult Patients With Severe Hypertriglyceridemia for the Prevention of Recurrent Acute Pancreatitis

Baseline characteristics by cohort

Measure	Placebo n=10 Participants Randomized 1:1	Evinacumab n=11 Participants Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.	Total n=21 Participants Total of all reporting groups
Age, Continuous	46.0 Years STANDARD_DEVIATION 15.22 • n=5 Participants	45.3 Years STANDARD_DEVIATION 9.46 • n=7 Participants	45.6 Years STANDARD_DEVIATION 12.21 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants	9 Participants n=7 Participants	16 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=5 Participants	8 Participants n=7 Participants	16 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) White	9 Participants n=5 Participants	9 Participants n=7 Participants	18 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

All AP (Acute Pancreatitis) episodes occurred post-study drug treatment.

Outcome measures

Measure	Placebo n=10 Participants Randomized 1:1 Participants received matched-placebo IV infusion Q4W starting from Day 1 up to 52 weeks.	Evinacumab n=11 Participants Randomized 1:1 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Percentage of Participants With at Least One Positively Adjudicated Acute Pancreatitis (AP) Episode	10.0 Percentage of Participants	27.3 Percentage of Participants

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Measure	Placebo Randomized 1:1 Participants received matched-placebo IV infusion Q4W starting from Day 1 up to 52 weeks.	Evinacumab n=1 Participants Randomized 1:1 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Percent Change in Apolipoprotein C3 (ApoC3) From Baseline to Week 52	—	-73.78 Percent Change

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Placebo Randomized 1:1 Participants received matched-placebo IV infusion Q4W starting from Day 1 up to 52 weeks.	Evinacumab n=1 Participants Randomized 1:1 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Percent Change in Fasting Triglycerides (TGs) - (From Baseline to Week 52)	—	-92.88 Percent Change

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Placebo Randomized 1:1 Participants received matched-placebo IV infusion Q4W starting from Day 1 up to 52 weeks.	Evinacumab n=1 Participants Randomized 1:1 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Percent Change in Total Cholesterol (TC) - (Baseline to Week 52)	—	-57.62 Percent Change

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Placebo Randomized 1:1 Participants received matched-placebo IV infusion Q4W starting from Day 1 up to 52 weeks.	Evinacumab n=1 Participants Randomized 1:1 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Percent Change in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) - (From Baseline to Week 52)	—	-60.12 Percent Change

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Placebo Randomized 1:1 Participants received matched-placebo IV infusion Q4W starting from Day 1 up to 52 weeks.	Evinacumab n=1 Participants Randomized 1:1 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Percent Change in Apolipoprotein B48 (ApoB48) From Baseline to Week 52	—	-92.09 Percent Change

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Placebo Randomized 1:1 Participants received matched-placebo IV infusion Q4W starting from Day 1 up to 52 weeks.	Evinacumab n=1 Participants Randomized 1:1 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Percent Change in Apolipoprotein B100 (ApoB100) Levels From Baseline to Week 52	—	225.14 Percent Change

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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

Outcome measures

Measure	Placebo n=10 Participants Randomized 1:1 Participants received matched-placebo IV infusion Q4W starting from Day 1 up to 52 weeks.	Evinacumab n=11 Participants Randomized 1:1 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Number of Independently Adjudicated Positive Episodes of Acute Pancreatitis (AP) Per Participant	0.1 Number of Episodes Standard Deviation 0.32	0.4 Number of Episodes Standard Deviation 0.67

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	Placebo n=10 Participants Randomized 1:1 Participants received matched-placebo IV infusion Q4W starting from Day 1 up to 52 weeks.	Evinacumab n=11 Participants Randomized 1:1 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs Participants with TEAEs	10 Participants	7 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs Participants with Serious TEAEs	4 Participants	5 Participants

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Measure	Placebo n=10 Participants Randomized 1:1 Participants received matched-placebo IV infusion Q4W starting from Day 1 up to 52 weeks.	Evinacumab n=11 Participants Randomized 1:1 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Number of Participants With TEAEs Based on Severity Participants with Mild TEAEs	3 Participants	3 Participants
Number of Participants With TEAEs Based on Severity Participants with Moderate TEAEs	3 Participants	1 Participants
Number of Participants With TEAEs Based on Severity Participants with Severe TEAEs	4 Participants	3 Participants

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Measure	Placebo n=10 Participants Randomized 1:1 Participants received matched-placebo IV infusion Q4W starting from Day 1 up to 52 weeks.	Evinacumab n=11 Participants Randomized 1:1 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Number of Participants With Clinically Significant Changes From Baseline in Laboratory Parameters	0 Participants	0 Participants

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Measure	Placebo n=8 Participants Randomized 1:1 Participants received matched-placebo IV infusion Q4W starting from Day 1 up to 52 weeks.	Evinacumab n=7 Participants Randomized 1:1 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Number of Participants With Positive Treatment-emergent Anti-Drug Antibodies (ADA) Participants with Treatment-Emergent Response: Persistent	0 Participants	0 Participants
Number of Participants With Positive Treatment-emergent Anti-Drug Antibodies (ADA) Participants with Treatment-Emergent Response: Transient	0 Participants	0 Participants
Number of Participants With Positive Treatment-emergent Anti-Drug Antibodies (ADA) Participants with Treatment-Emergent Response: Indeterminate	1 Participants	0 Participants

SECONDARY outcome

Timeframe: 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.

NAb positive was defined as presence of at least one positive nAb sample.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 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.

Percent Change in fasting HDL-C from Baseline to Week 52 was reported.

Outcome measures

Measure	Placebo Randomized 1:1 Participants received matched-placebo IV infusion Q4W starting from Day 1 up to 52 weeks.	Evinacumab n=1 Participants Randomized 1:1 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Percent Change in Fasting High-density Lipoprotein Cholesterol (HDL-C) From Baseline to Week 52	—	52.94 Percent change

SECONDARY outcome

Timeframe: 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.

LDL-C levels were determined in beta-quantification with ultracentrifugation method. Percent change in fasting LDL-C from Baseline to Week 52 was reported.

Outcome measures

Measure	Placebo Randomized 1:1 Participants received matched-placebo IV infusion Q4W starting from Day 1 up to 52 weeks.	Evinacumab n=1 Participants Randomized 1:1 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Percent Change in Fasting Low Density Lipoprotein (LDL-C) From Baseline to Week 52	—	695.00 Percent Change

SECONDARY outcome

Timeframe: 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.

Concentration of total evinacumab in serum by time at Pre-dose and End of Infusion were analyzed and reported.

Outcome measures

Measure	Placebo n=10 Participants Randomized 1:1 Participants received matched-placebo IV infusion Q4W starting from Day 1 up to 52 weeks.	Evinacumab Randomized 1:1 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Concentration of Total Evinacumab in Serum Week 0: Pre-dose	1.39 milligrams per liter (mg/L) Standard Deviation 4.40	—
Concentration of Total Evinacumab in Serum Week 0: EOI (End of Infusion)	573 milligrams per liter (mg/L) Standard Deviation 95.7	—
Concentration of Total Evinacumab in Serum Week 4: Pre-dose	97.6 milligrams per liter (mg/L) Standard Deviation 37.4	—
Concentration of Total Evinacumab in Serum Week 4: EOI (End of Infusion)	638 milligrams per liter (mg/L) Standard Deviation 286	—
Concentration of Total Evinacumab in Serum Week 8: Pre-dose	133 milligrams per liter (mg/L) Standard Deviation 88.6	—
Concentration of Total Evinacumab in Serum Week 8: EOI (End of Infusion)	724 milligrams per liter (mg/L) Standard Deviation 123	—
Concentration of Total Evinacumab in Serum Week 12: Pre-dose	158 milligrams per liter (mg/L) Standard Deviation 81.1	—
Concentration of Total Evinacumab in Serum Week 12: EOI (End of Infusion)	749 milligrams per liter (mg/L) Standard Deviation 215	—
Concentration of Total Evinacumab in Serum Week 16: Pre-dose	201 milligrams per liter (mg/L) Standard Deviation 68.7	—
Concentration of Total Evinacumab in Serum Week 36: Pre-dose	99.1 milligrams per liter (mg/L) Standard Deviation 171	—

SECONDARY outcome

Timeframe: 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.

Concentration of total ANGPTL3 in serum by time were analyzed and reported.

Outcome measures

Measure	Placebo n=10 Participants Randomized 1:1 Participants received matched-placebo IV infusion Q4W starting from Day 1 up to 52 weeks.	Evinacumab n=10 Participants Randomized 1:1 Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum Week 0: Pre-Dose	0.0930 mg/L Standard Deviation 0.0225	0.0907 mg/L Standard Deviation 0.0272
Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum Week 0: EOI (End of Infusion)	0.0826 mg/L Standard Deviation 0.0180	0.211 mg/L Standard Deviation 0.0362
Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum Week 4: Pre-Dose	0.0876 mg/L Standard Deviation 0.0257	0.248 mg/L Standard Deviation 0.105
Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum Week 4: EOI (End of Infusion)	0.0828 mg/L Standard Deviation 0.0260	0.306 mg/L Standard Deviation 0.0992
Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum Week 8: Pre-Dose	0.0914 mg/L Standard Deviation 0.0287	0.243 mg/L Standard Deviation 0.0811
Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum Week 8: EOI (End of Infusion)	0.0765 mg/L Standard Deviation 0.0280	0.298 mg/L Standard Deviation 0.0831
Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum Week 12: Pre-Dose	0.0767 mg/L Standard Deviation 0.0121	0.281 mg/L Standard Deviation 0.0851
Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum Week 12: EOI (End of Infusion)	0.0699 mg/L Standard Deviation 0.0106	0.322 mg/L Standard Deviation 0.0672
Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum Week 16: Pre-Dose	0.0812 mg/L Standard Deviation 0.0277	0.230 mg/L Standard Deviation 0.0560
Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum Week 16: EOI (End of Infusion)	0.0730 mg/L Standard Deviation 0.0164	—
Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum Week 28: Pre-Dose	0.0807 mg/L Standard Deviation 0.0103	—
Concentration of Total Angiopoietin-like 3 (ANGPTL3) in Serum Week 36: Pre-Dose	—	0.162 mg/L Standard Deviation 0.107

Adverse Events

Placebo

Serious events: 4 serious events

Other events: 8 other events

Deaths: 0 deaths

Evinacumab 20 mg

Serious events: 5 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Measure	Placebo n=10 participants at risk Randomized 1:1	Evinacumab 20 mg n=11 participants at risk Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Gastrointestinal disorders Pancreatitis acute	10.0% 1/10 • Number of events 2 • From start of study drug administration up to off drug follow-up (72 weeks)	18.2% 2/11 • Number of events 3 • From start of study drug administration up to off drug follow-up (72 weeks)
Gastrointestinal disorders Pancreatitis	20.0% 2/10 • Number of events 2 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Gastrointestinal disorders Abdominal pain	10.0% 1/10 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)	0.00% 0/11 • From start of study drug administration up to off drug follow-up (72 weeks)
Cardiac disorders Angina pectoris	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Cardiac disorders Acute myocardial infarction	10.0% 1/10 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)	0.00% 0/11 • From start of study drug administration up to off drug follow-up (72 weeks)
General disorders Pyrexia	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Immune system disorders Anaphylactic reaction	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Infections and infestations Respiratory syncytial virus infection	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)

Other adverse events

Measure	Placebo n=10 participants at risk Randomized 1:1	Evinacumab 20 mg n=11 participants at risk Participants received evinacumab 20 mg/kg IV infusion Q4W starting from Day 1 up to 52 weeks.
Gastrointestinal disorders Abdominal pain	30.0% 3/10 • Number of events 6 • From start of study drug administration up to off drug follow-up (72 weeks)	36.4% 4/11 • Number of events 5 • From start of study drug administration up to off drug follow-up (72 weeks)
Gastrointestinal disorders Duodenitis	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Gastrointestinal disorders Gastrooesophageal reflux disease	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Gastrointestinal disorders Nausea	20.0% 2/10 • Number of events 2 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Gastrointestinal disorders Pancreatic pseudocyst	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Gastrointestinal disorders Vomiting	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Gastrointestinal disorders Abdominal pain upper	20.0% 2/10 • Number of events 2 • From start of study drug administration up to off drug follow-up (72 weeks)	0.00% 0/11 • From start of study drug administration up to off drug follow-up (72 weeks)
Gastrointestinal disorders Diarrhoea	10.0% 1/10 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)	0.00% 0/11 • From start of study drug administration up to off drug follow-up (72 weeks)
Gastrointestinal disorders Dyspepsia	10.0% 1/10 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)	0.00% 0/11 • From start of study drug administration up to off drug follow-up (72 weeks)
Infections and infestations COVID-19	10.0% 1/10 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)	27.3% 3/11 • Number of events 3 • From start of study drug administration up to off drug follow-up (72 weeks)
Infections and infestations Bronchitis	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Infections and infestations Cellulitis	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Infections and infestations Nasopharyngitis	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Infections and infestations Wound infection	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 2 • From start of study drug administration up to off drug follow-up (72 weeks)
Infections and infestations Hand-foot-and-mouth disease	10.0% 1/10 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)	0.00% 0/11 • From start of study drug administration up to off drug follow-up (72 weeks)
Infections and infestations Influenza	10.0% 1/10 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)	0.00% 0/11 • From start of study drug administration up to off drug follow-up (72 weeks)
Blood and lymphatic system disorders Anaemia	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Hepatobiliary disorders Cholelithiasis	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Immune system disorders Drug hypersensitivity	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Injury, poisoning and procedural complications Epicondylitis	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Injury, poisoning and procedural complications Limb injury	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Investigations Pedal pulse decreased	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Nervous system disorders Headache	10.0% 1/10 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Nervous system disorders Dizziness	10.0% 1/10 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)	0.00% 0/11 • From start of study drug administration up to off drug follow-up (72 weeks)
Nervous system disorders Somnolence	10.0% 1/10 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)	0.00% 0/11 • From start of study drug administration up to off drug follow-up (72 weeks)
Psychiatric disorders Insomnia	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Skin and subcutaneous tissue disorders Rash	0.00% 0/10 • From start of study drug administration up to off drug follow-up (72 weeks)	9.1% 1/11 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)
Skin and subcutaneous tissue disorders Urticaria	10.0% 1/10 • Number of events 3 • From start of study drug administration up to off drug follow-up (72 weeks)	0.00% 0/11 • From start of study drug administration up to off drug follow-up (72 weeks)
General disorders Chest discomfort	10.0% 1/10 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)	0.00% 0/11 • From start of study drug administration up to off drug follow-up (72 weeks)
General disorders Fatigue	10.0% 1/10 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)	0.00% 0/11 • From start of study drug administration up to off drug follow-up (72 weeks)
Metabolism and nutrition disorders Food intolerance	10.0% 1/10 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)	0.00% 0/11 • From start of study drug administration up to off drug follow-up (72 weeks)
Metabolism and nutrition disorders Hypoglycaemia	10.0% 1/10 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)	0.00% 0/11 • From start of study drug administration up to off drug follow-up (72 weeks)
Musculoskeletal and connective tissue disorders Arthralgia	10.0% 1/10 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)	0.00% 0/11 • From start of study drug administration up to off drug follow-up (72 weeks)
Vascular disorders Hot flush	10.0% 1/10 • Number of events 1 • From start of study drug administration up to off drug follow-up (72 weeks)	0.00% 0/11 • From start of study drug administration up to off drug follow-up (72 weeks)

Additional Information

Clinical Trials Administrator

Regeneron Pharmaceuticals, Inc

Phone: 8447346643

Email: clinicaltrials@regeneron.com

Results disclosure agreements

• Principal investigator is a sponsor employee The investigator has the right to independently publish study results from the investigator's site after a multi-center publication, or a defined period after the completion of the study by all sites. The investigator must provide the sponsor a copy of any such publication derived from the study for review and comment in advance of any submission, and delay publication, if requested, to allow the Sponsor to preserve its proprietary rights.
• Publication restrictions are in place

Restriction type: OTHER