Trial Outcomes & Findings for A Phase 3 Study of ARO-APOC3 / VSA001 / SAR449124 (Plozasiran) in Chinese Adults With Familial Chylomicronemia Syndrome (NCT NCT05902598)

Last Updated: 2026-02-27

Results Overview

Blood samples for lipid parameters were collected at the specified time points. Fasting serum TG at Month 10 was defined as geometric mean of 2 measurements taken during Month 10, or the other non-missing measurement if any one of the two measurements was missing during Month 10. Analysis was performed on the basis of the handling strategy for intercurrent events, missing fasting serum was imputed mainly based on the Pattern Mixture Models (PMM). Descriptive statistics were calculated based on non-imputed data.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

37 participants

Primary outcome timeframe

Baseline to Month 10

Results posted on

2026-02-27

Participant Flow

A total of 63 participants were screened from 10-July-2023 to 19-Jan-2024, of which 26 participants were screen failures mainly due to selection criteria not met.

A total of 37 participants were randomized and treated in this study. Participants were randomized in 1:1:1 ratio to receive Plozasiran 25 mg dose group, Plozasiran 50 mg dose group and matching placebo group.

Participant milestones

Participant milestones
Measure	Plozasiran 25 mg Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Plozasiran 50 mg Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Placebo Matching placebo administered every 3 months for 12 months
Overall Study STARTED	12	12	13
Overall Study COMPLETED	10	11	11
Overall Study NOT COMPLETED	2	1	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Plozasiran 25 mg Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Plozasiran 50 mg Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Placebo Matching placebo administered every 3 months for 12 months
Overall Study Withdrawal by Subject	1	0	0
Overall Study Lost to Follow-up	0	1	0
Overall Study Poor compliance and unable to receive treatment and attend visits as scheduled	1	0	0
Overall Study Other reason	0	0	1
Overall Study Subjects were transfered to the extension phase after unblinding due to an acute pancreatitis event	0	0	1

Baseline Characteristics

A Phase 3 Study of ARO-APOC3 / VSA001 / SAR449124 (Plozasiran) in Chinese Adults With Familial Chylomicronemia Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Plozasiran 25 mg n=12 Participants Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Plozasiran 50 mg n=12 Participants Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Placebo n=13 Participants Matching placebo administered every 3 months for 12 months	Total n=37 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=24 Participants	0 Participants n=20 Participants	0 Participants n=40 Participants	0 Participants n=565 Participants
Age, Categorical Between 18 and 65 years	12 Participants n=24 Participants	12 Participants n=20 Participants	13 Participants n=40 Participants	37 Participants n=565 Participants
Age, Categorical >=65 years	0 Participants n=24 Participants	0 Participants n=20 Participants	0 Participants n=40 Participants	0 Participants n=565 Participants
Sex: Female, Male Female	4 Participants n=24 Participants	2 Participants n=20 Participants	3 Participants n=40 Participants	9 Participants n=565 Participants
Sex: Female, Male Male	8 Participants n=24 Participants	10 Participants n=20 Participants	10 Participants n=40 Participants	28 Participants n=565 Participants
Race/Ethnicity, Customized Race · Asian	12 Participants n=24 Participants	12 Participants n=20 Participants	13 Participants n=40 Participants	37 Participants n=565 Participants
Race/Ethnicity, Customized Race · Others	0 Participants n=24 Participants	0 Participants n=20 Participants	0 Participants n=40 Participants	0 Participants n=565 Participants
Race/Ethnicity, Customized Ethnicity · Han	11 Participants n=24 Participants	12 Participants n=20 Participants	12 Participants n=40 Participants	35 Participants n=565 Participants
Race/Ethnicity, Customized Ethnicity · Others	1 Participants n=24 Participants	0 Participants n=20 Participants	1 Participants n=40 Participants	2 Participants n=565 Participants
Region of Enrollment China	12 Participants n=24 Participants	12 Participants n=20 Participants	13 Participants n=40 Participants	37 Participants n=565 Participants
Fasting serum TG (mg/dL)	1786.1 mg/dL STANDARD_DEVIATION 894.04 • n=24 Participants	1640.2 mg/dL STANDARD_DEVIATION 856.15 • n=20 Participants	1250.2 mg/dL STANDARD_DEVIATION 444.26 • n=40 Participants	1550.5 mg/dL STANDARD_DEVIATION 766.64 • n=565 Participants

PRIMARY outcome

Timeframe: Baseline to Month 10

Population: Full analysis set (FAS), All randomized participants were included in FAS. All efficacy analyses were performed using the FAS. Participants were analyzed according to the study treatment assigned at randomization. For descriptive statistics, only participants with data collected for this outcome measure are reported.

Outcome measures

Outcome measures
Measure	Plozasiran 25 mg n=10 Participants Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Plozasiran 50 mg n=11 Participants Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Placebo n=11 Participants Matching placebo administered every 3 months for 12 months
Percent Change From Baseline in Fasting Serum Triglyceride (TG) at Month 10	-85.7 Percent change Interval -97.3 to -43.9	-89.5 Percent change Interval -94.7 to -18.1	38.8 Percent change Interval -56.2 to 136.7

SECONDARY outcome

Timeframe: Baseline to Months 10 and 12 (averaged)

Blood samples for lipid parameters were collected at the specified time points. Fasting serum TG at Month 10 and Month 12 was defined as the geometric mean of the measurements in Month 10 and Month 12 or the non-missing measurement in the other month if measurement in any one of the two months was missing. Analysis was performed on the basis of the handling strategy for intercurrent events, missing fasting serum TG was imputed mainly based on the PMM. Descriptive statistics were calculated based on non-imputed data.

Outcome measures

Outcome measures
Measure	Plozasiran 25 mg n=10 Participants Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Plozasiran 50 mg n=11 Participants Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Placebo n=11 Participants Matching placebo administered every 3 months for 12 months
Percent Change From Baseline in Fasting Serum TG at Months 10 and 12 (Averaged)	-82.1 Percent change Interval -96.8 to -41.5	-86.6 Percent change Interval -93.3 to -26.1	49.5 Percent change Interval -56.4 to 457.7

SECONDARY outcome

Timeframe: Baseline to Month 10

Blood samples for lipid parameters were collected at the specified time points. Baseline of fasting serum APOC 3 was defined as the last non-missing value prior to or on the first dosing date. Analysis was performed on the basis of the handling strategy for intercurrent events, missing values were imputed using the PMM. Descriptive statistics were calculated based on non-imputed data.

Outcome measures

Outcome measures
Measure	Plozasiran 25 mg n=10 Participants Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Plozasiran 50 mg n=10 Participants Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Placebo n=11 Participants Matching placebo administered every 3 months for 12 months
Percent Change From Baseline in Fasting Serum Apolipoprotein C3 (APOC3) at Month 10	-92.55 Percent change Interval -97.57 to -85.7	-91.83 Percent change Interval -99.78 to -84.19	19.91 Percent change Interval -28.35 to 79.48

SECONDARY outcome

Timeframe: Baseline to Month 12

Outcome measures

Outcome measures
Measure	Plozasiran 25 mg n=10 Participants Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Plozasiran 50 mg n=10 Participants Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Placebo n=11 Participants Matching placebo administered every 3 months for 12 months
Percent Change From Baseline in Fasting Serum APOC3 at Month 12	-83.98 Percent change Interval -93.41 to -13.93	-88.16 Percent change Interval -94.83 to -71.13	22.56 Percent change Interval -29.38 to 334.31

SECONDARY outcome

Timeframe: Baseline to Month 10

Population: Full analysis set (FAS), All randomized participants were included in FAS. All efficacy analyses were performed using the FAS. Participants were analyzed according to the study treatment assigned at randomization. Only participants with data collected for this outcome measure are reported.

Blood samples for lipid parameters were collected at the specified time points. Baseline was defined as the last non-missing value prior to or on the first dosing date.

Outcome measures

Outcome measures
Measure	Plozasiran 25 mg n=10 Participants Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Plozasiran 50 mg n=11 Participants Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Placebo n=11 Participants Matching placebo administered every 3 months for 12 months
Percent Change From Baseline in Fasting Serum Non-high Density Lipoprotein Cholesterol (Non-HDL-C) and High Density Lipoprotein Cholesterol (HDL-C) at Month 10 Percent change in fasting serum Non-HDL-C	-37.76 Percent change Interval -63.8 to 37.5	-17.83 Percent change Interval -58.0 to 360.4	40.34 Percent change Interval -3.1 to 218.2
Percent Change From Baseline in Fasting Serum Non-high Density Lipoprotein Cholesterol (Non-HDL-C) and High Density Lipoprotein Cholesterol (HDL-C) at Month 10 Percent change in fasting serum HDL-C	71.62 Percent change Interval 0.0 to 166.1	86.84 Percent change Interval 47.7 to 190.2	-3.51 Percent change Interval -43.1 to 14.3

SECONDARY outcome

Timeframe: Baseline to Month 12

Population: Full analysis set (FAS), All randomized participants were included in FAS. All efficacy analyses were performed using the FAS. Participants were analyzed according to the study treatment assigned at randomization. Only participants with data collected for this outcome measure are reported.

Blood samples for lipid parameters were collected at the specified time points. Non-HDL-C and HDL-C baseline was defined as the last non-missing value prior to or on the first dosing date.

Outcome measures

Outcome measures
Measure	Plozasiran 25 mg n=12 Participants Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Plozasiran 50 mg n=12 Participants Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Placebo n=13 Participants Matching placebo administered every 3 months for 12 months
Percent Change From Baseline in Fasting Serum TG, Non-HDL-C, and HDL-C at Month 12 Percent change in fasting serum Non-HDL-C	-7.21 Percent change Interval -48.6 to 63.3	-29.22 Percent change Interval -61.7 to 315.4	40.67 Percent change Interval -2.3 to 96.7
Percent Change From Baseline in Fasting Serum TG, Non-HDL-C, and HDL-C at Month 12 Percent change in fasting serum HDL-C	51.32 Percent change Interval -23.2 to 130.4	65.33 Percent change Interval 21.5 to 178.4	-11.86 Percent change Interval -93.3 to 40.4
Percent Change From Baseline in Fasting Serum TG, Non-HDL-C, and HDL-C at Month 12 Percent change in fasting serum TG	-71.56 Percent change Interval -96.2 to 59.1	-79.22 Percent change Interval -92.5 to -13.4	79.41 Percent change Interval -62.1 to 1213.9

SECONDARY outcome

Timeframe: 10 Month

Population: Full analysis set (FAS), All randomized participants were included in FAS. All efficacy analyses were performed using the FAS. Participants were analyzed according to the study treatment assigned at randomization. Only participants with data collected for this outcome measure are reported.

Blood samples for lipid parameters were collected at the specified time points.

Outcome measures

Outcome measures
Measure	Plozasiran 25 mg n=10 Participants Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Plozasiran 50 mg n=11 Participants Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Placebo n=11 Participants Matching placebo administered every 3 months for 12 months
Percentage of Participants Achieving Fasting Serum TG of <500 mg/dL at Month 10	90.0 Percentage of participants	81.8 Percentage of participants	9.1 Percentage of participants

SECONDARY outcome

Timeframe: 12 Month

Blood samples for lipid parameters were collected at the specified time points.

Outcome measures

Outcome measures
Measure	Plozasiran 25 mg n=10 Participants Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Plozasiran 50 mg n=11 Participants Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Placebo n=11 Participants Matching placebo administered every 3 months for 12 months
Percentage of Participants Achieving Fasting Serum TG of <500 mg/dL at Month 12	60.0 Percentage of participants	72.7 Percentage of participants	18.2 Percentage of participants

SECONDARY outcome

Timeframe: From baseline up to Month 12, at Months 1,2,3,4,5,6,7,8,9,10,11 and 12

Population: Full analysis set (FAS), All randomized participants were included in FAS. All efficacy analyses were performed using the FAS. Participants were analyzed according to the study treatment assigned at randomization. Only participants with data collected for this outcome measure are reported.

Blood samples for lipid parameters were collected at the specified time points.

Outcome measures

Outcome measures
Measure	Plozasiran 25 mg n=12 Participants Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Plozasiran 50 mg n=12 Participants Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Placebo n=13 Participants Matching placebo administered every 3 months for 12 months
Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Change from baseline in fasting serum TG at month 4	-11.849 mmol/L Interval -35.81 to -6.55	-15.933 mmol/L Interval -27.27 to -7.69	0.372 mmol/L Interval -7.28 to 21.88
Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Change from baseline in fasting serum TG at month 5	-11.804 mmol/L Interval -34.98 to 0.62	-11.967 mmol/L Interval -20.5 to -4.98	-2.778 mmol/L Interval -9.19 to 25.4
Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Change from baseline in fasting serum TG at month 6	-11.434 mmol/L Interval -34.96 to 8.34	-12.921 mmol/L Interval -29.17 to -7.74	-1.008 mmol/L Interval -9.81 to 54.54
Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Change from baseline in fasting serum TG at month 7	-14.784 mmol/L Interval -35.59 to -7.5	-14.279 mmol/L Interval -33.73 to -1.95	-0.809 mmol/L Interval -8.87 to 10.15
Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Change from baseline in fasting serum TG at month 8	-10.856 mmol/L Interval -35.56 to -1.49	-8.815 mmol/L Interval -31.97 to 32.42	-2.684 mmol/L Interval -7.73 to 23.63
Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Change from baseline in fasting serum TG at month 9	-9.684 mmol/L Interval -34.83 to -4.3	-11.134 mmol/L Interval -26.37 to 8.07	-2.325 mmol/L Interval -7.86 to 24.53
Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Change from baseline in fasting serum TG at month 10	-12.124 mmol/L Interval -35.57 to -7.09	-11.667 mmol/L Interval -23.97 to -5.57	3.794 mmol/L Interval -8.4 to 16.34
Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Change from baseline in fasting serum TG at month 11	-10.674 mmol/L Interval -35.22 to 14.64	-14.040 mmol/L Interval -25.62 to -6.41	-0.453 mmol/L Interval -7.87 to 12.34
Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Change from baseline in fasting serum TG at month 12	-10.208 mmol/L Interval -35.19 to 12.17	-13.750 mmol/L Interval -35.5 to -1.37	10.627 mmol/L Interval -12.76 to 102.27
Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Change from baseline in fasting serum TG at month 2	-12.029 mmol/L Interval -34.49 to -6.11	-10.892 mmol/L Interval -18.72 to 0.15	-3.079 mmol/L Interval -16.56 to 59.98
Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Change from baseline in fasting serum TG at month 3	-9.789 mmol/L Interval -35.0 to -2.65	-12.248 mmol/L Interval -27.59 to -0.49	-4.617 mmol/L Interval -14.28 to 12.6
Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Change from baseline in fasting serum TG at month 1	-12.716 mmol/L Interval -33.29 to -4.4	-13.610 mmol/L Interval -24.48 to -7.87	0.568 mmol/L Interval -15.04 to 10.3

SECONDARY outcome

Timeframe: From baseline up to Month 12, at Months 1,2,3,4,5,6,7,8,9,10,11 and 12

Population: Full analysis set (FAS), All randomized participants were included in FAS. All efficacy analyses were performed using the FAS. Participants were analyzed according to the study treatment assigned at randomization. Only participants with data collected for this outcome measure are reported.

Blood samples for lipid parameters were collected at the specified time points.

Outcome measures

Outcome measures
Measure	Plozasiran 25 mg n=12 Participants Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Plozasiran 50 mg n=12 Participants Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Placebo n=13 Participants Matching placebo administered every 3 months for 12 months
Percent Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Percent change from baseline in fasting serum TG at month 1	-87.17 Percent change Interval -94.1 to -37.0	-91.71 Percent change Interval -96.8 to -42.6	3.86 Percent change Interval -73.4 to 55.1
Percent Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Percent change from baseline in fasting serum TG at month 2	-87.31 Percent change Interval -94.3 to -58.3	-81.86 Percent change Interval -95.2 to 0.3	-30.22 Percent change Interval -80.8 to 377.3
Percent Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Percent change from baseline in fasting serum TG at month 3	-84.49 Percent change Interval -95.7 to -10.1	-77.24 Percent change Interval -96.8 to -2.3	-47.54 Percent change Interval -71.6 to 129.0
Percent Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Percent change from baseline in fasting serum TG at month 4	-91.24 Percent change Interval -97.9 to -25.3	-87.47 Percent change Interval -95.5 to -60.9	-4.74 Percent change Interval -63.8 to 126.8
Percent Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Percent change from baseline in fasting serum TG at month 6	-83.97 Percent change Interval -95.6 to 40.5	-75.58 Percent change Interval -96.6 to -56.3	-4.92 Percent change Interval -68.6 to 732.8
Percent Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Percent change from baseline in fasting serum TG at month 7	-89.17 Percent change Interval -97.3 to -77.8	-87.41 Percent change Interval -96.3 to -14.4	-10.27 Percent change Interval -62.0 to 103.9
Percent Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Percent change from baseline in fasting serum TG at month 8	-82.13 Percent change Interval -97.2 to -13.8	-70.49 Percent change Interval -92.9 to 128.1	-27.06 Percent change Interval -54.8 to 191.3
Percent Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Percent change from baseline in fasting serum TG at month 9	-78.53 Percent change Interval -95.7 to -16.3	-82.26 Percent change Interval -93.8 to 40.2	-22.94 Percent change Interval -52.5 to 251.1
Percent Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Percent change from baseline in fasting serum TG at month 10	-85.69 Percent change Interval -97.3 to -43.9	-89.46 Percent change Interval -94.7 to -18.1	38.84 Percent change Interval -56.2 to 136.7
Percent Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Percent change from baseline in fasting serum TG at month 11	-75.00 Percent change Interval -96.3 to 71.1	-86.11 Percent change Interval -95.6 to -30.7	-3.67 Percent change Interval -55.0 to 133.6
Percent Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Percent change from baseline in fasting serum TG at month 12	-71.56 Percent change Interval -96.2 to 59.1	-79.22 Percent change Interval -92.5 to -13.4	79.41 Percent change Interval -62.1 to 1213.9
Percent Change From Baseline at Each Scheduled Assessment in Fasting Serum TG up to Month 12 Percent change from baseline in fasting serum TG at month 5	-89.10 Percent change Interval -95.7 to 6.8	-88.04 Percent change Interval -94.5 to -25.8	-22.44 Percent change Interval -56.5 to 341.3

SECONDARY outcome

Timeframe: From first administration of study treatment (Day 1) through Month 12.

Any AEs and SAEs reported by the investigator during the study that were consistent with acute pancreatitis events were adjudicated by the blinded Data Safety Committee based on the Atlanta Classification for Acute Pancreatitis 2013 for fulfillment of any 2 of the following 3 criteria: 1. Abdominal pain consistent with symptoms of acute pancreatitis (acute episodes of persistent, severe upper abdominal pain often radiating to the back) 2. Serum lipase activity (or amylase activity) ≥3 × upper limit of normal (ULN) 3. Characteristic findings of acute pancreatitis on contrast-enhanced computed tomography (CECT) or magnetic resonance imaging (MRI) or transabdominal ultrasonography

Outcome measures

Outcome measures
Measure	Plozasiran 25 mg n=12 Participants Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Plozasiran 50 mg n=12 Participants Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Placebo n=13 Participants Matching placebo administered every 3 months for 12 months
Number of Participants With Positively Adjudicated Events of Acute Pancreatitis	0 Participants	0 Participants	1 Participants

Adverse Events

Plozasiran 25 mg

Serious events: 1 serious events

Other events: 11 other events

Deaths: 0 deaths

Plozasiran 50 mg

Serious events: 0 serious events

Other events: 10 other events

Deaths: 0 deaths

Placebo

Serious events: 4 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Plozasiran 25 mg n=12 participants at risk Plozasiran 25 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Plozasiran 50 mg n=12 participants at risk Plozasiran 50 mg administered every 3 months for 12 months (randomized period), followed by an open-label period of 12 months	Placebo n=13 participants at risk Matching placebo administered every 3 months for 12 months
Gastrointestinal disorders Abdominal distension	0.00% 0/12 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.	0.00% 0/12 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.	7.7% 1/13 • Number of events 1 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.
Gastrointestinal disorders Pancreatitis acute	0.00% 0/12 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.	0.00% 0/12 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.	7.7% 1/13 • Number of events 1 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.
Gastrointestinal disorders Duodenal ulcer	0.00% 0/12 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.	0.00% 0/12 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.	7.7% 1/13 • Number of events 1 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.
Hepatobiliary disorders Cholecystitis acute	0.00% 0/12 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.	0.00% 0/12 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.	7.7% 1/13 • Number of events 1 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.
Injury, poisoning and procedural complications Foot fracture	8.3% 1/12 • Number of events 1 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.	0.00% 0/12 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.	0.00% 0/13 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.
Renal and urinary disorders Renal hydrocele	8.3% 1/12 • Number of events 1 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.	0.00% 0/12 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.	0.00% 0/13 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.
Renal and urinary disorders Ureterolithiasis	8.3% 1/12 • Number of events 1 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.	0.00% 0/12 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.	0.00% 0/13 • Adverse events (AE) data was collected from first dose of study treatment (Day 1) up to the Month 12. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion. Analysis was performed on the Safety population that included all randomized participants who had received at least 1 dose of the IP. The data collection is ongoing for the Open-Label Extension period and the AE section will be updated after study completion.

Other adverse events

Additional Information

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Sanofi aventis recherche & développement

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Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
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