Trial Outcomes & Findings for Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia (NCT NCT04233918)

NCT ID: NCT04233918

Last Updated: 2024-07-16

Results Overview

Cmax was obtained directly from the plasma concentration versus time curve.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

20 participants

Primary outcome timeframe

At day 12

Results posted on

2024-07-16

Participant Flow

A total of 23 participants were screened to Part A and Part B. 6 participants were enrolled in Part A, 14 participants in Part B. 3 participants were considered screen failures. 2 withdrew consent, 1 due to Other. Participants who enrolled in Part A of study were not eligible to participate in Part B. All 20 participants completed part C.

Participant milestones

Participant milestones
Measure	Part A: Evinacumab 15mg/Kg IV In Part A, Participants received single IV infusion of evinacumab at a dose of 15 mg/kg on Day 1.	Part B: Evinacumab 15mg/Kg IV Q4W In Part B, Participants received IV infusion of evinacumab at a dose of 15 mg/kg Q4W from Day 1 up to Week 24.	Part A to C All participants who completed Part A received IV infusion of evinacumab at a dose of 15 mg/kg Q4W from Day 1 up to Week 48 in Part C.	Part B to C All participants who completed Part B received IV infusion of evinacumab at a dose of 15 mg/kg Q4W from Day 1 up to Week 48 in Part C.
Period 1: Part A and B STARTED	6	14	0	0
Period 1: Part A and B COMPLETED	6	14	0	0
Period 1: Part A and B NOT COMPLETED	0	0	0	0
Part C (Extension Period) STARTED	0	0	6	14
Part C (Extension Period) COMPLETED	0	0	6	14
Part C (Extension Period) NOT COMPLETED	0	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Part A: Evinacumab 15mg/Kg IV n=6 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W n=14 Participants Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.	Total n=20 Participants Total of all reporting groups
Age, Continuous	8.8 years STANDARD_DEVIATION 1.72 • n=5 Participants	9.1 years STANDARD_DEVIATION 1.94 • n=7 Participants	9 years STANDARD_DEVIATION 1.84 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants	8 Participants n=7 Participants	12 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	6 Participants n=7 Participants	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=5 Participants	13 Participants n=7 Participants	18 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	2 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	6 Participants n=5 Participants	8 Participants n=7 Participants	14 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	2 Participants n=7 Participants	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: At day 12

Population: Pharmacokinetic (PK) Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug.

Cmax was obtained directly from the plasma concentration versus time curve.

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=6 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part A: Maximum Observed Serum Concentration (Cmax) of Evinacumab	238 Milligrams per Liter (mg/L) Standard Deviation 90.8	—

PRIMARY outcome

Timeframe: Up to Week 12

Population: PK Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug.

AUClast was defined as area under the serum concentration time-curve from zero to the last measured concentration.

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=6 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part A: Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) of Evinacumab	4576 DaysMilligrams per Liter (daymg/L) Standard Deviation 1568	—

PRIMARY outcome

Timeframe: Up to week 12

Population: PK Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug.

T1/2 was defined as the time required for the plasma concentration of drug to decrease 50 percent in the final stage of its elimination.

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=6 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part A: Terminal Half-Life (t1/2) of Evinacumab	7.69 Days Interval 6.18 to 12.4	—

PRIMARY outcome

Timeframe: Baseline to Week 24

Population: The intent-to-treat (ITT) population included all participants who received at least 1 dose or part of a dose of study drug in Part B.

Percent change was calculated as 100 multiplied by (calculated LDL-C value at Week 24 minus calculated LDL-C value at baseline) divided by calculated LDL-C value at baseline.

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=14 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24	-48.3 Percentage of Change Interval -68.8 to -27.8	—

SECONDARY outcome

Timeframe: Part A: up to Week 24; Part B: up to Week 48

Population: The safety analysis set (SAF) includes all patients in Part A or Part B who received at least 1 dose or part of a dose of study drug in the respective study treatment period.

Any untoward medical occurrence in a participant who received investigational medicinal product (IMP) was considered an AE without regard to possibility of causal relationship with this treatment. A serious adverse event (SAE) was defined as any untoward medical occurrence that resulted in any of the following outcomes: death, life-threatening, required initial or prolonged in-patient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event. TEAE was defined as AE starting/worsening after first intake of study drug. TEAE included participants with both serious and non-serious AEs. 1 participant experienced an AE during part B that was recorded after Part B database lock. This was not reflected in the reported endpoint number of participants of 10.

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=6 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W n=14 Participants Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part A and Part B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	5 Participants	10 Participants

SECONDARY outcome

Timeframe: Baseline to Week 24

Population: ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B.

Percent change in Apo B from baseline to Week 24 was reported.

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=14 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part B: Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24	-41.3 Percentage of Change Interval -58.9 to -23.8	—

SECONDARY outcome

Timeframe: Baseline to Week 24

Population: ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B.

Percent change in Non-HDL-C from baseline to Week 24 was reported.

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=14 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part B: Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24	-48.9 Percentage of Change Interval -68.1 to -29.7	—

SECONDARY outcome

Timeframe: Baseline to Week 24

Population: ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B.

Percent change in TC from baseline to Week 24 was reported.

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=14 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part B: Percent Change in Total Cholesterol (TC) From Baseline to Week 24	-49.1 Percentage of Change Interval -64.9 to -33.2	—

SECONDARY outcome

Timeframe: Week 24

Population: ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B.

Percentage of participants who achieved reduction in calculated LDL-C ≥ 50% at Week 24 was reported.

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=14 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part B: Percentage of Participants With ≥50 Percent (%) Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24	78.6 Percentage of Participants	—

SECONDARY outcome

Timeframe: Baseline to Week 24

Population: ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure and "Number Analyzed" signifies those participants who were evaluable at specified category.

Participants with HoFH was classified based on the phenotype of the Low-density lipoprotein receptor (LDLR) mutation(s), ranging from defective mutations (where the LDLR retains some LDL-binding functionality) to null or negative mutations where no functioning LDLR was expressed. Participants who have LDLR activity \<15% are considered null and participants whose LDLR activity was impaired but \>15% are LDLR defective. Percent change in calculated LDL-C from baseline to Week 24 in participants who have negative/negative and null/null mutations was reported.

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=4 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 in Participants Who Have Negative/Negative and Null/Null Mutations Negative/negative mutations	-67.7 Percentage of Change Standard Error 6.5	—
Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 in Participants Who Have Negative/Negative and Null/Null Mutations Null/Null mutations	-57.2 Percentage of Change	—

SECONDARY outcome

Timeframe: Baseline to Week 24

Population: ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B.

Percent change in Lp(a) from baseline to Week 24 was reported.

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=14 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part B: Percent Change in Lipoprotein A (Lp[a]) From Baseline to Week 24	-37.3 Percentage of Change Interval -42.2 to -32.3	—

SECONDARY outcome

Timeframe: Baseline, Week 24

Population: ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B.

Absolute change in LDL-C from baseline at Week 24 was reported

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=14 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part B: Absolute Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline at Week 24	-131.9 Milligrams per Deciliter (mg/dL) Standard Error 30.0	—

SECONDARY outcome

Timeframe: Pre-dose at Weeks 0, 4, 8, 12; End of infusion at Weeks 0.006, 4.006, 8.006, 12.006 and 24

Population: PK Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug. Here, "Number Analyzed" signifies those participants who were evaluable for this outcome measure at specified timepoints.

Serum concentration of total evinacumab was reported. Pre-dose samples at week 0 were assayed and the reported value is based on actual measurement.

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=14 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part B: Serum Concentration of Total Evinacumab Week 0	0 Milligrams per Liter (mg/L) Standard Deviation 0	—
Part B: Serum Concentration of Total Evinacumab Week 0.006	256 Milligrams per Liter (mg/L) Standard Deviation 58.0	—
Part B: Serum Concentration of Total Evinacumab Week 4	62.6 Milligrams per Liter (mg/L) Standard Deviation 22.6	—
Part B: Serum Concentration of Total Evinacumab Week 4.006	293 Milligrams per Liter (mg/L) Standard Deviation 92.3	—
Part B: Serum Concentration of Total Evinacumab Week 8	98.8 Milligrams per Liter (mg/L) Standard Deviation 37.7	—
Part B: Serum Concentration of Total Evinacumab Week 8.006	356 Milligrams per Liter (mg/L) Standard Deviation 76	—
Part B: Serum Concentration of Total Evinacumab Week 12	120 Milligrams per Liter (mg/L) Standard Deviation 46.5	—
Part B: Serum Concentration of Total Evinacumab Week 12.006	363 Milligrams per Liter (mg/L) Standard Deviation 82.1	—
Part B: Serum Concentration of Total Evinacumab Week 24	140 Milligrams per Liter (mg/L) Standard Deviation 92.5	—

SECONDARY outcome

Timeframe: Post-dose up to day 169

Population: PK Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug.

Maximum serum concentration (Cmax,ss) steady state following drug administration.

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=14 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part B: Maximum Serum Concentration at Steady State (Cmax,ss) of Evinacumab	428.9 Milligrams per Liter (mg/L) Standard Deviation 113.7	—

SECONDARY outcome

Timeframe: Post-dose up to day 169

Population: PK Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug.

AUCtau.ss was defined as area under the serum concentration-time curve at steady state of evinacumab

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=14 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part B: Area Under the Serum Concentration-time Curve at Steady State (AUCtau.ss) of Evinacumab	7019 DayMilligrams per Liter (Daymg/L) Standard Deviation 2561	—

SECONDARY outcome

Timeframe: Post-dose up to day 169

Population: PK Analysis Set included all participants who received any study drug and who had at least 1 non-missing result for concentration of evinacumab following the first dose of study drug.

Ctrough.ss was defined as minimum serum concentration at steady state of evinacumab

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=14 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part B: Minimum Serum Concentration at Steady State (Ctrough.ss) of Evinacumab	171.8 Milligrams per Liter (mg/L) Standard Deviation 79.5	—

SECONDARY outcome

Timeframe: Baseline to Week 24

Population: ITT population included all participants who received at least 1 dose or part of a dose of study drug in Part B.

Outcome measures

Outcome measures
Measure	Part A: Evinacumab 15mg/Kg IV n=14 Participants Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B: Evinacumab 15mg/Kg IV Q4W Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.
Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 in Participants Who Have by Null/Null vs. Non-null/Null and Negative/Negative vs.Non-negative/Negative Mutations Negative/Negative	-67.7 Percentage of Change Standard Error 6.5	—
Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 in Participants Who Have by Null/Null vs. Non-null/Null and Negative/Negative vs.Non-negative/Negative Mutations Non-Negative/Negative	-43.0 Percentage of Change Standard Error 12.8	—
Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 in Participants Who Have by Null/Null vs. Non-null/Null and Negative/Negative vs.Non-negative/Negative Mutations Null/Null	-57.2 Percentage of Change Standard Error NA Standard Error cannot be calculated with only 1 participant	—
Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 in Participants Who Have by Null/Null vs. Non-null/Null and Negative/Negative vs.Non-negative/Negative Mutations Non-Null/Null	-47.6 Percentage of Change Standard Error 11.3	—

Adverse Events

Part A Evinacumab 15mg

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Part B Evinacumab 15mg

Serious events: 1 serious events

Other events: 11 other events

Deaths: 0 deaths

Part A-C Evinacumab 15mg

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Part B-C Evinacumab 15mg

Serious events: 1 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Part A Evinacumab 15mg n=6 participants at risk Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A.	Part B Evinacumab 15mg n=14 participants at risk Participants received IV infusion of evinacumab at a dose of 15 mg/kg every four weeks (Q4W) from Day 1 up to Week 24 in Part B.	Part A-C Evinacumab 15mg n=6 participants at risk All participants who completed Part A received IV infusion of evinacumab at a dose of 15 mg/kg Q4W from Day 1 up to Week 48 in Part C.	Part B-C Evinacumab 15mg n=14 participants at risk All participants who completed Part B received IV infusion of evinacumab at a dose of 15 mg/kg Q4W from Day 1 up to Week 48 in Part C.
Infections and infestations Tonsillitis	0.00% 0/6 • From first dose up to week 96 (24 weeks in Part A/B + 48 weeks of treatment in Part C + 24 weeks of follow-up) Part A - up to week 24 Part B - up to Week 48 or up to the day before the first dose in Part C for participants entering Part C; 1 participant experienced an AE during part B that was recorded after Part B database lock. Part C - up to a 48-week treatment period and 24-week follow-up	7.1% 1/14 • Number of events 1 • From first dose up to week 96 (24 weeks in Part A/B + 48 weeks of treatment in Part C + 24 weeks of follow-up) Part A - up to week 24 Part B - up to Week 48 or up to the day before the first dose in Part C for participants entering Part C; 1 participant experienced an AE during part B that was recorded after Part B database lock. Part C - up to a 48-week treatment period and 24-week follow-up	0.00% 0/6 • From first dose up to week 96 (24 weeks in Part A/B + 48 weeks of treatment in Part C + 24 weeks of follow-up) Part A - up to week 24 Part B - up to Week 48 or up to the day before the first dose in Part C for participants entering Part C; 1 participant experienced an AE during part B that was recorded after Part B database lock. Part C - up to a 48-week treatment period and 24-week follow-up	0.00% 0/14 • From first dose up to week 96 (24 weeks in Part A/B + 48 weeks of treatment in Part C + 24 weeks of follow-up) Part A - up to week 24 Part B - up to Week 48 or up to the day before the first dose in Part C for participants entering Part C; 1 participant experienced an AE during part B that was recorded after Part B database lock. Part C - up to a 48-week treatment period and 24-week follow-up
Cardiac disorders Aortic valve stenosis	0.00% 0/6 • From first dose up to week 96 (24 weeks in Part A/B + 48 weeks of treatment in Part C + 24 weeks of follow-up) Part A - up to week 24 Part B - up to Week 48 or up to the day before the first dose in Part C for participants entering Part C; 1 participant experienced an AE during part B that was recorded after Part B database lock. Part C - up to a 48-week treatment period and 24-week follow-up	0.00% 0/14 • From first dose up to week 96 (24 weeks in Part A/B + 48 weeks of treatment in Part C + 24 weeks of follow-up) Part A - up to week 24 Part B - up to Week 48 or up to the day before the first dose in Part C for participants entering Part C; 1 participant experienced an AE during part B that was recorded after Part B database lock. Part C - up to a 48-week treatment period and 24-week follow-up	0.00% 0/6 • From first dose up to week 96 (24 weeks in Part A/B + 48 weeks of treatment in Part C + 24 weeks of follow-up) Part A - up to week 24 Part B - up to Week 48 or up to the day before the first dose in Part C for participants entering Part C; 1 participant experienced an AE during part B that was recorded after Part B database lock. Part C - up to a 48-week treatment period and 24-week follow-up	7.1% 1/14 • Number of events 1 • From first dose up to week 96 (24 weeks in Part A/B + 48 weeks of treatment in Part C + 24 weeks of follow-up) Part A - up to week 24 Part B - up to Week 48 or up to the day before the first dose in Part C for participants entering Part C; 1 participant experienced an AE during part B that was recorded after Part B database lock. Part C - up to a 48-week treatment period and 24-week follow-up

Other adverse events

Additional Information

Clinical Trials Administrator

Regeneron Pharmaceuticals, Inc

Phone: 844-734-6643

Email: [email protected]

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