Homozygous Familial Hypercholesterolemia
Conditions
Keywords
HoFH
Brief summary
The primary objective for Part A of the study is to assess the pharmacokinetics (PK) of evinacumab in pediatric patients with homozygous familial hypercholesterolemia (HoFH). The primary objective for Part B of the study is to demonstrate a reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab in pediatric (5 to 11 years of age) patients with HoFH. The secondary objective for Part A of the study is to evaluate the safety and tolerability of evinacumab administered intravenous (IV) in pediatric patients with HoFH. The secondary objectives for Part B of the study are: * To evaluate the effect of evinacumab on other lipid parameters (ie, apolipoprotein B (Apo B), non-high-density lipoprotein cholesterol (non-HDL-C), total cholesterol (TC), lipoprotein a \[Lp(a)\]) in pediatric patients with HoFH * To evaluate the safety and tolerability of evinacumab administered IV in pediatric patients with HoFH * To assess the PK of evinacumab in pediatric patients with HoFH * To assess the immunogenicity of evinacumab in pediatric patients with HoFH over time * To evaluate patient efficacy by mutation status
Detailed description
Part A is Phase 1b Part B is Phase 3
Interventions
DRUGEvinacumab
Part A: Single IV dose Part B & C: IV dose Q4W
Sponsors
Regeneron Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
5 Years to 11 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. Diagnosis of functional HoFH by either genetic or clinical criteria as defined in the protocol 2. LDL-C \>130 mg/dL at the screening visit 3. Body weight ≥15 kg 4. Receiving stable maximally tolerated therapy\*at the screening visit \*Maximally tolerated therapy could include a daily statin. 5. Willing and able to comply with clinic visits and study-related procedures 6. Parent(s) or legal guardian(s) must provide the signed informed consent form (ICF). Patients ≥5 years of age (or above age determined by the IRB/EC and in accordance with the local regulations and requirements) must also provide informed assent forms (IAFs) to enroll in the study, and sign and date a separate IAF or ICF signed by the parent(s)/legal guardian(s) (as appropriate based on local regulations and requirements) Key
Exclusion criteria
1. Background pharmacologic LMT, nutraceuticals or over-the-counter (OTC) therapies known to affect lipids, at a dose/regimen that has not been stable for at least 4 weeks (8 weeks for PCSK9 inhibitors) before the screening visit and patient is unwilling to enter the run-in period 2. For patients entering Part A, unable to temporarily discontinue apheresis from the baseline visit through the week 4 visit 3. Receiving lipid apheresis, a setting (if applicable) and schedule that has not been stable for approximately 8 weeks before the screening visit or an apheresis schedule that is not anticipated to be stable over the duration of the treatment period (48 weeks). 4. Plasmapheresis within 8 weeks of the screening visit, or plans to undergo plasmapheresis during Part A or Part B 5. Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins 6. Newly diagnosed (within 3 months prior to randomization visit) diabetes mellitus or poorly controlled diabetes as defined in the protocol Note: Other protocol-defined criteria apply
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Part A: Maximum Observed Serum Concentration (Cmax) of Evinacumab | At day 12 | Cmax was obtained directly from the plasma concentration versus time curve. |
| Part A: Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) of Evinacumab | Up to Week 12 | AUClast was defined as area under the serum concentration time-curve from zero to the last measured concentration. |
| Part A: Terminal Half-Life (t1/2) of Evinacumab | Up to week 12 | 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. |
| Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 | Baseline to Week 24 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Part B: Percentage of Participants With ≥50 Percent (%) Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 | Week 24 | Percentage of participants who achieved reduction in calculated LDL-C ≥ 50% at Week 24 was reported. |
| 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 | Baseline to Week 24 | 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. |
| Part B: Percent Change in Lipoprotein A (Lp[a]) From Baseline to Week 24 | Baseline to Week 24 | Percent change in Lp(a) from baseline to Week 24 was reported. |
| Part B: Absolute Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline at Week 24 | Baseline, Week 24 | Absolute change in LDL-C from baseline at Week 24 was reported |
| Part A and Part B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Part A: up to Week 24; Part B: up to Week 48 | 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. |
| Part B: Maximum Serum Concentration at Steady State (Cmax,ss) of Evinacumab | Post-dose up to day 169 | Maximum serum concentration (Cmax,ss) steady state following drug administration. |
| Part B: Area Under the Serum Concentration-time Curve at Steady State (AUCtau.ss) of Evinacumab | Post-dose up to day 169 | AUCtau.ss was defined as area under the serum concentration-time curve at steady state of evinacumab |
| Part B: Minimum Serum Concentration at Steady State (Ctrough.ss) of Evinacumab | Post-dose up to day 169 | Ctrough.ss was defined as minimum serum concentration at steady state of evinacumab |
| 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 | Baseline to Week 24 | — |
| Part B: Serum Concentration of Total Evinacumab | Pre-dose at Weeks 0, 4, 8, 12; End of infusion at Weeks 0.006, 4.006, 8.006, 12.006 and 24 | Serum concentration of total evinacumab was reported. Pre-dose samples at week 0 were assayed and the reported value is based on actual measurement. |
| Part B: Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24 | Baseline to Week 24 | Percent change in Apo B from baseline to Week 24 was reported. |
| Part B: Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24 | Baseline to Week 24 | Percent change in Non-HDL-C from baseline to Week 24 was reported. |
| Part B: Percent Change in Total Cholesterol (TC) From Baseline to Week 24 | Baseline to Week 24 | Percent change in TC from baseline to Week 24 was reported. |
Countries
Australia, Austria, Netherlands, Taiwan, Ukraine, United States
Participant flow
Pre-assignment details
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.
Participants by arm
| Arm | Count |
|---|---|
| Part A: Evinacumab 15mg/Kg IV Participants received single intravenous (IV) infusion of evinacumab at a dose of 15 milligrams per kilogram (mg/kg) on Day 1 in Part A. | 6 |
| 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. | 14 |
| Total | 20 |
Baseline characteristics
| Characteristic | Part B: Evinacumab 15mg/Kg IV Q4W | Total | Part A: Evinacumab 15mg/Kg IV |
|---|---|---|---|
| Age, Continuous | 9.1 years STANDARD_DEVIATION 1.94 | 9 years STANDARD_DEVIATION 1.84 | 8.8 years STANDARD_DEVIATION 1.72 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 1 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 13 Participants | 18 Participants | 5 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 1 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants | 2 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 2 Participants | 2 Participants | 0 Participants |
| Race (NIH/OMB) White | 8 Participants | 14 Participants | 6 Participants |
| Sex: Female, Male Female | 8 Participants | 12 Participants | 4 Participants |
| Sex: Female, Male Male | 6 Participants | 8 Participants | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 6 | 0 / 14 | 0 / 6 | 0 / 14 |
| other Total, other adverse events | 5 / 6 | 11 / 14 | 6 / 6 | 11 / 14 |
| serious Total, serious adverse events | 0 / 6 | 1 / 14 | 0 / 6 | 1 / 14 |
Outcome results
Primary
Part A: Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) of Evinacumab
AUClast was defined as area under the serum concentration time-curve from zero to the last measured concentration.
Time frame: 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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | Part A: Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) of Evinacumab | 4576 Days*Milligrams per Liter (day*mg/L) | Standard Deviation 1568 |
Primary
Part A: Maximum Observed Serum Concentration (Cmax) of Evinacumab
Cmax was obtained directly from the plasma concentration versus time curve.
Time frame: 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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | Part A: Maximum Observed Serum Concentration (Cmax) of Evinacumab | 238 Milligrams per Liter (mg/L) | Standard Deviation 90.8 |
Primary
Part A: Terminal Half-Life (t1/2) of Evinacumab
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.
Time frame: 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.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | Part A: Terminal Half-Life (t1/2) of Evinacumab | 7.69 Days |
Primary
Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24
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.
Time frame: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 | -48.3 Percentage of Change |
Secondary
Part A and Part B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
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.
Time frame: 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.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | Part A and Part B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | 5 Participants |
| Part B: Evinacumab 15mg/Kg IV Q4W | Part A and Part B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | 10 Participants |
Secondary
Part B: Absolute Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline at Week 24
Absolute change in LDL-C from baseline at Week 24 was reported
Time frame: 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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | 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 |
Secondary
Part B: Area Under the Serum Concentration-time Curve at Steady State (AUCtau.ss) of Evinacumab
AUCtau.ss was defined as area under the serum concentration-time curve at steady state of evinacumab
Time frame: 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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | Part B: Area Under the Serum Concentration-time Curve at Steady State (AUCtau.ss) of Evinacumab | 7019 Day*Milligrams per Liter (Day*mg/L) | Standard Deviation 2561 |
Secondary
Part B: Maximum Serum Concentration at Steady State (Cmax,ss) of Evinacumab
Maximum serum concentration (Cmax,ss) steady state following drug administration.
Time frame: 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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | Part B: Maximum Serum Concentration at Steady State (Cmax,ss) of Evinacumab | 428.9 Milligrams per Liter (mg/L) | Standard Deviation 113.7 |
Secondary
Part B: Minimum Serum Concentration at Steady State (Ctrough.ss) of Evinacumab
Ctrough.ss was defined as minimum serum concentration at steady state of evinacumab
Time frame: 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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | Part B: Minimum Serum Concentration at Steady State (Ctrough.ss) of Evinacumab | 171.8 Milligrams per Liter (mg/L) | Standard Deviation 79.5 |
Secondary
Part B: Percentage of Participants With ≥50 Percent (%) Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24
Percentage of participants who achieved reduction in calculated LDL-C ≥ 50% at Week 24 was reported.
Time frame: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | 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
Part B: Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24
Percent change in Apo B from baseline to Week 24 was reported.
Time frame: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | Part B: Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24 | -41.3 Percentage of Change |
Secondary
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
Time frame: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | 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 A: Evinacumab 15mg/Kg IV | 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 A: Evinacumab 15mg/Kg IV | 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 | — |
| Part A: Evinacumab 15mg/Kg IV | 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 |
Secondary
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
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.
Time frame: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | 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 A: Evinacumab 15mg/Kg IV | 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
Part B: Percent Change in Lipoprotein A (Lp[a]) From Baseline to Week 24
Percent change in Lp(a) from baseline to Week 24 was reported.
Time frame: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | Part B: Percent Change in Lipoprotein A (Lp[a]) From Baseline to Week 24 | -37.3 Percentage of Change |
Secondary
Part B: Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24
Percent change in Non-HDL-C from baseline to Week 24 was reported.
Time frame: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | Part B: Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24 | -48.9 Percentage of Change |
Secondary
Part B: Percent Change in Total Cholesterol (TC) From Baseline to Week 24
Percent change in TC from baseline to Week 24 was reported.
Time frame: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | Part B: Percent Change in Total Cholesterol (TC) From Baseline to Week 24 | -49.1 Percentage of Change |
Secondary
Part B: Serum Concentration of Total Evinacumab
Serum concentration of total evinacumab was reported. Pre-dose samples at week 0 were assayed and the reported value is based on actual measurement.
Time frame: 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.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Part A: Evinacumab 15mg/Kg IV | Part B: Serum Concentration of Total Evinacumab | Week 0 | 0 Milligrams per Liter (mg/L) | Standard Deviation 0 |
| Part A: Evinacumab 15mg/Kg IV | Part B: Serum Concentration of Total Evinacumab | Week 0.006 | 256 Milligrams per Liter (mg/L) | Standard Deviation 58 |
| Part A: Evinacumab 15mg/Kg IV | Part B: Serum Concentration of Total Evinacumab | Week 4 | 62.6 Milligrams per Liter (mg/L) | Standard Deviation 22.6 |
| Part A: Evinacumab 15mg/Kg IV | Part B: Serum Concentration of Total Evinacumab | Week 4.006 | 293 Milligrams per Liter (mg/L) | Standard Deviation 92.3 |
| Part A: Evinacumab 15mg/Kg IV | Part B: Serum Concentration of Total Evinacumab | Week 8 | 98.8 Milligrams per Liter (mg/L) | Standard Deviation 37.7 |
| Part A: Evinacumab 15mg/Kg IV | Part B: Serum Concentration of Total Evinacumab | Week 8.006 | 356 Milligrams per Liter (mg/L) | Standard Deviation 76 |
| Part A: Evinacumab 15mg/Kg IV | Part B: Serum Concentration of Total Evinacumab | Week 12 | 120 Milligrams per Liter (mg/L) | Standard Deviation 46.5 |
| Part A: Evinacumab 15mg/Kg IV | Part B: Serum Concentration of Total Evinacumab | Week 12.006 | 363 Milligrams per Liter (mg/L) | Standard Deviation 82.1 |
| Part A: Evinacumab 15mg/Kg IV | Part B: Serum Concentration of Total Evinacumab | Week 24 | 140 Milligrams per Liter (mg/L) | Standard Deviation 92.5 |