Familial Hypercholesterolemia
Conditions
Keywords
Hypercholesterolemia, Elevated Cholesterol, High Cholesterol, PCSK9 mutations, Severe Familial Hypercholesterolemia, evolocumab, Repatha, Heterozygous Familial Hypercholesterolemia, Homozygous Familial Hypercholesterolemia, Pediatric, Paediatric
Brief summary
The main purpose of this study is to describe the safety and tolerability of 80 weeks of subcutaneous (SC) evolocumab when added to standard of care in children 10 to 17 years of age with familial hypercholesterolemia.
Interventions
BIOLOGICALEvolocumab
Administered by subcutaneous injection
Sponsors
Amgen
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
10 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
Heterozygous Familial Hypercholesterolemia (HeFH): -Completed Study 20120123 (NCT02392559) while still on assigned investigational product and did not experience a treatment-related serious adverse event Homozygous Familial Hypercholesterolemia (HoFH): * Male or female, ≥ 10 to ≤ 17 years of age at time of enrollment * Diagnosis of HoFH * On a low-fat diet and receiving background lipid-lowering therapy * Lipid-lowering therapy unchanged for ≥ 4 weeks prior to LDL-C screening; fibrates must be stable for at least 6 weeks prior to screening. * Fasting LDL-C at screening ≥ 130 mg/dL (3.4 mmol/L) * Fasting triglycerides ≤ 400 mg/dL (4.5 mmol/L)
Exclusion criteria
-Currently receiving treatment in another investigational device or drug study, or less than 30 days since ending treatment on another investigational device or drug study(s); except Study 20120123 HoFH: * Moderate to severe renal dysfunction * Active liver disease or hepatic dysfunction, * Creatine kinase \> 3 times the upper limit of normal (ULN) at screening
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | From first dose of evolocumab in this study up to and including 30 days after the last dose or up to the end of study date, whichever was earlier; up to 80 weeks. | An adverse event is defined as any untoward medical occurrence in a clinical trial participant, not necessarily having a causal relationship with study treatment. A serious AE is as an AE that met at least 1 of the following criteria: * fatal; * life threatening; * required in-patient hospitalization or prolongation of existing hospitalization; * resulted in persistent or significant disability/incapacity; * congenital anomaly/birth defect; * other medically important serious event. AEs were graded for severity using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0: Grade 1: Mild; asymptomatic or mild symptoms; Grade 2: Moderate; minimal, local or noninvasive intervention indicated; Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; Grade 4: Life-threatening consequences; urgent intervention indicated; Grade 5: Death related to AE. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HoFH Participants | Baseline and week 80 | For HoFH participants baseline was defined as the baseline value in this study (20120124). |
| Percent Change From Baseline to Week 80 in Non-HDL-C in HeFH Participants | Baseline and week 80 | For HeFH participants baseline was defined as the baseline value of the parent study 20120123. |
| Percent Change From Baseline to Week 80 in Non-HDL-C in HoFH Participants | Baseline and week 80 | For HoFH participants baseline was defined as the baseline value in this study (20120124). |
| Percent Change From Baseline to Week 80 in Apolipoprotein B in HeFH Participants | Baseline and week 80 | For HeFH participants baseline was defined as the baseline value in the parent study 20120123. |
| Percent Change From Baseline to Week 80 in Apolipoprotein B in HoFH Participants | Baseline and week 80 | For HoFH participants baseline was defined as the baseline value in this study (20120124). |
| Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HeFH Participants | Baseline and week 80 | For HeFH participants baseline was defined as the baseline value in the parent study 20120123. |
| Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HoFH Participants | Baseline and week 80 | For HoFH participants baseline was defined as the baseline value in this study (20120124). |
| Percent Change From Baseline to Week 80 in Apolipoprotein B / Apolipoprotein A1 Ratio in HeFH Participants | Baseline and week 80 | For HeFH participants baseline was defined as the baseline value in the parent study 20120123. |
| Percent Change From Baseline to Week 80 in Apolipoprotein B/Apolipoprotein A1 Ratio in HoFH Participants | Baseline and week 80 | For HoFH participants baseline was defined as the baseline value in this study (20120124). |
| Change From Baseline to Week 80 in LDL-C in HeFH Participants | Baseline and week 80 | For HeFH participants baseline was defined as the baseline value of the parent study 20120123. |
| Change From Baseline to Week 80 in LDL-C in HoFH Participants | Baseline and week 80 | For HoFH participants baseline was defined as the baseline value in this study (20120124). |
| Change From Baseline to Week 80 in Estradiol Levels | Baseline and week 80 | For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124). |
| Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HeFH Participants | Baseline and week 80 | For HeFH participants baseline was defined as the baseline value of the parent study 20120123. |
| Change From Baseline to Week 80 in Follicle Stimulating Hormone (FSH) Levels | Baseline and week 80 | For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124). |
| Change From Baseline to Week 80 in Luteinizing Hormone (LH) Levels | Baseline and week 80 | For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124). |
| Change From Baseline to Week 80 in Adenocorticotropic Hormone (ACTH) Levels | Baseline and week 80 | For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124). |
| Change From Baseline to Week 80 in Dehydroepiandrosterone Sulfate (DHEA-S) Levels | Baseline and week 80 | For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124). |
| Change From Baseline to Week 80 in Cortisol Levels | Baseline and week 80 | For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124). |
| Number of Participants With Liver Function Test Abnormalities at Week 80 | Week 80 | Liver function tests included alanine aminotransferase (ALT) levels, aspartate aminotransferase (AST) levels and total bilirubin levels. |
| Number of Participants With Abnormalities in Levels of Creatine Kinase (CK) at Week 80 | Week 80 | The number of participants with levels of creatine kinase greater than 5 times the upper limit of normal (ULN) and greater than 10 times the ULN, measured by the central laboratory. |
| Change From Baseline to Week 80 in Carotid Intima-media Thickness (cIMT) | Baseline and week 80 | Carotid intima-media thickness measures the thickness of the intima and media, the inner two layers of the carotid artery, and is used to determine the extent of plaque buildup in the walls of the arteries (atherosclerosis) supplying blood to the head. CIMT was measured by ultrasonography and analyzed at a core laboratory. The largest values measured in the left common carotid artery (LCCA) and the right common carotid artery (RCCA) are averaged in this analysis. |
| Change From Baseline in Height at Weeks 24, 48, and 80 | Baseline and weeks 24, 48, and 80 | — |
| Change From Baseline in Weight at Weeks 24, 48, and 80 | Baseline and weeks 24, 48, and 80 | — |
| Number of Participants With Change in Tanner Staging From Baseline to Week 80 | Baseline and week 80 | Pubertal growth and sexual maturity was assessed separately for males and females using the 5 Tanner stages where stage 1 = prepubertal and stage 5 = mature. The number of participants with any change in Tanner Stage from baseline is reported. |
| Change From Baseline to Week 80 in Testosterone Levels | Baseline and week 80 | For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124). |
Countries
Australia, Austria, Belgium, Brazil, Canada, Colombia, Czechia, Greece, Hungary, Italy, Malaysia, Netherlands, Norway, Poland, Portugal, Russia, Slovenia, South Africa, Spain, Switzerland, Turkey (Türkiye), United Kingdom, United States
Participant flow
Recruitment details
This study was conducted at 46 centers in 23 countries (Australia, Austria, Belgium, Brazil, Canada, Colombia, Czech Republic, Greece, Hungary, Italy, Malaysia, Netherlands, Norway, Poland, Portugal, Russia, Slovenia, South Africa, Spain, Switzerland, Turkey, United Kingdom, and United States of America).
Pre-assignment details
This study enrolled participants with heterozygous familial hypercholesterolemia (HeFH) who had completed the parent study 20120123 (NCT02392559) without experiencing a treatment-related serious adverse event, and children 10 to 17 years of age with a diagnosis of homozygous familial hypercholesterolemia (HoFH).
Participants by arm
| Arm | Count |
|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM Participants with HeFH who had received placebo in the parent study received 420 mg evolocumab administered by subcutaneous injection every 4 weeks (QM) for up to 80 weeks. | 49 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM Participants with HeFH who had received evolocumab in the parent study received 420 mg evolocumab administered by subcutaneous injection every 4 weeks for up to 80 weeks. | 101 |
| HoFH: Evolocumab 420 mg QM Participants with HoFH received 420 mg evolocumab administered by subcutaneous injection every 4 weeks for up to 80 weeks. | 13 |
| Total | 163 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Lost to Follow-up | 0 | 0 | 1 |
| Overall Study | Withdrawal by Subject | 1 | 3 | 1 |
Baseline characteristics
| Characteristic | HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | HoFH: Evolocumab 420 mg QM | Total |
|---|---|---|---|---|
| Age, Continuous | 13.8 years STANDARD_DEVIATION 2.5 | 14.2 years STANDARD_DEVIATION 2.4 | 12.4 years STANDARD_DEVIATION 2 | 14.0 years STANDARD_DEVIATION 2.5 |
| Age, Customized 12 - 17 years | 37 Participants | 76 Participants | 7 Participants | 120 Participants |
| Age, Customized 18 - 64 years | 1 Participants | 7 Participants | 0 Participants | 8 Participants |
| Age, Customized 2 - 11 years | 11 Participants | 18 Participants | 6 Participants | 35 Participants |
| Apolipoprotein B (ApoB) Concentration | 119.1 mg/dL STANDARD_DEVIATION 28.1 | 123.1 mg/dL STANDARD_DEVIATION 27.4 | 250.1 mg/dL STANDARD_DEVIATION 84.9 | 131.5 mg/dL STANDARD_DEVIATION 48.6 |
| Apolipoprotein B/Apolipoprotein A1 Ratio | 0.943 ratio STANDARD_DEVIATION 0.265 | 0.972 ratio STANDARD_DEVIATION 0.306 | 2.388 ratio STANDARD_DEVIATION 1.036 | 1.070 ratio STANDARD_DEVIATION 0.545 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 7 Participants | 6 Participants | 0 Participants | 13 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 42 Participants | 95 Participants | 13 Participants | 150 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Low-density Lipoprotein Cholesterol (LDL-C) Concentration | 184.0 mg/dL STANDARD_DEVIATION 48.3 | 184.4 mg/dL STANDARD_DEVIATION 45.2 | 426.0 mg/dL STANDARD_DEVIATION 166.4 | 202.2 mg/dL STANDARD_DEVIATION 88.8 |
| Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) Concentration | 201.0 mg/dL STANDARD_DEVIATION 49.3 | 203.4 mg/dL STANDARD_DEVIATION 47.5 | 443.7 mg/dL STANDARD_DEVIATION 170.8 | 220.5 mg/dL STANDARD_DEVIATION 90.2 |
| Race/Ethnicity, Customized American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Asian | 0 Participants | 2 Participants | 2 Participants | 4 Participants |
| Race/Ethnicity, Customized Black or African American | 0 Participants | 2 Participants | 0 Participants | 2 Participants |
| Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Other | 9 Participants | 11 Participants | 2 Participants | 22 Participants |
| Race/Ethnicity, Customized White | 40 Participants | 86 Participants | 9 Participants | 135 Participants |
| Region Asia Pacific | 0 Participants | 6 Participants | 6 Participants | 12 Participants |
| Region Europe | 33 Participants | 65 Participants | 7 Participants | 105 Participants |
| Region Latin America | 8 Participants | 18 Participants | 0 Participants | 26 Participants |
| Region North America | 8 Participants | 12 Participants | 0 Participants | 20 Participants |
| Sex: Female, Male Female | 24 Participants | 59 Participants | 2 Participants | 85 Participants |
| Sex: Female, Male Male | 25 Participants | 42 Participants | 11 Participants | 78 Participants |
| Total Cholesterol/High-density Lipoprotein Cholesterol (HDL-C) Ratio | 5.546 ratio STANDARD_DEVIATION 1.541 | 5.716 ratio STANDARD_DEVIATION 1.809 | 14.707 ratio STANDARD_DEVIATION 7.891 | 6.331 ratio STANDARD_DEVIATION 3.557 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 49 | 0 / 101 | 0 / 13 |
| other Total, other adverse events | 27 / 49 | 45 / 101 | 7 / 12 |
| serious Total, serious adverse events | 2 / 49 | 2 / 101 | 2 / 12 |
Outcome results
Primary
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
An adverse event is defined as any untoward medical occurrence in a clinical trial participant, not necessarily having a causal relationship with study treatment. A serious AE is as an AE that met at least 1 of the following criteria: * fatal; * life threatening; * required in-patient hospitalization or prolongation of existing hospitalization; * resulted in persistent or significant disability/incapacity; * congenital anomaly/birth defect; * other medically important serious event. AEs were graded for severity using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0: Grade 1: Mild; asymptomatic or mild symptoms; Grade 2: Moderate; minimal, local or noninvasive intervention indicated; Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; Grade 4: Life-threatening consequences; urgent intervention indicated; Grade 5: Death related to AE.
Time frame: From first dose of evolocumab in this study up to and including 30 days after the last dose or up to the end of study date, whichever was earlier; up to 80 weeks.
Population: Full analysis set
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | TEAE ≥ Grade 2 | 25 Participants |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Serious adverse events | 2 Participants |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | TEAE ≥ Grade 4 | 0 Participants |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Any treatment-emergent adverse event (TEAE) | 36 Participants |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Fatal adverse events | 0 Participants |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | TEAE leading to discontinuation of evolocumab | 0 Participants |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | TEAE ≥ Grade 3 | 4 Participants |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | TEAE ≥ Grade 4 | 1 Participants |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Any treatment-emergent adverse event (TEAE) | 69 Participants |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | TEAE ≥ Grade 2 | 56 Participants |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | TEAE ≥ Grade 3 | 2 Participants |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Serious adverse events | 2 Participants |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | TEAE leading to discontinuation of evolocumab | 0 Participants |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Fatal adverse events | 0 Participants |
| HoFH: Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Serious adverse events | 2 Participants |
| HoFH: Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | TEAE ≥ Grade 2 | 5 Participants |
| HoFH: Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Fatal adverse events | 0 Participants |
| HoFH: Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | TEAE leading to discontinuation of evolocumab | 0 Participants |
| HoFH: Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | TEAE ≥ Grade 4 | 0 Participants |
| HoFH: Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | TEAE ≥ Grade 3 | 2 Participants |
| HoFH: Evolocumab 420 mg QM | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Any treatment-emergent adverse event (TEAE) | 7 Participants |
Secondary
Change From Baseline in Height at Weeks 24, 48, and 80
Time frame: Baseline and weeks 24, 48, and 80
Population: Full analysis set with available data at each time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Females: Baseline | 158.1 cm | Standard Error 2.3 |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Females: Change at week 24 | 2.8 cm | Standard Error 0.7 |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Females: Change at week 48 | 4.2 cm | Standard Error 1 |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Females: Change at week 80 | 4.0 cm | Standard Error 1.7 |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Males: Baseline | 158.2 cm | Standard Error 3 |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Males: Change at week 24 | 3.4 cm | Standard Error 0.5 |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Males: Change at week 48 | 6.2 cm | Standard Error 0.8 |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Males: Change at week 80 | 9.3 cm | Standard Error 1.5 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Females: Change at week 48 | 2.8 cm | Standard Error 0.5 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Males: Change at week 48 | 6.2 cm | Standard Error 0.7 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Females: Change at week 80 | 3.4 cm | Standard Error 0.7 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Males: Baseline | 163.7 cm | Standard Error 1.9 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Males: Change at week 24 | 3.8 cm | Standard Error 0.5 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Females: Baseline | 157.9 cm | Standard Error 1.3 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Females: Change at week 24 | 2.0 cm | Standard Error 0.4 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Males: Change at week 80 | 9.0 cm | Standard Error 1.1 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Females: Change at week 48 | 1.4 cm | Standard Error 2.4 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Females: Change at week 24 | 1.6 cm | Standard Error 1.1 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Females: Baseline | 149.4 cm | Standard Error 7.1 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Females: Change at week 80 | 2.4 cm | Standard Error 3.9 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Males: Change at week 48 | 5.3 cm | Standard Error 1.2 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Males: Change at week 24 | 3.8 cm | Standard Error 0.8 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Males: Baseline | 158.9 cm | Standard Error 4.8 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline in Height at Weeks 24, 48, and 80 | Males: Change at week 80 | 9.2 cm | Standard Error 1.6 |
Secondary
Change From Baseline in Weight at Weeks 24, 48, and 80
Time frame: Baseline and weeks 24, 48, and 80
Population: Full analysis set with available data at each time point
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Males: Change at week 80 | 10.9 kg | Standard Error 1.6 |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Females: Baseline | 52.8 kg | Standard Error 2.9 |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Females: Change at week 80 | 5.6 kg | Standard Error 1.3 |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Males: Change at week 48 | 6.8 kg | Standard Error 1.3 |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Females: Change at week 24 | 3.3 kg | Standard Error 0.8 |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Males: Baseline | 54.1 kg | Standard Error 3.6 |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Females: Change at week 48 | 4.3 kg | Standard Error 1.1 |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Males: Change at week 24 | 4.4 kg | Standard Error 0.9 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Females: Change at week 24 | 2.3 kg | Standard Error 0.6 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Males: Change at week 24 | 4.6 kg | Standard Error 0.7 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Females: Baseline | 57.0 kg | Standard Error 2 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Males: Change at week 80 | 11.2 kg | Standard Error 1.4 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Males: Change at week 48 | 7.4 kg | Standard Error 1 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Females: Change at week 48 | 3.5 kg | Standard Error 0.7 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Females: Change at week 80 | 5.2 kg | Standard Error 0.8 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Males: Baseline | 61.0 kg | Standard Error 3.2 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Males: Change at week 80 | 10.6 kg | Standard Error 2.3 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Females: Baseline | 42.7 kg | Standard Error 1.3 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Females: Change at week 24 | 3.4 kg | Standard Error 2.1 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Females: Change at week 48 | 4.7 kg | Standard Error 5.3 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Females: Change at week 80 | 5.5 kg | Standard Error 4.2 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Males: Baseline | 51.7 kg | Standard Error 4.9 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Males: Change at week 24 | 4.6 kg | Standard Error 0.9 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline in Weight at Weeks 24, 48, and 80 | Males: Change at week 48 | 7.6 kg | Standard Error 1.2 |
Secondary
Change From Baseline to Week 80 in Adenocorticotropic Hormone (ACTH) Levels
For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in Adenocorticotropic Hormone (ACTH) Levels | 0.78 pmol/L | Standard Error 0.55 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in Adenocorticotropic Hormone (ACTH) Levels | 0.55 pmol/L | Standard Error 0.61 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline to Week 80 in Adenocorticotropic Hormone (ACTH) Levels | -0.75 pmol/L | Standard Error 1.79 |
Secondary
Change From Baseline to Week 80 in Carotid Intima-media Thickness (cIMT)
Carotid intima-media thickness measures the thickness of the intima and media, the inner two layers of the carotid artery, and is used to determine the extent of plaque buildup in the walls of the arteries (atherosclerosis) supplying blood to the head. CIMT was measured by ultrasonography and analyzed at a core laboratory. The largest values measured in the left common carotid artery (LCCA) and the right common carotid artery (RCCA) are averaged in this analysis.
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in Carotid Intima-media Thickness (cIMT) | -0.019 mm | Standard Error 0.007 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in Carotid Intima-media Thickness (cIMT) | -0.012 mm | Standard Error 0.006 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline to Week 80 in Carotid Intima-media Thickness (cIMT) | 0.006 mm | Standard Error 0.032 |
Secondary
Change From Baseline to Week 80 in Cortisol Levels
For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in Cortisol Levels | 29.81 nmol/L | Standard Error 28.34 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in Cortisol Levels | 51.18 nmol/L | Standard Error 25.19 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline to Week 80 in Cortisol Levels | 57.26 nmol/L | Standard Error 56.11 |
Secondary
Change From Baseline to Week 80 in Dehydroepiandrosterone Sulfate (DHEA-S) Levels
For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in Dehydroepiandrosterone Sulfate (DHEA-S) Levels | 1.051 μmol/L | Standard Error 0.222 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in Dehydroepiandrosterone Sulfate (DHEA-S) Levels | 0.956 μmol/L | Standard Error 0.126 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline to Week 80 in Dehydroepiandrosterone Sulfate (DHEA-S) Levels | 0.944 μmol/L | Standard Error 0.247 |
Secondary
Change From Baseline to Week 80 in Estradiol Levels
For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).
Time frame: Baseline and week 80
Population: Full analysis set; female participants with available data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in Estradiol Levels | 131.3 pmol/L | Standard Error 45.3 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in Estradiol Levels | 48.2 pmol/L | Standard Error 58.1 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline to Week 80 in Estradiol Levels | 283.0 pmol/L | Standard Error 130 |
Secondary
Change From Baseline to Week 80 in Follicle Stimulating Hormone (FSH) Levels
For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in Follicle Stimulating Hormone (FSH) Levels | 1.88 IU/L | Standard Error 0.9 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in Follicle Stimulating Hormone (FSH) Levels | 0.60 IU/L | Standard Error 0.37 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline to Week 80 in Follicle Stimulating Hormone (FSH) Levels | 1.18 IU/L | Standard Error 0.35 |
Secondary
Change From Baseline to Week 80 in LDL-C in HeFH Participants
For HeFH participants baseline was defined as the baseline value of the parent study 20120123.
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in LDL-C in HeFH Participants | -67.2 mg/dL | Standard Error 8.2 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in LDL-C in HeFH Participants | -63.1 mg/dL | Standard Error 5.6 |
Secondary
Change From Baseline to Week 80 in LDL-C in HoFH Participants
For HoFH participants baseline was defined as the baseline value in this study (20120124).
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in LDL-C in HoFH Participants | -36.5 mg/dL |
Secondary
Change From Baseline to Week 80 in Luteinizing Hormone (LH) Levels
For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in Luteinizing Hormone (LH) Levels | 2.88 IU/L | Standard Error 1.51 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in Luteinizing Hormone (LH) Levels | 1.04 IU/L | Standard Error 0.95 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline to Week 80 in Luteinizing Hormone (LH) Levels | 1.76 IU/L | Standard Error 0.84 |
Secondary
Change From Baseline to Week 80 in Testosterone Levels
For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).
Time frame: Baseline and week 80
Population: Full analysis set; male participants with available data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in Testosterone Levels | 5.282 nmol/L | Standard Error 1.567 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Change From Baseline to Week 80 in Testosterone Levels | 3.230 nmol/L | Standard Error 1.167 |
| HoFH: Evolocumab 420 mg QM | Change From Baseline to Week 80 in Testosterone Levels | 2.916 nmol/L | Standard Error 0.984 |
Secondary
Number of Participants With Abnormalities in Levels of Creatine Kinase (CK) at Week 80
The number of participants with levels of creatine kinase greater than 5 times the upper limit of normal (ULN) and greater than 10 times the ULN, measured by the central laboratory.
Time frame: Week 80
Population: Full analysis set with available data at week 80
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Number of Participants With Abnormalities in Levels of Creatine Kinase (CK) at Week 80 | CK > 5 x ULN | 0 Participants |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Number of Participants With Abnormalities in Levels of Creatine Kinase (CK) at Week 80 | CK > 10 x ULN | 0 Participants |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Number of Participants With Abnormalities in Levels of Creatine Kinase (CK) at Week 80 | CK > 5 x ULN | 0 Participants |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Number of Participants With Abnormalities in Levels of Creatine Kinase (CK) at Week 80 | CK > 10 x ULN | 0 Participants |
| HoFH: Evolocumab 420 mg QM | Number of Participants With Abnormalities in Levels of Creatine Kinase (CK) at Week 80 | CK > 5 x ULN | 1 Participants |
| HoFH: Evolocumab 420 mg QM | Number of Participants With Abnormalities in Levels of Creatine Kinase (CK) at Week 80 | CK > 10 x ULN | 0 Participants |
Secondary
Number of Participants With Change in Tanner Staging From Baseline to Week 80
Pubertal growth and sexual maturity was assessed separately for males and females using the 5 Tanner stages where stage 1 = prepubertal and stage 5 = mature. The number of participants with any change in Tanner Stage from baseline is reported.
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Number of Participants With Change in Tanner Staging From Baseline to Week 80 | Females: Staging by breast development | 11 Participants |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Number of Participants With Change in Tanner Staging From Baseline to Week 80 | Males: Staging by genital size | 13 Participants |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Number of Participants With Change in Tanner Staging From Baseline to Week 80 | Females: Staging by pubic hair | 11 Participants |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Number of Participants With Change in Tanner Staging From Baseline to Week 80 | Males: Staging by pubic hair | 14 Participants |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Number of Participants With Change in Tanner Staging From Baseline to Week 80 | Females: Staging by breast development | 27 Participants |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Number of Participants With Change in Tanner Staging From Baseline to Week 80 | Males: Staging by pubic hair | 21 Participants |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Number of Participants With Change in Tanner Staging From Baseline to Week 80 | Males: Staging by genital size | 20 Participants |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Number of Participants With Change in Tanner Staging From Baseline to Week 80 | Females: Staging by pubic hair | 26 Participants |
| HoFH: Evolocumab 420 mg QM | Number of Participants With Change in Tanner Staging From Baseline to Week 80 | Females: Staging by pubic hair | 1 Participants |
| HoFH: Evolocumab 420 mg QM | Number of Participants With Change in Tanner Staging From Baseline to Week 80 | Males: Staging by genital size | 6 Participants |
| HoFH: Evolocumab 420 mg QM | Number of Participants With Change in Tanner Staging From Baseline to Week 80 | Males: Staging by pubic hair | 6 Participants |
| HoFH: Evolocumab 420 mg QM | Number of Participants With Change in Tanner Staging From Baseline to Week 80 | Females: Staging by breast development | 1 Participants |
Secondary
Number of Participants With Liver Function Test Abnormalities at Week 80
Liver function tests included alanine aminotransferase (ALT) levels, aspartate aminotransferase (AST) levels and total bilirubin levels.
Time frame: Week 80
Population: Full analysis set with available data at week 80
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Number of Participants With Liver Function Test Abnormalities at Week 80 | ALT or AST > 5 x ULN | 0 Participants |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Number of Participants With Liver Function Test Abnormalities at Week 80 | ALT or AST > 3 x ULN | 0 Participants |
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Number of Participants With Liver Function Test Abnormalities at Week 80 | Total bilirubin > 2 x ULN | 0 Participants |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Number of Participants With Liver Function Test Abnormalities at Week 80 | ALT or AST > 5 x ULN | 0 Participants |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Number of Participants With Liver Function Test Abnormalities at Week 80 | ALT or AST > 3 x ULN | 0 Participants |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Number of Participants With Liver Function Test Abnormalities at Week 80 | Total bilirubin > 2 x ULN | 2 Participants |
| HoFH: Evolocumab 420 mg QM | Number of Participants With Liver Function Test Abnormalities at Week 80 | ALT or AST > 3 x ULN | 0 Participants |
| HoFH: Evolocumab 420 mg QM | Number of Participants With Liver Function Test Abnormalities at Week 80 | Total bilirubin > 2 x ULN | 1 Participants |
| HoFH: Evolocumab 420 mg QM | Number of Participants With Liver Function Test Abnormalities at Week 80 | ALT or AST > 5 x ULN | 0 Participants |
Secondary
Percent Change From Baseline to Week 80 in Apolipoprotein B / Apolipoprotein A1 Ratio in HeFH Participants
For HeFH participants baseline was defined as the baseline value in the parent study 20120123.
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Percent Change From Baseline to Week 80 in Apolipoprotein B / Apolipoprotein A1 Ratio in HeFH Participants | -31.00 percent change | Standard Error 3.66 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Percent Change From Baseline to Week 80 in Apolipoprotein B / Apolipoprotein A1 Ratio in HeFH Participants | -29.89 percent change | Standard Error 2.8 |
Secondary
Percent Change From Baseline to Week 80 in Apolipoprotein B/Apolipoprotein A1 Ratio in HoFH Participants
For HoFH participants baseline was defined as the baseline value in this study (20120124).
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Percent Change From Baseline to Week 80 in Apolipoprotein B/Apolipoprotein A1 Ratio in HoFH Participants | -2.96 percent change |
Secondary
Percent Change From Baseline to Week 80 in Apolipoprotein B in HeFH Participants
For HeFH participants baseline was defined as the baseline value in the parent study 20120123.
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Percent Change From Baseline to Week 80 in Apolipoprotein B in HeFH Participants | -27.10 percent change | Standard Error 3.32 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Percent Change From Baseline to Week 80 in Apolipoprotein B in HeFH Participants | -24.15 percent change | Standard Error 2.99 |
Secondary
Percent Change From Baseline to Week 80 in Apolipoprotein B in HoFH Participants
For HoFH participants baseline was defined as the baseline value in this study (20120124).
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Percent Change From Baseline to Week 80 in Apolipoprotein B in HoFH Participants | -19.17 percent change |
Secondary
Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HeFH Participants
For HeFH participants baseline was defined as the baseline value of the parent study 20120123.
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HeFH Participants | -36.01 percent change | Standard Error 4.28 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HeFH Participants | -34.96 percent change | Standard Error 3.05 |
Secondary
Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HoFH Participants
For HoFH participants baseline was defined as the baseline value in this study (20120124).
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HoFH Participants | -14.29 percent change |
Secondary
Percent Change From Baseline to Week 80 in Non-HDL-C in HeFH Participants
For HeFH participants baseline was defined as the baseline value of the parent study 20120123.
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Percent Change From Baseline to Week 80 in Non-HDL-C in HeFH Participants | -32.37 percent change | Standard Error 3.96 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Percent Change From Baseline to Week 80 in Non-HDL-C in HeFH Participants | -31.95 percent change | Standard Error 2.89 |
Secondary
Percent Change From Baseline to Week 80 in Non-HDL-C in HoFH Participants
For HoFH participants baseline was defined as the baseline value in this study (20120124).
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Percent Change From Baseline to Week 80 in Non-HDL-C in HoFH Participants | -13.03 percent change |
Secondary
Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HeFH Participants
For HeFH participants baseline was defined as the baseline value in the parent study 20120123.
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HeFH Participants | -28.78 percent change | Standard Error 3.48 |
| HeFH (Evolocumab in Parent Study): Evolocumab 420 mg QM | Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HeFH Participants | -28.32 percent change | Standard Error 2.47 |
Secondary
Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HoFH Participants
For HoFH participants baseline was defined as the baseline value in this study (20120124).
Time frame: Baseline and week 80
Population: Full analysis set with available data
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| HeFH (Placebo in Parent Study): Evolocumab 420 mg QM | Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HoFH Participants | 3.71 percent change |