Familial HypercholesterolemiaPhase 3HAUSER-OLENCT02624869
Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia)
A Phase 3 study testing Evolocumab in people with Familial Hypercholesterolemia, run by Amgen at 46 sites in 23 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 10 to 17 years
- Conditions the study is for
- Familial Hypercholesterolemia
- What is being tested?
- Evolocumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 163 participants
- Where
- 46 sitesin 23 countries
- Study dates
- 10 Sep 2016to 1 Jun 2021
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Show all 9Show fewer
- 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)
Who cannot join
- -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
Trial locations and contacts
46 sites in 23 countries.
46 sites in 23 countries
Italy5 sites
Research Site
Palermo, 90127
Research Site
Pisa, 56124
Research Site
Roma, 00161
Research Site
Roma, 00165
Research Site
Torino, 10126
Brazil4 sites
Research Site
Brasília, Federal District, 71625-175
Research Site
Fortaleza, Ceará, 60430-270
Research Site
São Paulo, 04040-000
Research Site
São Paulo, 05403-000
United States3 sites
Research Site
Cincinnati, Ohio, 45227
Research Site
Nashville, Tennessee, 37212
Research Site
The Bronx, New York, 10467
Austria3 sites
Research Site
Feldkirch, 6800
Research Site
Salzburg, 5020
Research Site
Vienna, 1090
Belgium3 sites
Research Site
Ghent, 9000
Research Site
La Louvière, 7100
Research Site
Leuven, 3000
Canada3 sites
Research Site
Chicoutimi, Quebec, G7H 5H6
Research Site
Chicoutimi, Quebec, G7H 7K9
Research Site
Québec, Quebec, G1V 4W2
Spain3 sites
Research Site
A Coruña, Galicia, 15001
Research Site
Córdoba, Andalusia, 14004
Research Site
Lugo, Galicia, 27003
Colombia2 sites
Research Site
Barranquilla, Atlántico, 080020
Research Site
Bucaramanga, Santander Department, 81004
South Africa2 sites
Research Site
Parktown, Gauteng, 2193
Research Site
Parow, Western Cape, 7505
Turkey (Türkiye)2 sites
Research Site
Ankara, 06500
Research Site
Izmir, 35100
About this study
Official title: Open-label, Single-Arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH)
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.
- Conditions
- Familial Hypercholesterolemia
- Being tested
- Evolocumab
- Keywords
- Hypercholesterolemia, Elevated Cholesterol, High Cholesterol, PCSK9 mutations, Severe Familial Hypercholesterolemia, evolocumab, Repatha, Heterozygous Familial Hypercholesterolemia, Homozygous Familial Hypercholesterolemia, Pediatric, Paediatric
- Medical subject headings
- Hyperlipoproteinemia Type II, Hypercholesterolemia, Homozygous Familial Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EvolocumabBiological
Also called Repatha®, AMG 145
Administered by subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| Evolocumab | Experimental | Participants receive 420 mg evolocumab administered by subcutaneous injection every 4 weeks (QM) for up to 80 weeks. Receives: Biological: Evolocumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Secondary outcomes (25)
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.
Measured: Baseline and week 80
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).
Measured: Baseline and week 80
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.
Measured: Baseline and week 80
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).
Measured: Baseline and week 80
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.
Measured: Baseline and week 80
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).
Measured: Baseline and week 80
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.
Measured: Baseline and week 80
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).
Measured: Baseline and week 80
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.
Measured: Baseline and week 80
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).
Measured: Baseline and week 80
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.
Measured: Baseline and week 80
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).
Measured: Baseline and week 80
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).
Measured: Baseline and week 80
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).
Measured: Baseline and week 80
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).
Measured: Baseline and week 80
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).
Measured: Baseline and week 80
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).
Measured: Baseline and week 80
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).
Measured: Baseline and week 80
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).
Measured: Baseline and week 80
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.
Measured: Week 80
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.
Measured: Week 80
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.
Measured: Baseline and week 80
Change From Baseline in Height at Weeks 24, 48, and 80
Measured: Baseline and weeks 24, 48, and 80
Change From Baseline in Weight at Weeks 24, 48, and 80
Measured: Baseline and weeks 24, 48, and 80
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.
Measured: Baseline and week 80
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2015 (estimated)
- Study start10 Sep 2016
- Primary completion1 Jun 2021
- Study completion1 Jun 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Familial HypercholesterolemiaStudies of the same condition, open and under way.
- More trials run by AmgenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 163 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amgen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- MD, Study director, Amgen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02624869
- Sponsor's protocol ID
- 20120124
- Other IDs
- 2015-002276-25, 2015
- Acronym
- HAUSER-OLE
- Status last verified
- May 2024
- First submitted
- 22 Oct 2015
- Last updated
- 29 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
Publications
Santos RD, Ruzza A, Hovingh GK, Stefanutti C, Mach F, Descamps OS, Bergeron J, Wang B, Bartuli A, Buonuomo PS, Greber-Platzer S, Luirink I, Bhatia AK, Raal FJ, Kastelein JJP, Wiegman A, Gaudet D. Paediatric patients with heterozygous familial hypercholesterolaemia treated with evolocumab for 80 weeks (HAUSER-OLE): a single-arm, multicentre, open-label extension of HAUSER-RCT. Lancet Diabetes Endocrinol. 2022 Oct;10(10):732-740. doi: 10.1016/S2213-8587(22)00221-2. Epub 2022 Sep 5.
PubMed 36075246Raal FJ, Hegele RA, Ruzza A, Lopez JAG, Bhatia AK, Wu J, Wang H, Gaudet D, Wiegman A, Wang J, Santos RD. Evolocumab Treatment in Pediatric Patients With Homozygous Familial Hypercholesterolemia: Pooled Data From Three Open-Label Studies. Arterioscler Thromb Vasc Biol. 2024 May;44(5):1156-1164. doi: 10.1161/ATVBAHA.123.320268. Epub 2024 Mar 28.
PubMed 38545781Santos RD, Ruzza A, Wang B, Maruff P, Schembri A, Bhatia AK, Mach F, Bergeron J, Gaudet I, St Pierre J, Kastelein JJP, Hovingh GK, Wiegman A, Gaudet D, Raal FJ. Evolocumab in paediatric heterozygous familial hypercholesterolaemia: cognitive function during 80 weeks of open-label extension treatment. Eur J Prev Cardiol. 2024 Feb 15;31(3):302-310. doi: 10.1093/eurjpc/zwad332.
PubMed 37855448Wiegman A, Ruzza A, Hovingh GK, Santos RD, Mach F, Stefanutti C, Luirink IK, Bridges I, Wang B, Bhatia AK, Raal FJ, Kastelein JJP, Gaudet D. Evolocumab treatment reduces carotid intima-media thickness in paediatric patients with heterozygous familial hypercholesterolaemia. Eur J Prev Cardiol. 2026 Jul 14;33(9):1548-1556. doi: 10.1093/eurjpc/zwae369.
PubMed 39531353
Study at a glance
From the registry record, last updated by the sponsor on 29 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-label, Single-Arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH)
- ClinicalTrials.gov ID
- NCT02624869
- Acronym / sponsor ID
- HAUSER-OLE / 20120124
- Conditions
- Familial Hypercholesterolemia
- Keywords
- Hypercholesterolemia, Elevated Cholesterol, High Cholesterol, PCSK9 mutations, Severe Familial Hypercholesterolemia, evolocumab, Repatha, Heterozygous Familial Hypercholesterolemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 163 participants (actual)
- Lead sponsor
- AmgenIndustry (pharma, biotech, devices)
- Study start
- 10 Sep 2016 (actual)
- Primary completion
- 1 Jun 2021 (actual)
- Study completion
- 1 Jun 2021 (actual)
- First posted
- 9 Dec 2015 (estimated)
- Results
- Posted 14 Jan 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Evolocumab | Experimental | Biological: EvolocumabParticipants receive 420 mg evolocumab administered by subcutaneous injection every 4 weeks (QM) for up to 80 weeks. |
Who may qualify
- Ages
- 10 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- -Completed Study 20120123 (NCT02392559) while still on assigned investigational product and did not experience a treatment-related serious adverse event
- 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.
Key exclusion criteria (5 listed)
- -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
- Moderate to severe renal dysfunction
- Active liver disease or hepatic dysfunction,
- Creatine kinase > 3 times the upper limit of normal (ULN) at screening
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EvolocumabAlso: Repatha®, AMG 145Administered by subcutaneous injection | Biological | Evolocumab |
Primary outcome
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
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.
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.
25 secondary outcomes
Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HeFH Participants
Time frame: Baseline and week 80
Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HoFH Participants
Time frame: Baseline and week 80
Percent Change From Baseline to Week 80 in Non-HDL-C in HeFH Participants
Time frame: Baseline and week 80
Percent Change From Baseline to Week 80 in Non-HDL-C in HoFH Participants
Time frame: Baseline and week 80
Percent Change From Baseline to Week 80 in Apolipoprotein B in HeFH Participants
Time frame: Baseline and week 80
Percent Change From Baseline to Week 80 in Apolipoprotein B in HoFH Participants
Time frame: Baseline and week 80
Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HeFH Participants
Time frame: Baseline and week 80
Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HoFH Participants
Time frame: Baseline and week 80
Percent Change From Baseline to Week 80 in Apolipoprotein B / Apolipoprotein A1 Ratio in HeFH Participants
Time frame: Baseline and week 80
Percent Change From Baseline to Week 80 in Apolipoprotein B/Apolipoprotein A1 Ratio in HoFH Participants
Time frame: Baseline and week 80
Change From Baseline to Week 80 in LDL-C in HeFH Participants
Time frame: Baseline and week 80
Change From Baseline to Week 80 in LDL-C in HoFH Participants
Time frame: Baseline and week 80
Change From Baseline to Week 80 in Estradiol Levels
Time frame: Baseline and week 80
Change From Baseline to Week 80 in Testosterone Levels
Time frame: Baseline and week 80
Change From Baseline to Week 80 in Follicle Stimulating Hormone (FSH) Levels
Time frame: Baseline and week 80
Change From Baseline to Week 80 in Luteinizing Hormone (LH) Levels
Time frame: Baseline and week 80
Change From Baseline to Week 80 in Adenocorticotropic Hormone (ACTH) Levels
Time frame: Baseline and week 80
Change From Baseline to Week 80 in Dehydroepiandrosterone Sulfate (DHEA-S) Levels
Time frame: Baseline and week 80
Change From Baseline to Week 80 in Cortisol Levels
Time frame: Baseline and week 80
Number of Participants With Liver Function Test Abnormalities at Week 80
Time frame: Week 80
Number of Participants With Abnormalities in Levels of Creatine Kinase (CK) at Week 80
Time frame: Week 80
Change From Baseline to Week 80 in Carotid Intima-media Thickness (cIMT)
Time frame: Baseline and week 80
Change From Baseline in Height at Weeks 24, 48, and 80
Time frame: Baseline and weeks 24, 48, and 80
Change From Baseline in Weight at Weeks 24, 48, and 80
Time frame: Baseline and weeks 24, 48, and 80
Number of Participants With Change in Tanner Staging From Baseline to Week 80
Time frame: Baseline and week 80
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 36075246Santos RD, Ruzza A, Hovingh GK, Stefanutti C, Mach F, Descamps OS, Bergeron J, Wang B, Bartuli A, Buonuomo PS, Greber-Platzer S, Luirink I, Bhatia AK, Raal FJ, Kastelein JJP, Wiegman A, Gaudet D. Paediatric patients with heterozygous familial hypercholesterolaemia treated with evolocumab for 80 weeks (HAUSER-OLE): a single-arm, multicentre, open-label extension of HAUSER-RCT. Lancet Diabetes Endocrinol. 2022 Oct;10(10):732-740. doi: 10.1016/S2213-8587(22)00221-2. Epub 2022 Sep 5.
- Background: PubMed 38545781Raal FJ, Hegele RA, Ruzza A, Lopez JAG, Bhatia AK, Wu J, Wang H, Gaudet D, Wiegman A, Wang J, Santos RD. Evolocumab Treatment in Pediatric Patients With Homozygous Familial Hypercholesterolemia: Pooled Data From Three Open-Label Studies. Arterioscler Thromb Vasc Biol. 2024 May;44(5):1156-1164. doi: 10.1161/ATVBAHA.123.320268. Epub 2024 Mar 28.
- Background: PubMed 37855448Santos RD, Ruzza A, Wang B, Maruff P, Schembri A, Bhatia AK, Mach F, Bergeron J, Gaudet I, St Pierre J, Kastelein JJP, Hovingh GK, Wiegman A, Gaudet D, Raal FJ. Evolocumab in paediatric heterozygous familial hypercholesterolaemia: cognitive function during 80 weeks of open-label extension treatment. Eur J Prev Cardiol. 2024 Feb 15;31(3):302-310. doi: 10.1093/eurjpc/zwad332.
- Derived from this study: PubMed 39531353Wiegman A, Ruzza A, Hovingh GK, Santos RD, Mach F, Stefanutti C, Luirink IK, Bridges I, Wang B, Bhatia AK, Raal FJ, Kastelein JJP, Gaudet D. Evolocumab treatment reduces carotid intima-media thickness in paediatric patients with heterozygous familial hypercholesterolaemia. Eur J Prev Cardiol. 2026 Jul 14;33(9):1548-1556. doi: 10.1093/eurjpc/zwae369.
- AmgenTrials clinical trials websiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
MD study director
Amgen
46 sites in 23 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Evolocumab in people with Familial Hypercholesterolemia, run by Amgen at 46 sites in 23 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 46 sites in Italy, Brazil, United States, Austria, Belgium and 18 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Jan 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment-emergent Adverse Events (TEAEs) (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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02624869, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 May 2024.