Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia)
NCT ID: NCT02624869
Last Updated: 2024-05-29
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
163 participants
INTERVENTIONAL
2016-09-10
2021-06-01
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Evolocumab
Participants receive 420 mg evolocumab administered by subcutaneous injection every 4 weeks (QM) for up to 80 weeks.
Evolocumab
Administered by subcutaneous injection
Interventions
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Evolocumab
Administered by subcutaneous injection
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
-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
HoFH:
* Moderate to severe renal dysfunction
* Active liver disease or hepatic dysfunction,
* Creatine kinase \> 3 times the upper limit of normal (ULN) at screening
10 Years
17 Years
ALL
No
Sponsors
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Amgen
INDUSTRY
Responsible Party
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Principal Investigators
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MD
Role: STUDY_DIRECTOR
Amgen
Locations
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Research Site
The Bronx, New York, United States
Research Site
Cincinnati, Ohio, United States
Research Site
Nashville, Tennessee, United States
Research Site
Camperdown, New South Wales, Australia
Research Site
Feldkirch, , Austria
Research Site
Salzburg, , Austria
Research Site
Vienna, , Austria
Research Site
Ghent, , Belgium
Research Site
La Louvière, , Belgium
Research Site
Leuven, , Belgium
Research Site
Fortaleza, Ceará, Brazil
Research Site
Brasília, Federal District, Brazil
Research Site
São Paulo, , Brazil
Research Site
São Paulo, , Brazil
Research Site
Chicoutimi, Quebec, Canada
Research Site
Chicoutimi, Quebec, Canada
Research Site
Québec, Quebec, Canada
Research Site
Barranquilla, Atlántico, Colombia
Research Site
Bucaramanga, Santander Department, Colombia
Research Site
Svitavy, , Czechia
Research Site
Athens, , Greece
Research Site
Thessaloniki, , Greece
Research Site
Budapest, , Hungary
Research Site
Palermo, , Italy
Research Site
Pisa, , Italy
Research Site
Roma, , Italy
Research Site
Roma, , Italy
Research Site
Torino, , Italy
Research Site
Kota Bharu, Kelantan, Malaysia
Research Site
Amsterdam, , Netherlands
Research Site
Bergen, , Norway
Research Site
Oslo, , Norway
Research Site
Gdansk, , Poland
Research Site
Guimarães, , Portugal
Research Site
Saint Petersburg, , Russia
Research Site
Ljubljana, , Slovenia
Research Site
Parktown, Gauteng, South Africa
Research Site
Parow, Western Cape, South Africa
Research Site
Córdoba, Andalusia, Spain
Research Site
A Coruña, Galicia, Spain
Research Site
Lugo, Galicia, Spain
Research Site
Geneva, , Switzerland
Research Site
Reinach, , Switzerland
Research Site
Ankara, , Turkey (Türkiye)
Research Site
Izmir, , Turkey (Türkiye)
Research Site
Birmingham, , United Kingdom
Countries
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References
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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.
Raal 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.
Santos 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.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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AmgenTrials clinical trials website
Other Identifiers
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2015-002276-25
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
20120124
Identifier Type: -
Identifier Source: org_study_id
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