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Trial record 1 of 1 for:    NCT02624869
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Open Label Study to Evaluate Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Pediatric Subjects (10 to 17 Years of Age) With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH). (HAUSER-OLE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02624869
Recruitment Status : Active, not recruiting
First Posted : December 9, 2015
Last Update Posted : December 10, 2019
Sponsor:
Information provided by (Responsible Party):
Amgen

Brief Summary:
To describe the safety and tolerability of 80 weeks of subcutaneous (SC) evolocumab (AMG 145) when added to standard of care in pediatric subjects 10 to 17 years of age with HeFH or HoFH.

Condition or disease Intervention/treatment Phase
Familial Hypercholesterolemia Biological: evolocumab (AMG 145) Phase 3

Detailed Description:
An open label, single arm study to evaluate the safety and tolerability of 80 weeks of SC evolocumab when added to standard of care in pediatric subjects 10 to 17 years of age with HeFH or HoFH.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 163 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : September 10, 2016
Estimated Primary Completion Date : June 7, 2021
Estimated Study Completion Date : June 7, 2021

Resource links provided by the National Library of Medicine

Drug Information available for: Evolocumab

Arm Intervention/treatment
Experimental: evolocumab (AMG 145)
Single arm all subjects receive evolocumab (AMG 145) every 4 weeks (QM)
Biological: evolocumab (AMG 145)
Administered by subcutaneous injection
Other Name: Repatha




Primary Outcome Measures :
  1. Number of participants with treatment-related Adverse Events as assessed by CTCAE V4.0 [ Time Frame: 80 weeks ]

Secondary Outcome Measures :
  1. Percent change from baseline in low-density lipoprotein cholesterol (LDL-C) [ Time Frame: Week 80 ]
  2. Percent change from baseline in non-HDL-C (non high density lipoprotein cholesterol) [ Time Frame: Week 80 ]
  3. Percent change from baseline in apoliprotein-b (ApoB) [ Time Frame: Week 80 ]
  4. Percent change from baseline total cholesterol/HDL-C ratio [ Time Frame: Week 80 ]
    The change in total cholesterol/HDL-C ratio from baseline at week 80

  5. Percent change from baseline ApoB/ApoA1 ratio [ Time Frame: Week 80 ]
    The change in ApoB/ApoA1 ratio from baseline at week 80

  6. Change from baseline in LDL-C [ Time Frame: Week 80 ]
  7. Change from baseline in steroid hormones [ Time Frame: Week 80 ]
    FSH, LH, ACTH, DHEA-S, cortisol, estradiol/testosterone [females/males, respectively]

  8. Change from baseline in Carotid intima-media thickness (cIMT) at week 80 [ Time Frame: Week 80 ]
  9. Subject incidence of abnormal muscle enzyme levels [ Time Frame: Week 80 ]
  10. Subject incidence of abnormal liver enzyme levels [ Time Frame: Week 80 ]
  11. Change from baseline in height [ Time Frame: Week 24, 48 and 80 ]
  12. Change from baseline in weight. [ Time Frame: Week 24, 48 and 80 ]
  13. Change from baseline in pubertal development [ Time Frame: Week 24, 48 and 80 ]
    Tanner Staging

  14. Number of participants with abnormal neurological examination findings [ Time Frame: Week 80 ]
    The neurologic examination will include assessments of motor, sensory, reflexes, coordination and gait.

  15. Change from baseline score in the components of the Cogstate battery (cognitive function assessments) [ Time Frame: Week 24, 48, and 80 ]
    The Cogstate battery will include the following tests: Groton Maze Learning Task (GMLT; Executive Function); One Card Learning Test (OCL; Visual Memory); Identification Test (IDN; Attention/Vigilance); Detection Test (DET; Psychomotor Speed)



Information from the National Library of Medicine

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Ages Eligible for Study:   10 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Subjects with HeFH:

-Completed Study 20120123 while still on assigned investigational product and did not experience a treatment‑related serious adverse event

Subjects with 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:

All Subjects

-Currently receiving treatment in another investigational device or drug study, or less than 30 days since ending treament on another investigational device or drug study(s); except Study 20120123

Subjects with HoFH:

  • Moderate to severe renal dysfunction
  • Active liver disease or hepatic dysfunction,
  • CK > 3 times the ULN at screening

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02624869


Locations
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United States, New York
Research Site
Bronx, New York, United States, 10467
United States, Ohio
Research Site
Cincinnati, Ohio, United States, 45227
United States, Tennessee
Research Site
Nashville, Tennessee, United States, 37212
Australia, New South Wales
Research Site
Camperdown, New South Wales, Australia, 2050
Austria
Research Site
Feldkirch, Austria, 6800
Research Site
Salzburg, Austria, 5020
Research Site
Wien, Austria, 1090
Belgium
Research Site
Gent, Belgium, 9000
Research Site
La Louvière, Belgium, 7100
Research Site
Leuven, Belgium, 3000
Brazil
Research Site
Fortaleza, Ceará, Brazil, 60430-270
Research Site
Brasília, Distrito Federal, Brazil, 71625-175
Research Site
São Paulo, Brazil, 04040-000
Research Site
São Paulo, Brazil, 05403-000
Canada, Quebec
Research Site
Chicoutimi, Quebec, Canada, G7H 5H6
Research Site
Chicoutimi, Quebec, Canada, G7H 7K9
Canada
Research Site
Quebec, Canada, G1V 4W2
Colombia
Research Site
Barranquilla, Atlántico, Colombia, 080020
Research Site
Bucaramanga, Santander, Colombia, 81004
Czechia
Research Site
Svitavy, Czechia, 568 25
Greece
Research Site
Athens, Greece, 17674
Research Site
Thessaloniki, Greece, 54642
Hungary
Research Site
Budapest, Hungary, 1094
Italy
Research Site
Palermo, Italy, 90127
Research Site
Pisa, Italy, 56124
Research Site
Roma, Italy, 00161
Research Site
Roma, Italy, 00165
Research Site
Torino, Italy, 10126
Malaysia
Research Site
Kota Bharu, Kelantan, Malaysia, 16150
Netherlands
Research Site
Amsterdam, Netherlands, 1066 EC
Norway
Research Site
Bergen, Norway, 5021
Research Site
Oslo, Norway, 0586
Poland
Research Site
Gdansk, Poland, 80-952
Portugal
Research Site
Guimaraes, Portugal, 4835-044
Russian Federation
Research Site
Saint Petersburg, Russian Federation, 191025
Slovenia
Research Site
Ljubljana, Slovenia, 1000
South Africa
Research Site
Parktown, Gauteng, South Africa, 2193
Research Site
Parow, Western Cape, South Africa, 7505
Spain
Research Site
Cordoba, Andalucía, Spain, 14004
Research Site
A Coruña, Galicia, Spain, 15001
Research Site
Lugo, Galicia, Spain, 27003
Switzerland
Research Site
Geneva 14, Switzerland, 1211
Research Site
Reinach, Switzerland, 4153
Turkey
Research Site
Ankara, Turkey, 06500
Research Site
Izmir, Turkey, 35100
United Kingdom
Research Site
Birmingham, United Kingdom, B4 6NH
Sponsors and Collaborators
Amgen
Investigators
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Study Director: MD Amgen
Additional Information:
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Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT02624869    
Other Study ID Numbers: 20120124
2015-002276-25 ( EudraCT Number )
First Posted: December 9, 2015    Key Record Dates
Last Update Posted: December 10, 2019
Last Verified: December 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Time Frame: Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication (or other new use) have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
Access Criteria: Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors, and if not approved, may be further arbitrated by a Data Sharing Independent Review Panel. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the link below.
URL: https://www.amgen.com/datasharing
Keywords provided by Amgen:
Hypercholesterolemia
Elevated Cholesterol
High Cholesterol
PCSK9 mutations
Severe Familial Hypercholesterolemia
evolocumab
Repatha
Heterozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia
Pediatric
Paediatric
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type II
Hypercholesterolemia
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias
Evolocumab
Anticholesteremic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents