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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)

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ClinicalTrials.gov Identifier: NCT02624869
Recruitment Status : Recruiting
First Posted : December 9, 2015
Last Update Posted : December 13, 2018
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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 115 participants
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 29, 2021
Estimated Study Completion Date : June 29, 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)



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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


Contacts
Contact: Amgen Call Center 866-572-6436 medinfo@amgen.com

  Show 35 Study Locations
Sponsors and Collaborators
Amgen
Investigators
Study Director: MD Amgen

Additional Information:
Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT02624869     History of Changes
Other Study ID Numbers: 20120124
2015-002276-25 ( EudraCT Number )
First Posted: December 9, 2015    Key Record Dates
Last Update Posted: December 13, 2018
Last Verified: December 2018
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:
Hypercholesterolemia
Hyperlipoproteinemia Type II
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias
Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs