Help guide our efforts to modernize
Send us your comments by March 14, 2020. Menu
Trial record 1 of 1 for:    20120123
Previous Study | Return to List | Next Study

Trial Assessing Efficacy, Safety and Tolerability of PCSK9 Inhibition in Paediatric Subjects With Genetic LDL Disorders (HAUSER-RCT)

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. Identifier: NCT02392559
Recruitment Status : Completed
First Posted : March 19, 2015
Last Update Posted : December 10, 2019
Information provided by (Responsible Party):

Brief Summary:
A study to assess safety and efficacy of evolocumab (AMG-145) in paediatric subjects aged 10-17 years diagnosed with heterozygous familial hypercholesterolemia.

Condition or disease Intervention/treatment Phase
Heterozygous Familial Hypercholesterolemia Drug: Evolocumab Drug: Placebo Phase 3

Detailed Description:
A study to evaluate the effect of 24 weeks of subcutaneous (SC) evolocumab compared with placebo,when added to standard of care, on percent change from baseline in low‑density lipoprotein cholesterol (LDL-C) in pediatric subjects 10 to 17 years of age with HeFH

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 159 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: Double-blind, Randomized, Multicenter, Placebo-Controlled Study to Characterize the Efficacy, Safety, and Tolerability of 24 Weeks of Evolocumab for LDL-C Reduction in Pediatric Subjects 10 to 17 Years of Age With HeFH
Actual Study Start Date : February 15, 2016
Actual Primary Completion Date : November 25, 2019
Actual Study Completion Date : November 25, 2019

Resource links provided by the National Library of Medicine

Drug Information available for: Evolocumab

Arm Intervention/treatment
Experimental: QM evolocumab
Evolocumab subcutaneous injection every 4 weeks (QM)
Drug: Evolocumab
Dose of subcutaneous Evolocumab every 4 weeks

Placebo Comparator: Placebo
Matching subcutaneous injection every 4 weeks (QM)
Drug: Placebo
Dose of subcutaneous placebo treatment every 4 weeks

Primary Outcome Measures :
  1. Percentage change from baseline in low density lipoprotein-cholesterol levels [ Time Frame: Week 24 ]
    Same as outcome measure

Secondary Outcome Measures :
  1. Change from baseline in LDL-C levels [ Time Frame: Week 24 ]
    Same as outcome measure

  2. Percentage change from baseline in apoliprotein-b (ApoB) [ Time Frame: Week 24 ]
    Same as outcome measure

  3. Percentage change from baseline in total cholesterol:HDL-C ratio [ Time Frame: Week 24 ]
    Same as outcome measure

  4. Change in ApoB:ApoA1 ratio [ Time Frame: Week 24 ]
    The change in ApoB:ApoA1 ratio between baseline and week 24

  5. Percentage change in from baseline in non-HDL-C [ Time Frame: Week 24 ]
    Same as outcome measure

  6. Mean percentage change from baseline in low density lipoprotein-cholesterol levels at week 22 and week 24 [ Time Frame: Weeks 22 and 24 ]
    Same as outcome measure

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   10 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male or female ≥ 10 to ≤ 17 years of age (before 18th birthday)
  • Diagnosis of heterozygous familial hypercholesterolemia
  • On an approved statin with stable optimized dose for ≥ 4 weeks
  • Other lipid-lowering therapy stable for ≥ 4 weeks (fibrates must be stable for ≥ 6 weeks)
  • Fasting LDL-C ≥ 130 mg/dL (3.4 mmol/L)
  • Fasting triglycerides ≤ 400 mg/dL (4.5 mmol/L)

Exclusion Criteria:

  • Type 1 diabetes, or type 2 diabetes that is or poorly controlled
  • Uncontrolled hyperthyroidism or hypothyroidism
  • Cholesterylester transfer protein (CETP) inhibitor in the last 12 months, or mipomersen or lomitapide in the last 5 months
  • Previously received evolocumab or any other investigational therapy to inhibit PCSK9.
  • Lipid apheresis within the last 12 weeks prior to screening.
  • Homozygous Familial Hypercholesterolemia

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 identifier (NCT number): NCT02392559

Show Show 66 study locations
Sponsors and Collaborators
Layout table for investigator information
Study Director: MD Amgen

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Amgen Identifier: NCT02392559    
Other Study ID Numbers: 20120123
2014-002277-11 ( EudraCT Number )
First Posted: March 19, 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.
Keywords provided by Amgen:
Elevated Cholesterol
High Cholesterol
Heterozygous Familial Hypercholesterolemia
PCSK9 mutations
Childhood Familial Hypercholesterolemia
Additional relevant MeSH terms:
Layout table for MeSH terms
Hyperlipoproteinemia Type II
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Antibodies, Monoclonal
Anticholesteremic Agents
Hypolipidemic Agents
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents
Immunologic Factors
Physiological Effects of Drugs