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Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Paediatric Subjects With Genetic Low-Density Lipoprotein (LDL) Disorders (HAUSER-RCT)

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ClinicalTrials.gov Identifier: NCT02392559
Recruitment Status : Completed
First Posted : March 19, 2015
Results First Posted : July 15, 2020
Last Update Posted : July 15, 2020
Sponsor:
Information provided by (Responsible Party):
Amgen

Study Description
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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 heterozygous familial hypercholesterolemia (HeFH).
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 158 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 : March 24, 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

Arms and Interventions
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Arm Intervention/treatment
Placebo Comparator: Placebo
Matching subcutaneous injection every 4 weeks (QM)
 Drug: Placebo
Dose of subcutaneous placebo treatment every 4 weeks
Experimental: EvoMab 420 mg QM
Evolocumab subcutaneous injection QM
 Drug: Evolocumab
Dose of subcutaneous evolocumab every 4 weeks
Other Name: AMG 145; EvoMab


Outcome Measures
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Primary Outcome Measures :
  1. Percent Change From Baseline to Week 24 in LDL-C [ Time Frame: Baseline, Week 24 ]
    Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from interactive voice response system [IVRS]), scheduled visit and the interaction of treatment with scheduled visit as covariates. The model uses an unstructured covariance.


Secondary Outcome Measures :
  1. Mean Percent Change From Baseline to Mean of Weeks 22 and 24 in LDL-C [ Time Frame: Baseline, Week 22, Week 24 ]
    Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates.

  2. Change From Baseline to Week 24 in LDL-C [ Time Frame: Baseline, Week 24 ]
    Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates.

  3. Percent Change From Baseline to Week 24 in Non-HDL-C [ Time Frame: Baseline, Week 24 ]
    Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates

  4. Percent Change From Baseline to Week 24 in Apoliprotein-B (ApoB) [ Time Frame: Baseline, Week 24 ]
    Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates.

  5. Percent Change From Baseline to Week 24 in Total Cholesterol/HDL-C Ratio [ Time Frame: Baseline, Week 24 ]
    Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates.

  6. Percent Change From Baseline to Week 24 in ApoB:ApoA1 Ratio [ Time Frame: Baseline, Week 24 ]
    Least squares mean is from the repeated measures model which includes treatment group, stratification factors of age and screening LDL-C (from IVRS), scheduled visit and the interaction of treatment with scheduled visit as covariates.

  7. Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation (DC), Fatal TEAEs, and Device-Related TEAEs [ Time Frame: From first dose of study drug up to and including 30 days after the last dose or end of study date (Week 24), whichever was earlier. Mean (SD) duration on study was 5.664 (0.278) and 5.608 (0.137) months for Placebo and EvoMab arms, respectively. ]
    An adverse event (AE) is defined as any untoward medical occurrence that does not necessarily have a causal relationship with study treatment. An SAE is defined as an adverse event that: is fatal; is a life threatening; requires in-patient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; or other medically important serious event. Events were graded by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) grading scale (1=mild; 2=moderate; 3=severe; 4=life-threatening; 5=death). Events were defined as treatment emergent if they occurred after the first dose of study drug and up to and including 30 days after the last dose or the end of study date, whichever is earlier.

  8. Number of Participants With Maximum Post-Baseline Laboratory Toxicities of Grade ≥ 3 [ Time Frame: Week 24 ]
    Laboratory toxicity grading was based on NCI CTCAE grading. Grade 3 indicates severe toxicity and Grade 4 indicates life-threatening toxicity. Values representing a worsening from baseline are shown.

  9. Change From Baseline Over Time in Systolic Blood Pressure [ Time Frame: Baseline, Week 4, Week 12, Week 20, Week 22, Week 24 ]
  10. Change From Baseline Over Time in Diastolic Blood Pressure [ Time Frame: Baseline, Week 4, Week 12, Week 20, Week 22, Week 24 ]
  11. Change From Baseline Over Time in Heart Rate [ Time Frame: Baseline, Week 4, Week 12, Week 20, Week 22, Week 24 ]
  12. Number of Participants Testing Positive for Anti-Evolocumab Antibodies [ Time Frame: up to Week 24 ]
  13. Serum Evolocumab Concentrations Over Time [ Time Frame: Week 12, Week 22, Week 24 ]

Eligibility Criteria
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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:

  • 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 proprotein convertase subtilisin/kexin type 9 (PCSK9).
  • Lipid apheresis within the last 12 weeks prior to screening.
  • Homozygous familial hypercholesterolemia
Contacts and Locations
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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): NCT02392559


Locations
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Sponsors and Collaborators
Amgen
Investigators
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Study Director: MD Amgen
  Study Documents (Full-Text)

Documents provided by Amgen:
Study Protocol  [PDF] September 1, 2015
Statistical Analysis Plan  [PDF] September 16, 2019

More Information
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Additional Information:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT02392559    
Other Study ID Numbers: 20120123
2014-002277-11 ( EudraCT Number )
First Posted: March 19, 2015    Key Record Dates
Results First Posted: July 15, 2020
Last Update Posted: July 15, 2020
Last Verified: July 2020
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
Heterozygous Familial Hypercholesterolemia
 PCSK9 mutations
Paediatric
pediatric
Childhood Familial Hypercholesterolemia
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