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Trial record 1 of 1 for:    20120123
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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.
 
ClinicalTrials.gov Identifier: NCT02392559
Recruitment Status : Completed
First Posted : March 19, 2015
Last Update Posted : December 10, 2019
Sponsor:
Information provided by (Responsible Party):
Amgen

Tracking Information
First Submitted Date  ICMJE February 25, 2015
First Posted Date  ICMJE March 19, 2015
Last Update Posted Date December 10, 2019
Actual Study Start Date  ICMJE February 15, 2016
Actual Primary Completion Date November 25, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 10, 2015)
Percentage change from baseline in low density lipoprotein-cholesterol levels [ Time Frame: Week 24 ]
Same as outcome measure
Original Primary Outcome Measures  ICMJE
 (submitted: March 13, 2015)
Percentage change from baseline in low density lipoprotein-cholesterol levels [ Time Frame: 24 weeks ]
Percentage change from baseline in low density lipoprotein-cholesterol levels from baseline to 24 weeks.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 10, 2015)
  • Change from baseline in LDL-C levels [ Time Frame: Week 24 ]
    Same as outcome measure
  • Percentage change from baseline in apoliprotein-b (ApoB) [ Time Frame: Week 24 ]
    Same as outcome measure
  • Percentage change from baseline in total cholesterol:HDL-C ratio [ Time Frame: Week 24 ]
    Same as outcome measure
  • Change in ApoB:ApoA1 ratio [ Time Frame: Week 24 ]
    The change in ApoB:ApoA1 ratio between baseline and week 24
  • Percentage change in from baseline in non-HDL-C [ Time Frame: Week 24 ]
    Same as outcome measure
  • 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
Original Secondary Outcome Measures  ICMJE
 (submitted: March 13, 2015)
  • Incidence of treatment-emergent adverse effects [ Time Frame: 24 weeks ]
    The number of treatment-emergent adverse events recorded during the study period
  • Percentage change from baseline in non-HDL-C [ Time Frame: 24 weeks ]
    Percentage change in non-HDL-C levels from baseline to week 24
  • Serum evolocumab concentrations [ Time Frame: Weeks 12, 22 and 24 ]
    Serum levels of evolocumab will be measured
  • Change in LDL-C levels [ Time Frame: 24 weeks ]
    Change in true levels of low density lipoprotein-cholesterol between baseline and week 24.
  • Percentage change from baseline in apoliprotein-b (ApoB) [ Time Frame: 24 weeks ]
    The percentage change in ApoB levels between baseline and week 24
  • The change from baseline in cholesterol:HDL-C ratio [ Time Frame: 24 weeks ]
    The change in cholesterol:HDL-C ratio between baseline and week 24
  • Change in ApoB:ApoA1 ratio [ Time Frame: 24 weeks ]
    The change in ApoB:ApoA1 ratio between baseline and week 24
  • Incidence of abnormal laboratory values and vital signs [ Time Frame: 24 weeks ]
    Incidence of laboratory values and vital signs recorded that classify as abnormal
  • Incidence of anti-evolocumab antibody formation [ Time Frame: 24 weeks ]
    Incidence of anti-evolocumab (neutralized or biding) formation during the study period
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures
 (submitted: March 13, 2015)
  • Percentage change in total cholesterol [ Time Frame: 24 weeks ]
    The percentage change in total cholesterol levels between baseline and week 24.
  • Percentage change in very low density lipoprotein-cholesterol (VLDL-C) [ Time Frame: 24 weeks ]
    The percentage change in VLDL-C levels between baseline and week 24
  • Percentage change in high density lipoprotein-cholesterol (HDL-C) [ Time Frame: 24 weeks ]
    The percentage change in HDL-C levels between baseline and week 24
  • Percentage change in apolipoprotein-A1 (ApoA1) levels [ Time Frame: 24 weeks ]
    The percentage change in ApoA1 levels between baseline and week 24
  • Percentage change in triglycerides [ Time Frame: 24 weeks ]
    The percentage change intriglyceride levels between baseline and week 24
  • Percentage change in lipoprotein-a (Lp(a)) levels [ Time Frame: 24 weeks ]
    The percentage change in Lp(a) levels between baseline and week 24
 
Descriptive Information
Brief Title  ICMJE Trial Assessing Efficacy, Safety and Tolerability of PCSK9 Inhibition in Paediatric Subjects With Genetic LDL Disorders
Official Title  ICMJE 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
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.
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
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Heterozygous Familial Hypercholesterolemia
Intervention  ICMJE
  • Drug: Evolocumab
    Dose of subcutaneous Evolocumab every 4 weeks
  • Drug: Placebo
    Dose of subcutaneous placebo treatment every 4 weeks
Study Arms  ICMJE
  • Experimental: QM evolocumab
    Evolocumab subcutaneous injection every 4 weeks (QM)
    Intervention: Drug: Evolocumab
  • Placebo Comparator: Placebo
    Matching subcutaneous injection every 4 weeks (QM)
    Intervention: Drug: Placebo
Publications * Gaudet D, Langslet G, Gidding SS, Luirink IK, Ruzza A, Kurtz C, Lu C, Somaratne R, Raal FJ, Wiegman A. Efficacy, safety, and tolerability of evolocumab in pediatric patients with heterozygous familial hypercholesterolemia: Rationale and design of the HAUSER-RCT study. J Clin Lipidol. 2018 Sep - Oct;12(5):1199-1207. doi: 10.1016/j.jacl.2018.05.007. Epub 2018 May 22.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 25, 2019)
159
Original Estimated Enrollment  ICMJE
 (submitted: March 13, 2015)
150
Actual Study Completion Date  ICMJE November 25, 2019
Actual Primary Completion Date November 25, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 10 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Austria,   Belgium,   Brazil,   Canada,   Colombia,   Czechia,   Finland,   Greece,   Hungary,   Italy,   Malaysia,   Netherlands,   New Zealand,   Norway,   Poland,   Portugal,   Russian Federation,   Slovenia,   South Africa,   Spain,   Switzerland,   Taiwan,   Turkey,   United Kingdom,   United States
Removed Location Countries Czech Republic
 
Administrative Information
NCT Number  ICMJE NCT02392559
Other Study ID Numbers  ICMJE 20120123
2014-002277-11 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
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
Supporting Materials: Statistical Analysis Plan (SAP)
Supporting Materials: Informed Consent Form (ICF)
Supporting Materials: 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
Responsible Party Amgen
Study Sponsor  ICMJE Amgen
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: MD Amgen
PRS Account Amgen
Verification Date December 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP