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

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ClinicalTrials.gov Identifier: NCT02392559
Recruitment Status : Recruiting
First Posted : March 19, 2015
Last Update Posted : September 14, 2018
Sponsor:
Information provided by (Responsible Party):
Amgen

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

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 150 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
Estimated Primary Completion Date : December 17, 2019
Estimated Study Completion Date : December 17, 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


Other Outcome Measures:
  1. Percentage change from baseline in total cholesterol [ Time Frame: Week 24 ]
    Same as outcome measure

  2. Percentage change from baseline in very low density lipoprotein-cholesterol (VLDL-C) [ Time Frame: Week 24 ]
    Same as outcome measure

  3. Percentage change from baseline in high density lipoprotein-cholesterol (HDL-C) [ Time Frame: Week 24 ]
    Same as outcome measure

  4. Percentage change from baseline in apolipoprotein-A1 (ApoA1) levels [ Time Frame: Week 24 ]
    Same as outcome measure

  5. Percentage change from baseline in triglycerides [ Time Frame: Week 24 ]
    Same as outcome measure

  6. Percentage change from baseline in lipoprotein-a (Lp(a)) levels [ Time Frame: Week 24 ]
    Same as outcome measure



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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 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 ClinicalTrials.gov identifier (NCT number): NCT02392559


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

  Show 53 Study Locations
Sponsors and Collaborators
Amgen
Investigators
Study Director: MD Amgen

Additional Information:
Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT02392559     History of Changes
Other Study ID Numbers: 20120123
2014-002277-11 ( EudraCT Number )
First Posted: March 19, 2015    Key Record Dates
Last Update Posted: September 14, 2018
Last Verified: September 2018

Keywords provided by Amgen:
Hypercholesterolemia
Elevated Cholesterol
High Cholesterol
Heterozygous Familial Hypercholesterolemia
PCSK9 mutations
Paediatric
pediatric
Childhood Familial Hypercholesterolemia

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