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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02392559
Registration number
NCT02392559
Ethics application status
Date submitted
25/02/2015
Date registered
19/03/2015
Date last updated
8/11/2022
Titles & IDs
Public title
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
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Scientific 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
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Secondary ID [1]
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2014-002277-11
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Secondary ID [2]
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20120123
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Universal Trial Number (UTN)
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Trial acronym
HAUSER-RCT
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Heterozygous Familial Hypercholesterolemia
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Condition category
Condition code
Metabolic and Endocrine
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Other metabolic disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Evolocumab
Treatment: Drugs - Placebo
Placebo Comparator: Placebo - Matching subcutaneous injection every 4 weeks (QM)
Experimental: EvoMab 420 mg QM - Evolocumab subcutaneous injection QM
Treatment: Drugs: Evolocumab
Dose of subcutaneous evolocumab every 4 weeks
Treatment: Drugs: Placebo
Dose of subcutaneous placebo treatment every 4 weeks
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Percent Change From Baseline to Week 24 in LDL-C
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Assessment method [1]
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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.
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Timepoint [1]
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Baseline, Week 24
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Secondary outcome [1]
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Mean Percent Change From Baseline to Mean of Weeks 22 and 24 in LDL-C
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Assessment method [1]
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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.
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Timepoint [1]
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Baseline, Week 22, Week 24
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Secondary outcome [2]
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Change From Baseline to Week 24 in LDL-C
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Assessment method [2]
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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.
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Timepoint [2]
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Baseline, Week 24
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Secondary outcome [3]
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Percent Change From Baseline to Week 24 in Non-HDL-C
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Assessment method [3]
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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
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Timepoint [3]
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Baseline, Week 24
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Secondary outcome [4]
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Percent Change From Baseline to Week 24 in Apoliprotein-B (ApoB)
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Assessment method [4]
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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.
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Timepoint [4]
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Baseline, Week 24
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Secondary outcome [5]
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Percent Change From Baseline to Week 24 in Total Cholesterol/HDL-C Ratio
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Assessment method [5]
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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.
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Timepoint [5]
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Baseline, Week 24
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Secondary outcome [6]
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Percent Change From Baseline to Week 24 in ApoB:ApoA1 Ratio
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Assessment method [6]
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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.
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Timepoint [6]
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Baseline, Week 24
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Secondary outcome [7]
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation (DC), Fatal TEAEs, and Device-Related TEAEs
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Assessment method [7]
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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.
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Timepoint [7]
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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.
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Secondary outcome [8]
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Number of Participants With Maximum Post-Baseline Laboratory Toxicities of Grade = 3
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Assessment method [8]
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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.
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Timepoint [8]
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Week 24
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Secondary outcome [9]
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Change From Baseline Over Time in Systolic Blood Pressure
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Assessment method [9]
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Timepoint [9]
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Baseline, Week 4, Week 12, Week 20, Week 22, Week 24
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Secondary outcome [10]
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Change From Baseline Over Time in Diastolic Blood Pressure
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Assessment method [10]
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Timepoint [10]
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Baseline, Week 4, Week 12, Week 20, Week 22, Week 24
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Secondary outcome [11]
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Change From Baseline Over Time in Heart Rate
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Assessment method [11]
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Timepoint [11]
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Baseline, Week 4, Week 12, Week 20, Week 22, Week 24
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Secondary outcome [12]
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Number of Participants Testing Positive for Anti-Evolocumab Antibodies
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Assessment method [12]
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Timepoint [12]
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up to Week 24
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Secondary outcome [13]
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Serum Evolocumab Concentrations Over Time
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Assessment method [13]
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Timepoint [13]
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Week 12, Week 22, Week 24
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Eligibility
Key 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)
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Minimum age
10
Years
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Maximum age
17
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
24/03/2016
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
25/11/2019
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Sample size
Target
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Accrual to date
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Final
158
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Recruitment in Australia
Recruitment state(s)
NSW
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Recruitment hospital [1]
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Research Site - Camperdown
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Recruitment postcode(s) [1]
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2050 - Camperdown
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Recruitment outside Australia
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United States of America
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Connecticut
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Delaware
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Iowa
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Maryland
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Minnesota
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West Virginia
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Austria
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Feldkirch
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Austria
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Salzburg
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Austria
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Wien
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Bruxelles
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La Louvière
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Leuven
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Ceará
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Distrito Federal
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Brazil
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Espírito Santo
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Brazil
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Praha 5
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Svitavy
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Helsinki
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Taipei
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Turkey
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Izmir
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Birmingham
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United Kingdom
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Amgen
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Address
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02392559
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Trial related presentations / publications
Santos RD, Ruzza A, Hovingh GK, Wiegman A, Mach F, Kurtz CE, Hamer A, Bridges I, Bartuli A, Bergeron J, Szamosi T, Santra S, Stefanutti C, Descamps OS, Greber-Platzer S, Luirink I, Kastelein JJP, Gaudet D; HAUSER-RCT Investigators. Evolocumab in Pediatric Heterozygous Familial Hypercholesterolemia. N Engl J Med. 2020 Oct 1;383(14):1317-1327. doi: 10.1056/NEJMoa2019910. Epub 2020 Aug 29.
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.
Gaudet D, Ruzza A, Bridges I, Maruff P, Schembri A, Hamer A, Mach F, Bergeron J, Gaudet I, Pierre JS, Kastelein JJP, Hovingh GK, Wiegman A, Raal FJ, Santos RD. Cognitive function with evolocumab in pediatric heterozygous familial hypercholesterolemia. J Clin Lipidol. 2022 Sep-Oct;16(5):676-684. doi: 10.1016/j.jacl.2022.07.005. Epub 2022 Jul 21.
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Public notes
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Contacts
Principal investigator
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MD
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Address
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Amgen
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02392559
Download to PDF