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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02392559




Registration number
NCT02392559
Ethics application status
Date submitted
25/02/2015
Date registered
19/03/2015
Date last updated
10/12/2019

Titles & IDs
Public title
Trial Assessing Efficacy, Safety and Tolerability of PCSK9 Inhibition in Paediatric Subjects With Genetic LDL Disorders
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
Secondary ID [1] 0 0
2014-002277-11
Secondary ID [2] 0 0
20120123
Universal Trial Number (UTN)
Trial acronym
HAUSER-RCT
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Heterozygous Familial Hypercholesterolemia 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Evolocumab
Treatment: Drugs - Placebo

Experimental: QM evolocumab - Evolocumab subcutaneous injection every 4 weeks (QM)

Placebo Comparator: Placebo - Matching subcutaneous injection every 4 weeks (QM)


Treatment: Drugs: Evolocumab
Dose of subcutaneous Evolocumab every 4 weeks

Treatment: Drugs: Placebo
Dose of subcutaneous placebo treatment every 4 weeks

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage change from baseline in low density lipoprotein-cholesterol levels - Same as outcome measure
Timepoint [1] 0 0
Week 24
Secondary outcome [1] 0 0
Change from baseline in LDL-C levels - Same as outcome measure
Timepoint [1] 0 0
Week 24
Secondary outcome [2] 0 0
Percentage change from baseline in apoliprotein-b (ApoB) - Same as outcome measure
Timepoint [2] 0 0
Week 24
Secondary outcome [3] 0 0
Percentage change from baseline in total cholesterol:HDL-C ratio - Same as outcome measure
Timepoint [3] 0 0
Week 24
Secondary outcome [4] 0 0
Change in ApoB:ApoA1 ratio - The change in ApoB:ApoA1 ratio between baseline and week 24
Timepoint [4] 0 0
Week 24
Secondary outcome [5] 0 0
Percentage change in from baseline in non-HDL-C - Same as outcome measure
Timepoint [5] 0 0
Week 24
Secondary outcome [6] 0 0
Mean percentage change from baseline in low density lipoprotein-cholesterol levels at week 22 and week 24 - Same as outcome measure
Timepoint [6] 0 0
Weeks 22 and 24

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)
Minimum age
10 Years
Maximum age
17 Years
Gender
Both males and females
Can healthy volunteers participate?
No
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 PCSK9.

- Lipid apheresis within the last 12 weeks prior to screening.

- Homozygous Familial Hypercholesterolemia

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Research Site - Camperdown
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Connecticut
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United States of America
State/province [2] 0 0
Delaware
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United States of America
State/province [3] 0 0
Iowa
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United States of America
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Maryland
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United States of America
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Minnesota
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United States of America
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New York
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United States of America
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North Carolina
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United States of America
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Ohio
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United States of America
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Pennsylvania
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United States of America
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Tennessee
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United States of America
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Utah
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United States of America
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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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Belgium
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Bruxelles
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Belgium
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La Louvière
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Belgium
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Leuven
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Brazil
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Ceará
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Brazil
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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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São Paulo
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Canada
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Quebec
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Colombia
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Atlántico
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Colombia
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Santander
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Czechia
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Ostrava-Poruba
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Czechia
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Praha 5
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Czechia
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Svitavy
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Finland
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Helsinki
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Finland
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Kuopio
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Greece
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Athens
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Greece
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Thessaloniki
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Hungary
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Budapest
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Italy
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Palermo
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Italy
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Pisa
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Italy
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Roma
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Italy
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Torino
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Malaysia
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Kelantan
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Netherlands
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Amsterdam
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New Zealand
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Christchurch
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Norway
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Bergen
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Norway
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Oslo
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Poland
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Gdansk
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Portugal
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Guimaraes
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Russian Federation
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Moscow
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Russian Federation
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Saint Petersburg
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Slovenia
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Ljubljana
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South Africa
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Gauteng
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South Africa
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Western Cape
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Spain
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Andalucía
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Spain
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Cataluña
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Spain
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Galicia
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Switzerland
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Geneva 14
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Switzerland
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Reinach
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Taiwan
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Taipei
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Turkey
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Ankara
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Turkey
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Izmir
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United Kingdom
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Birmingham
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United Kingdom
State/province [59] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Amgen
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/show/NCT02392559
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
MD
Address 0 0
Amgen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications