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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT02392559
Ethics application status
Date submitted
25/02/2015
Date registered
19/03/2015

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
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

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

Outcomes
Primary outcome [1] 0 0
Percent Change From Baseline to Week 24 in LDL-C
Timepoint [1] 0 0
Baseline, Week 24
Secondary outcome [1] 0 0
Mean Percent Change From Baseline to Mean of Weeks 22 and 24 in LDL-C
Timepoint [1] 0 0
Baseline, Week 22, Week 24
Secondary outcome [2] 0 0
Change From Baseline to Week 24 in LDL-C
Timepoint [2] 0 0
Baseline, Week 24
Secondary outcome [3] 0 0
Percent Change From Baseline to Week 24 in Non-HDL-C
Timepoint [3] 0 0
Baseline, Week 24
Secondary outcome [4] 0 0
Percent Change From Baseline to Week 24 in Apoliprotein-B (ApoB)
Timepoint [4] 0 0
Baseline, Week 24
Secondary outcome [5] 0 0
Percent Change From Baseline to Week 24 in Total Cholesterol/HDL-C Ratio
Timepoint [5] 0 0
Baseline, Week 24
Secondary outcome [6] 0 0
Percent Change From Baseline to Week 24 in ApoB:ApoA1 Ratio
Timepoint [6] 0 0
Baseline, Week 24
Secondary outcome [7] 0 0
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation (DC), Fatal TEAEs, and Device-Related TEAEs
Timepoint [7] 0 0
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.
Secondary outcome [8] 0 0
Number of Participants With Maximum Post-Baseline Laboratory Toxicities of Grade = 3
Timepoint [8] 0 0
Week 24
Secondary outcome [9] 0 0
Change From Baseline Over Time in Systolic Blood Pressure
Timepoint [9] 0 0
Baseline, Week 4, Week 12, Week 20, Week 22, Week 24
Secondary outcome [10] 0 0
Change From Baseline Over Time in Diastolic Blood Pressure
Timepoint [10] 0 0
Baseline, Week 4, Week 12, Week 20, Week 22, Week 24
Secondary outcome [11] 0 0
Change From Baseline Over Time in Heart Rate
Timepoint [11] 0 0
Baseline, Week 4, Week 12, Week 20, Week 22, Week 24
Secondary outcome [12] 0 0
Number of Participants Testing Positive for Anti-Evolocumab Antibodies
Timepoint [12] 0 0
up to Week 24
Secondary outcome [13] 0 0
Serum Evolocumab Concentrations Over Time
Timepoint [13] 0 0
Week 12, Week 22, Week 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
Sex
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 proprotein convertase subtilisin/kexin type 9 (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
24/03/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
25/11/2019
Sample size
Target
Accrual to date
Final
158
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
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
Minnesota
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
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
State/province [13] 0 0
Feldkirch
Country [14] 0 0
Austria
State/province [14] 0 0
Salzburg
Country [15] 0 0
Austria
State/province [15] 0 0
Wien
Country [16] 0 0
Belgium
State/province [16] 0 0
Bruxelles
Country [17] 0 0
Belgium
State/province [17] 0 0
La Louvière
Country [18] 0 0
Belgium
State/province [18] 0 0
Leuven
Country [19] 0 0
Brazil
State/province [19] 0 0
Ceará
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Brazil
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Distrito Federal
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Brazil
State/province [21] 0 0
Espírito Santo
Country [22] 0 0
Brazil
State/province [22] 0 0
São Paulo
Country [23] 0 0
Canada
State/province [23] 0 0
Quebec
Country [24] 0 0
Colombia
State/province [24] 0 0
Atlántico
Country [25] 0 0
Colombia
State/province [25] 0 0
Santander
Country [26] 0 0
Czechia
State/province [26] 0 0
Ostrava-Poruba
Country [27] 0 0
Czechia
State/province [27] 0 0
Praha 5
Country [28] 0 0
Czechia
State/province [28] 0 0
Svitavy
Country [29] 0 0
Finland
State/province [29] 0 0
Helsinki
Country [30] 0 0
Finland
State/province [30] 0 0
Kuopio
Country [31] 0 0
Greece
State/province [31] 0 0
Athens
Country [32] 0 0
Greece
State/province [32] 0 0
Thessaloniki
Country [33] 0 0
Hungary
State/province [33] 0 0
Budapest
Country [34] 0 0
Italy
State/province [34] 0 0
Palermo
Country [35] 0 0
Italy
State/province [35] 0 0
Pisa
Country [36] 0 0
Italy
State/province [36] 0 0
Roma
Country [37] 0 0
Italy
State/province [37] 0 0
Torino
Country [38] 0 0
Malaysia
State/province [38] 0 0
Kelantan
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Netherlands
State/province [39] 0 0
Amsterdam
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New Zealand
State/province [40] 0 0
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
State/province [43] 0 0
Gdansk
Country [44] 0 0
Portugal
State/province [44] 0 0
Guimaraes
Country [45] 0 0
Russian Federation
State/province [45] 0 0
Moscow
Country [46] 0 0
Russian Federation
State/province [46] 0 0
Saint Petersburg
Country [47] 0 0
Slovenia
State/province [47] 0 0
Ljubljana
Country [48] 0 0
South Africa
State/province [48] 0 0
Gauteng
Country [49] 0 0
South Africa
State/province [49] 0 0
Western Cape
Country [50] 0 0
Spain
State/province [50] 0 0
Andalucía
Country [51] 0 0
Spain
State/province [51] 0 0
Cataluña
Country [52] 0 0
Spain
State/province [52] 0 0
Galicia
Country [53] 0 0
Switzerland
State/province [53] 0 0
Geneva 14
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Switzerland
State/province [54] 0 0
Reinach
Country [55] 0 0
Taiwan
State/province [55] 0 0
Taipei
Country [56] 0 0
Turkey
State/province [56] 0 0
Ankara
Country [57] 0 0
Turkey
State/province [57] 0 0
Izmir
Country [58] 0 0
United Kingdom
State/province [58] 0 0
Birmingham
Country [59] 0 0
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
Trial website
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR)
When will data be available (start and end dates)?
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.
Available to whom?
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.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.amgen.com/datasharing


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT02392559