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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02624869




Registration number
NCT02624869
Ethics application status
Date submitted
22/10/2015
Date registered
9/12/2015

Titles & IDs
Public title
Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia)
Scientific title
Open-label, Single-Arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH)
Secondary ID [1] 0 0
2015-002276-25
Secondary ID [2] 0 0
20120124
Universal Trial Number (UTN)
Trial acronym
HAUSER-OLE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
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: Other - Evolocumab

Experimental: Evolocumab - Participants receive 420 mg evolocumab administered by subcutaneous injection every 4 weeks (QM) for up to 80 weeks.


Treatment: Other: Evolocumab
Administered by subcutaneous injection
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
From first dose of evolocumab in this study up to and including 30 days after the last dose or up to the end of study date, whichever was earlier; up to 80 weeks.
Secondary outcome [1] 0 0
Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HeFH Participants
Timepoint [1] 0 0
Baseline and week 80
Secondary outcome [2] 0 0
Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HoFH Participants
Timepoint [2] 0 0
Baseline and week 80
Secondary outcome [3] 0 0
Percent Change From Baseline to Week 80 in Non-HDL-C in HeFH Participants
Timepoint [3] 0 0
Baseline and week 80
Secondary outcome [4] 0 0
Percent Change From Baseline to Week 80 in Non-HDL-C in HoFH Participants
Timepoint [4] 0 0
Baseline and week 80
Secondary outcome [5] 0 0
Percent Change From Baseline to Week 80 in Apolipoprotein B in HeFH Participants
Timepoint [5] 0 0
Baseline and week 80
Secondary outcome [6] 0 0
Percent Change From Baseline to Week 80 in Apolipoprotein B in HoFH Participants
Timepoint [6] 0 0
Baseline and week 80
Secondary outcome [7] 0 0
Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HeFH Participants
Timepoint [7] 0 0
Baseline and week 80
Secondary outcome [8] 0 0
Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HoFH Participants
Timepoint [8] 0 0
Baseline and week 80
Secondary outcome [9] 0 0
Percent Change From Baseline to Week 80 in Apolipoprotein B / Apolipoprotein A1 Ratio in HeFH Participants
Timepoint [9] 0 0
Baseline and week 80
Secondary outcome [10] 0 0
Percent Change From Baseline to Week 80 in Apolipoprotein B/Apolipoprotein A1 Ratio in HoFH Participants
Timepoint [10] 0 0
Baseline and week 80
Secondary outcome [11] 0 0
Change From Baseline to Week 80 in LDL-C in HeFH Participants
Timepoint [11] 0 0
Baseline and week 80
Secondary outcome [12] 0 0
Change From Baseline to Week 80 in LDL-C in HoFH Participants
Timepoint [12] 0 0
Baseline and week 80
Secondary outcome [13] 0 0
Change From Baseline to Week 80 in Estradiol Levels
Timepoint [13] 0 0
Baseline and week 80
Secondary outcome [14] 0 0
Change From Baseline to Week 80 in Testosterone Levels
Timepoint [14] 0 0
Baseline and week 80
Secondary outcome [15] 0 0
Change From Baseline to Week 80 in Follicle Stimulating Hormone (FSH) Levels
Timepoint [15] 0 0
Baseline and week 80
Secondary outcome [16] 0 0
Change From Baseline to Week 80 in Luteinizing Hormone (LH) Levels
Timepoint [16] 0 0
Baseline and week 80
Secondary outcome [17] 0 0
Change From Baseline to Week 80 in Adenocorticotropic Hormone (ACTH) Levels
Timepoint [17] 0 0
Baseline and week 80
Secondary outcome [18] 0 0
Change From Baseline to Week 80 in Dehydroepiandrosterone Sulfate (DHEA-S) Levels
Timepoint [18] 0 0
Baseline and week 80
Secondary outcome [19] 0 0
Change From Baseline to Week 80 in Cortisol Levels
Timepoint [19] 0 0
Baseline and week 80
Secondary outcome [20] 0 0
Number of Participants With Liver Function Test Abnormalities at Week 80
Timepoint [20] 0 0
Week 80
Secondary outcome [21] 0 0
Number of Participants With Abnormalities in Levels of Creatine Kinase (CK) at Week 80
Timepoint [21] 0 0
Week 80
Secondary outcome [22] 0 0
Change From Baseline to Week 80 in Carotid Intima-media Thickness (cIMT)
Timepoint [22] 0 0
Baseline and week 80
Secondary outcome [23] 0 0
Change From Baseline in Height at Weeks 24, 48, and 80
Timepoint [23] 0 0
Baseline and weeks 24, 48, and 80
Secondary outcome [24] 0 0
Change From Baseline in Weight at Weeks 24, 48, and 80
Timepoint [24] 0 0
Baseline and weeks 24, 48, and 80
Secondary outcome [25] 0 0
Number of Participants With Change in Tanner Staging From Baseline to Week 80
Timepoint [25] 0 0
Baseline and week 80

Eligibility
Key inclusion criteria
Heterozygous Familial Hypercholesterolemia (HeFH):

-Completed Study 20120123 (NCT02392559) while still on assigned investigational product and did not experience a treatment-related serious adverse event

Homozygous Familial Hypercholesterolemia (HoFH):

* Male or female, = 10 to = 17 years of age at time of enrollment
* Diagnosis of HoFH
* On a low-fat diet and receiving background lipid-lowering therapy
* Lipid-lowering therapy unchanged for = 4 weeks prior to LDL-C screening; fibrates must be stable for at least 6 weeks prior to screening.
* Fasting LDL-C at screening = 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
-Currently receiving treatment in another investigational device or drug study, or less than 30 days since ending treatment on another investigational device or drug study(s); except Study 20120123

HoFH:

* Moderate to severe renal dysfunction
* Active liver disease or hepatic dysfunction,
* Creatine kinase > 3 times the upper limit of normal (ULN) at screening

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
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
10/09/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/06/2021
Sample size
Target
Accrual to date
Final
163
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
New York
Country [2] 0 0
United States of America
State/province [2] 0 0
Ohio
Country [3] 0 0
United States of America
State/province [3] 0 0
Tennessee
Country [4] 0 0
Austria
State/province [4] 0 0
Feldkirch
Country [5] 0 0
Austria
State/province [5] 0 0
Salzburg
Country [6] 0 0
Austria
State/province [6] 0 0
Wien
Country [7] 0 0
Belgium
State/province [7] 0 0
Gent
Country [8] 0 0
Belgium
State/province [8] 0 0
La Louvière
Country [9] 0 0
Belgium
State/province [9] 0 0
Leuven
Country [10] 0 0
Brazil
State/province [10] 0 0
Ceará
Country [11] 0 0
Brazil
State/province [11] 0 0
Distrito Federal
Country [12] 0 0
Brazil
State/province [12] 0 0
São Paulo
Country [13] 0 0
Canada
State/province [13] 0 0
Quebec
Country [14] 0 0
Colombia
State/province [14] 0 0
Atlántico
Country [15] 0 0
Colombia
State/province [15] 0 0
Santander
Country [16] 0 0
Czechia
State/province [16] 0 0
Svitavy
Country [17] 0 0
Greece
State/province [17] 0 0
Athens
Country [18] 0 0
Greece
State/province [18] 0 0
Thessaloniki
Country [19] 0 0
Hungary
State/province [19] 0 0
Budapest
Country [20] 0 0
Italy
State/province [20] 0 0
Palermo
Country [21] 0 0
Italy
State/province [21] 0 0
Pisa
Country [22] 0 0
Italy
State/province [22] 0 0
Roma
Country [23] 0 0
Italy
State/province [23] 0 0
Torino
Country [24] 0 0
Malaysia
State/province [24] 0 0
Kelantan
Country [25] 0 0
Netherlands
State/province [25] 0 0
Amsterdam
Country [26] 0 0
Norway
State/province [26] 0 0
Bergen
Country [27] 0 0
Norway
State/province [27] 0 0
Oslo
Country [28] 0 0
Poland
State/province [28] 0 0
Gdansk
Country [29] 0 0
Portugal
State/province [29] 0 0
Guimaraes
Country [30] 0 0
Russian Federation
State/province [30] 0 0
Saint Petersburg
Country [31] 0 0
Slovenia
State/province [31] 0 0
Ljubljana
Country [32] 0 0
South Africa
State/province [32] 0 0
Gauteng
Country [33] 0 0
South Africa
State/province [33] 0 0
Western Cape
Country [34] 0 0
Spain
State/province [34] 0 0
Andalucía
Country [35] 0 0
Spain
State/province [35] 0 0
Galicia
Country [36] 0 0
Switzerland
State/province [36] 0 0
Geneva 14
Country [37] 0 0
Switzerland
State/province [37] 0 0
Reinach
Country [38] 0 0
Turkey
State/province [38] 0 0
Ankara
Country [39] 0 0
Turkey
State/province [39] 0 0
Izmir
Country [40] 0 0
United Kingdom
State/province [40] 0 0
Birmingham

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/NCT02624869