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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT02624869
Ethics application status
Date submitted
22/10/2015
Date registered
9/12/2015
Date last updated
15/06/2021

Titles & IDs
Public title
Open Label Study to Evaluate Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Pediatric Subjects (10 to 17 Years of Age) With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH).
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
Other interventions - evolocumab (AMG 145)

Experimental: evolocumab (AMG 145) - Single arm all subjects receive evolocumab (AMG 145) every 4 weeks (QM)


Other interventions: evolocumab (AMG 145)
Administered by subcutaneous injection

Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants with treatment-related Adverse Events as assessed by CTCAE V4.0
Timepoint [1] 0 0
80 weeks
Secondary outcome [1] 0 0
Percent change from baseline in low-density lipoprotein cholesterol (LDL-C)
Timepoint [1] 0 0
Week 80
Secondary outcome [2] 0 0
Percent change from baseline in non-HDL-C (non high density lipoprotein cholesterol)
Timepoint [2] 0 0
Week 80
Secondary outcome [3] 0 0
Percent change from baseline in apoliprotein-b (ApoB)
Timepoint [3] 0 0
Week 80
Secondary outcome [4] 0 0
Percent change from baseline total cholesterol/HDL-C ratio - The change in total cholesterol/HDL-C ratio from baseline at week 80
Timepoint [4] 0 0
Week 80
Secondary outcome [5] 0 0
Percent change from baseline ApoB/ApoA1 ratio - The change in ApoB/ApoA1 ratio from baseline at week 80
Timepoint [5] 0 0
Week 80
Secondary outcome [6] 0 0
Change from baseline in LDL-C
Timepoint [6] 0 0
Week 80
Secondary outcome [7] 0 0
Change from baseline in steroid hormones - FSH, LH, ACTH, DHEA-S, cortisol, estradiol/testosterone [females/males, respectively]
Timepoint [7] 0 0
Week 80
Secondary outcome [8] 0 0
Change from baseline in Carotid intima-media thickness (cIMT) at week 80
Timepoint [8] 0 0
Week 80
Secondary outcome [9] 0 0
Subject incidence of abnormal muscle enzyme levels
Timepoint [9] 0 0
Week 80
Secondary outcome [10] 0 0
Subject incidence of abnormal liver enzyme levels
Timepoint [10] 0 0
Week 80
Secondary outcome [11] 0 0
Change from baseline in height
Timepoint [11] 0 0
Week 24, 48 and 80
Secondary outcome [12] 0 0
Change from baseline in weight.
Timepoint [12] 0 0
Week 24, 48 and 80
Secondary outcome [13] 0 0
Change from baseline in pubertal development - Tanner Staging
Timepoint [13] 0 0
Week 24, 48 and 80
Secondary outcome [14] 0 0
Number of participants with abnormal neurological examination findings - The neurologic examination will include assessments of motor, sensory, reflexes, coordination and gait.
Timepoint [14] 0 0
Week 80
Secondary outcome [15] 0 0
Change from baseline score in the components of the Cogstate battery (cognitive function assessments) - The Cogstate battery will include the following tests: Groton Maze Learning Task (GMLT; Executive Function); One Card Learning Test (OCL; Visual Memory); Identification Test (IDN; Attention/Vigilance); Detection Test (DET; Psychomotor Speed)
Timepoint [15] 0 0
Week 24, 48, and 80

Eligibility
Key inclusion criteria
Subjects with HeFH:

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

Subjects with 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
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
All Subjects

-Currently receiving treatment in another investigational device or drug study, or less
than 30 days since ending treament on another investigational device or drug study(s);
except Study 20120123

Subjects with HoFH:

- Moderate to severe renal dysfunction

- Active liver disease or hepatic dysfunction,

- CK > 3 times the ULN at screening

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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
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
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
To describe the safety and tolerability of 80 weeks of subcutaneous (SC) evolocumab (AMG 145)
when added to standard of care in pediatric subjects 10 to 17 years of age with HeFH or HoFH.
Trial website
https://clinicaltrials.gov/show/NCT02624869
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