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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)
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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)
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Secondary ID [1]
0
0
2015-002276-25
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Secondary ID [2]
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20120124
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Universal Trial Number (UTN)
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Trial acronym
HAUSER-OLE
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Familial Hypercholesterolemia
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0
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Condition category
Condition code
Metabolic and Endocrine
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0
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0
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Other metabolic disorders
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Human Genetics and Inherited Disorders
0
0
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0
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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: 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
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Intervention code [1]
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Treatment: Other
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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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Number of Participants With Treatment-emergent Adverse Events (TEAEs)
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Assessment method [1]
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An adverse event is defined as any untoward medical occurrence in a clinical trial participant, not necessarily having a causal relationship with study treatment.
A serious AE is as an AE that met at least 1 of the following criteria:
* fatal;
* life threatening;
* required in-patient hospitalization or prolongation of existing hospitalization;
* resulted in persistent or significant disability/incapacity;
* congenital anomaly/birth defect;
* other medically important serious event.
AEs were graded for severity using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0:
Grade 1: Mild; asymptomatic or mild symptoms; Grade 2: Moderate; minimal, local or noninvasive intervention indicated; Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; Grade 4: Life-threatening consequences; urgent intervention indicated; Grade 5: Death related to AE.
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Timepoint [1]
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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.
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Secondary outcome [1]
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0
Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HeFH Participants
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Assessment method [1]
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For HeFH participants baseline was defined as the baseline value of the parent study 20120123.
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Timepoint [1]
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Baseline and week 80
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Secondary outcome [2]
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0
Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HoFH Participants
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Assessment method [2]
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For HoFH participants baseline was defined as the baseline value in this study (20120124).
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Timepoint [2]
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Baseline and week 80
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Secondary outcome [3]
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Percent Change From Baseline to Week 80 in Non-HDL-C in HeFH Participants
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Assessment method [3]
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For HeFH participants baseline was defined as the baseline value of the parent study 20120123.
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Timepoint [3]
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Baseline and week 80
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Secondary outcome [4]
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Percent Change From Baseline to Week 80 in Non-HDL-C in HoFH Participants
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Assessment method [4]
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0
For HoFH participants baseline was defined as the baseline value in this study (20120124).
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Timepoint [4]
0
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Baseline and week 80
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Secondary outcome [5]
0
0
Percent Change From Baseline to Week 80 in Apolipoprotein B in HeFH Participants
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Assessment method [5]
0
0
For HeFH participants baseline was defined as the baseline value in the parent study 20120123.
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Timepoint [5]
0
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Baseline and week 80
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Secondary outcome [6]
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0
Percent Change From Baseline to Week 80 in Apolipoprotein B in HoFH Participants
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Assessment method [6]
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For HoFH participants baseline was defined as the baseline value in this study (20120124).
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Timepoint [6]
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Baseline and week 80
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Secondary outcome [7]
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Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HeFH Participants
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Assessment method [7]
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0
For HeFH participants baseline was defined as the baseline value in the parent study 20120123.
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Timepoint [7]
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0
Baseline and week 80
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Secondary outcome [8]
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0
Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HoFH Participants
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Assessment method [8]
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0
For HoFH participants baseline was defined as the baseline value in this study (20120124).
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Timepoint [8]
0
0
Baseline and week 80
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Secondary outcome [9]
0
0
Percent Change From Baseline to Week 80 in Apolipoprotein B / Apolipoprotein A1 Ratio in HeFH Participants
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Assessment method [9]
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0
For HeFH participants baseline was defined as the baseline value in the parent study 20120123.
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Timepoint [9]
0
0
Baseline and week 80
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Secondary outcome [10]
0
0
Percent Change From Baseline to Week 80 in Apolipoprotein B/Apolipoprotein A1 Ratio in HoFH Participants
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Assessment method [10]
0
0
For HoFH participants baseline was defined as the baseline value in this study (20120124).
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Timepoint [10]
0
0
Baseline and week 80
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Secondary outcome [11]
0
0
Change From Baseline to Week 80 in LDL-C in HeFH Participants
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Assessment method [11]
0
0
For HeFH participants baseline was defined as the baseline value of the parent study 20120123.
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Timepoint [11]
0
0
Baseline and week 80
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Secondary outcome [12]
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0
Change From Baseline to Week 80 in LDL-C in HoFH Participants
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Assessment method [12]
0
0
For HoFH participants baseline was defined as the baseline value in this study (20120124).
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Timepoint [12]
0
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Baseline and week 80
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Secondary outcome [13]
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Change From Baseline to Week 80 in Estradiol Levels
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Assessment method [13]
0
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For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).
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Timepoint [13]
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Baseline and week 80
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Secondary outcome [14]
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Change From Baseline to Week 80 in Testosterone Levels
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Assessment method [14]
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For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).
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Timepoint [14]
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Baseline and week 80
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Secondary outcome [15]
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Change From Baseline to Week 80 in Follicle Stimulating Hormone (FSH) Levels
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Assessment method [15]
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For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).
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Timepoint [15]
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0
Baseline and week 80
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Secondary outcome [16]
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Change From Baseline to Week 80 in Luteinizing Hormone (LH) Levels
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Assessment method [16]
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For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).
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Timepoint [16]
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Baseline and week 80
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Secondary outcome [17]
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Change From Baseline to Week 80 in Adenocorticotropic Hormone (ACTH) Levels
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Assessment method [17]
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For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).
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Timepoint [17]
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Baseline and week 80
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Secondary outcome [18]
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Change From Baseline to Week 80 in Dehydroepiandrosterone Sulfate (DHEA-S) Levels
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Assessment method [18]
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For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).
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Timepoint [18]
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Baseline and week 80
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Secondary outcome [19]
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Change From Baseline to Week 80 in Cortisol Levels
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Assessment method [19]
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For HeFH participants baseline was defined as the baseline value in the parent study 20120123. For HoFH participants baseline was defined as the baseline value in this study (20120124).
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Timepoint [19]
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Baseline and week 80
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Secondary outcome [20]
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Number of Participants With Liver Function Test Abnormalities at Week 80
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Assessment method [20]
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Liver function tests included alanine aminotransferase (ALT) levels, aspartate aminotransferase (AST) levels and total bilirubin levels.
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Timepoint [20]
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Week 80
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Secondary outcome [21]
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Number of Participants With Abnormalities in Levels of Creatine Kinase (CK) at Week 80
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Assessment method [21]
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The number of participants with levels of creatine kinase greater than 5 times the upper limit of normal (ULN) and greater than 10 times the ULN, measured by the central laboratory.
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Timepoint [21]
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0
Week 80
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Secondary outcome [22]
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Change From Baseline to Week 80 in Carotid Intima-media Thickness (cIMT)
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Assessment method [22]
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Carotid intima-media thickness measures the thickness of the intima and media, the inner two layers of the carotid artery, and is used to determine the extent of plaque buildup in the walls of the arteries (atherosclerosis) supplying blood to the head.
CIMT was measured by ultrasonography and analyzed at a core laboratory. The largest values measured in the left common carotid artery (LCCA) and the right common carotid artery (RCCA) are averaged in this analysis.
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Timepoint [22]
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0
Baseline and week 80
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Secondary outcome [23]
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0
Change From Baseline in Height at Weeks 24, 48, and 80
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Assessment method [23]
0
0
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Timepoint [23]
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Baseline and weeks 24, 48, and 80
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Secondary outcome [24]
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Change From Baseline in Weight at Weeks 24, 48, and 80
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Assessment method [24]
0
0
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Timepoint [24]
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Baseline and weeks 24, 48, and 80
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Secondary outcome [25]
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Number of Participants With Change in Tanner Staging From Baseline to Week 80
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Assessment method [25]
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Pubertal growth and sexual maturity was assessed separately for males and females using the 5 Tanner stages where stage 1 = prepubertal and stage 5 = mature.
The number of participants with any change in Tanner Stage from baseline is reported.
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Timepoint [25]
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Baseline and week 80
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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)
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Minimum age
10
Years
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Maximum age
17
Years
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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
-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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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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
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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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
10/09/2016
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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
1/06/2021
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Sample size
Target
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Accrual to date
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Final
163
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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
Country [1]
0
0
United States of America
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State/province [1]
0
0
New York
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Country [2]
0
0
United States of America
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State/province [2]
0
0
Ohio
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Country [3]
0
0
United States of America
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State/province [3]
0
0
Tennessee
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Country [4]
0
0
Austria
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State/province [4]
0
0
Feldkirch
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Country [5]
0
0
Austria
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State/province [5]
0
0
Salzburg
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Country [6]
0
0
Austria
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State/province [6]
0
0
Wien
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Country [7]
0
0
Belgium
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State/province [7]
0
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Gent
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Country [8]
0
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Belgium
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State/province [8]
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0
La Louvière
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Country [9]
0
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Belgium
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State/province [9]
0
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Leuven
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Country [10]
0
0
Brazil
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State/province [10]
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0
Ceará
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Country [11]
0
0
Brazil
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State/province [11]
0
0
Distrito Federal
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Country [12]
0
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Brazil
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State/province [12]
0
0
São Paulo
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Country [13]
0
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Canada
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State/province [13]
0
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Quebec
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Country [14]
0
0
Colombia
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State/province [14]
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Atlántico
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Colombia
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State/province [15]
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0
Santander
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Country [16]
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0
Czechia
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State/province [16]
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0
Svitavy
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0
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Greece
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State/province [17]
0
0
Athens
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Country [18]
0
0
Greece
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State/province [18]
0
0
Thessaloniki
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Country [19]
0
0
Hungary
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State/province [19]
0
0
Budapest
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Country [20]
0
0
Italy
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State/province [20]
0
0
Palermo
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Country [21]
0
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Italy
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State/province [21]
0
0
Pisa
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Country [22]
0
0
Italy
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State/province [22]
0
0
Roma
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Country [23]
0
0
Italy
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State/province [23]
0
0
Torino
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Country [24]
0
0
Malaysia
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State/province [24]
0
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Kelantan
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Country [25]
0
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Netherlands
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0
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Amsterdam
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0
0
Norway
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State/province [26]
0
0
Bergen
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Country [27]
0
0
Norway
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State/province [27]
0
0
Oslo
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Country [28]
0
0
Poland
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State/province [28]
0
0
Gdansk
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Country [29]
0
0
Portugal
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State/province [29]
0
0
Guimaraes
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Country [30]
0
0
Russian Federation
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State/province [30]
0
0
Saint Petersburg
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Country [31]
0
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Slovenia
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State/province [31]
0
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Ljubljana
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0
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South Africa
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Gauteng
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Country [33]
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South Africa
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State/province [33]
0
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Western Cape
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Country [34]
0
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Spain
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0
0
Andalucía
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Country [35]
0
0
Spain
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State/province [35]
0
0
Galicia
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Country [36]
0
0
Switzerland
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State/province [36]
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0
Geneva 14
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Country [37]
0
0
Switzerland
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State/province [37]
0
0
Reinach
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Country [38]
0
0
Turkey
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State/province [38]
0
0
Ankara
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Country [39]
0
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Turkey
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State/province [39]
0
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Izmir
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Country [40]
0
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United Kingdom
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State/province [40]
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Birmingham
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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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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The main purpose of this study is to describe the safety and tolerability of 80 weeks of subcutaneous (SC) evolocumab when added to standard of care in children 10 to 17 years of age with familial hypercholesterolemia.
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Trial website
https://clinicaltrials.gov/study/NCT02624869
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Trial related presentations / publications
Santos RD, Ruzza A, Hovingh GK, Stefanutti C, Mach F, Descamps OS, Bergeron J, Wang B, Bartuli A, Buonuomo PS, Greber-Platzer S, Luirink I, Bhatia AK, Raal FJ, Kastelein JJP, Wiegman A, Gaudet D. Paediatric patients with heterozygous familial hypercholesterolaemia treated with evolocumab for 80 weeks (HAUSER-OLE): a single-arm, multicentre, open-label extension of HAUSER-RCT. Lancet Diabetes Endocrinol. 2022 Oct;10(10):732-740. doi: 10.1016/S2213-8587(22)00221-2. Epub 2022 Sep 5. Raal FJ, Hegele RA, Ruzza A, Lopez JAG, Bhatia AK, Wu J, Wang H, Gaudet D, Wiegman A, Wang J, Santos RD. Evolocumab Treatment in Pediatric Patients With Homozygous Familial Hypercholesterolemia: Pooled Data From Three Open-Label Studies. Arterioscler Thromb Vasc Biol. 2024 May;44(5):1156-1164. doi: 10.1161/ATVBAHA.123.320268. Epub 2024 Mar 28. Santos RD, Ruzza A, Wang B, Maruff P, Schembri A, Bhatia AK, Mach F, Bergeron J, Gaudet I, St Pierre J, Kastelein JJP, Hovingh GK, Wiegman A, Gaudet D, Raal FJ. Evolocumab in paediatric heterozygous familial hypercholesterolaemia: cognitive function during 80 weeks of open-label extension treatment. Eur J Prev Cardiol. 2024 Feb 15;31(3):302-310. doi: 10.1093/eurjpc/zwad332.
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Public notes
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Contacts
Principal investigator
Name
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0
MD
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Address
0
0
Amgen
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Country
0
0
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Phone
0
0
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Fax
0
0
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Email
0
0
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Contact person for public queries
Name
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0
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Address
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Country
0
0
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Phone
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Fax
0
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Email
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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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)
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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.
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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.
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: https://www.amgen.com/datasharing
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What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/69/NCT02624869/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/69/NCT02624869/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT02624869