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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04233918
Registration number
NCT04233918
Ethics application status
Date submitted
6/01/2020
Date registered
18/01/2020
Titles & IDs
Public title
Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
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Scientific title
A Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
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Secondary ID [1]
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2019-001931-30
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Secondary ID [2]
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R1500-CL-17100
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Homozygous Familial Hypercholesterolemia
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Condition category
Condition code
Metabolic and Endocrine
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Other metabolic disorders
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Human Genetics and Inherited Disorders
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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: Drugs - Evinacumab
Experimental: Evinacumab - Part A: Single intravenous (IV) dose Part B: IV dose every 4 weeks (Q4W) until week 20 Part C: IV dose Q4W
Treatment: Drugs: Evinacumab
Part A: Single IV dose Part B \& C: IV dose Q4W
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Intervention code [1]
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Treatment: Drugs
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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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Part A: Maximum Observed Serum Concentration (Cmax) of Evinacumab
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Assessment method [1]
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Cmax was obtained directly from the plasma concentration versus time curve.
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Timepoint [1]
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At day 12
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Primary outcome [2]
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Part A: Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) of Evinacumab
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Assessment method [2]
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AUClast was defined as area under the serum concentration time-curve from zero to the last measured concentration.
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Timepoint [2]
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Up to Week 12
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Primary outcome [3]
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Part A: Terminal Half-Life (t1/2) of Evinacumab
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Assessment method [3]
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T1/2 was defined as the time required for the plasma concentration of drug to decrease 50 percent in the final stage of its elimination.
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Timepoint [3]
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Up to week 12
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Primary outcome [4]
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Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24
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Assessment method [4]
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Percent change was calculated as 100 multiplied by (calculated LDL-C value at Week 24 minus calculated LDL-C value at baseline) divided by calculated LDL-C value at baseline.
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Timepoint [4]
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Baseline to Week 24
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Secondary outcome [1]
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Part A and Part B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
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Assessment method [1]
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Any untoward medical occurrence in a participant who received investigational medicinal product (IMP) was considered an AE without regard to possibility of causal relationship with this treatment. A serious adverse event (SAE) was defined as any untoward medical occurrence that resulted in any of the following outcomes: death, life-threatening, required initial or prolonged in-patient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event. TEAE was defined as AE starting/worsening after first intake of study drug. TEAE included participants with both serious and non-serious AEs.
1 participant experienced an AE during part B that was recorded after Part B database lock. This was not reflected in the reported endpoint number of participants of 10.
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Timepoint [1]
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Part A: up to Week 24; Part B: up to Week 48
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Secondary outcome [2]
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Part B: Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24
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Assessment method [2]
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Percent change in Apo B from baseline to Week 24 was reported.
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Timepoint [2]
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Baseline to Week 24
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Secondary outcome [3]
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Part B: Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24
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Assessment method [3]
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Percent change in Non-HDL-C from baseline to Week 24 was reported.
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Timepoint [3]
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Baseline to Week 24
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Secondary outcome [4]
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Part B: Percent Change in Total Cholesterol (TC) From Baseline to Week 24
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Assessment method [4]
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Percent change in TC from baseline to Week 24 was reported.
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Timepoint [4]
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Baseline to Week 24
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Secondary outcome [5]
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Part B: Percentage of Participants With =50 Percent (%) Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24
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Assessment method [5]
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Percentage of participants who achieved reduction in calculated LDL-C = 50% at Week 24 was reported.
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Timepoint [5]
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Week 24
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Secondary outcome [6]
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Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 in Participants Who Have Negative/Negative and Null/Null Mutations
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Assessment method [6]
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Participants with HoFH was classified based on the phenotype of the Low-density lipoprotein receptor (LDLR) mutation(s), ranging from defective mutations (where the LDLR retains some LDL-binding functionality) to null or negative mutations where no functioning LDLR was expressed. Participants who have LDLR activity \<15% are considered null and participants whose LDLR activity was impaired but \>15% are LDLR defective. Percent change in calculated LDL-C from baseline to Week 24 in participants who have negative/negative and null/null mutations was reported.
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Timepoint [6]
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Baseline to Week 24
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Secondary outcome [7]
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Part B: Percent Change in Lipoprotein A (Lp[a]) From Baseline to Week 24
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Assessment method [7]
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Percent change in Lp(a) from baseline to Week 24 was reported.
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Timepoint [7]
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Baseline to Week 24
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Secondary outcome [8]
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Part B: Absolute Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline at Week 24
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Assessment method [8]
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Absolute change in LDL-C from baseline at Week 24 was reported
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Timepoint [8]
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Baseline, Week 24
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Secondary outcome [9]
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Part B: Serum Concentration of Total Evinacumab
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Assessment method [9]
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Serum concentration of total evinacumab was reported. Pre-dose samples at week 0 were assayed and the reported value is based on actual measurement.
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Timepoint [9]
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Pre-dose at Weeks 0, 4, 8, 12; End of infusion at Weeks 0.006, 4.006, 8.006, 12.006 and 24
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Secondary outcome [10]
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Part B: Maximum Serum Concentration at Steady State (Cmax,ss) of Evinacumab
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Assessment method [10]
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Maximum serum concentration (Cmax,ss) steady state following drug administration.
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Timepoint [10]
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Post-dose up to day 169
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Secondary outcome [11]
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Part B: Area Under the Serum Concentration-time Curve at Steady State (AUCtau.ss) of Evinacumab
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Assessment method [11]
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AUCtau.ss was defined as area under the serum concentration-time curve at steady state of evinacumab
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Timepoint [11]
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Post-dose up to day 169
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Secondary outcome [12]
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Part B: Minimum Serum Concentration at Steady State (Ctrough.ss) of Evinacumab
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Assessment method [12]
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Ctrough.ss was defined as minimum serum concentration at steady state of evinacumab
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Timepoint [12]
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Post-dose up to day 169
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Secondary outcome [13]
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Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 in Participants Who Have by Null/Null vs. Non-null/Null and Negative/Negative vs.Non-negative/Negative Mutations
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Assessment method [13]
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Timepoint [13]
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Baseline to Week 24
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Eligibility
Key inclusion criteria
Key
1. Diagnosis of functional HoFH by either genetic or clinical criteria as defined in the protocol
2. LDL-C >130 mg/dL at the screening visit
3. Body weight =15 kg
4. Receiving stable maximally tolerated therapy*at the screening visit *Maximally tolerated therapy could include a daily statin.
5. Willing and able to comply with clinic visits and study-related procedures
6. Parent(s) or legal guardian(s) must provide the signed informed consent form (ICF). Patients =5 years of age (or above age determined by the IRB/EC and in accordance with the local regulations and requirements) must also provide informed assent forms (IAFs) to enroll in the study, and sign and date a separate IAF or ICF signed by the parent(s)/legal guardian(s) (as appropriate based on local regulations and requirements)
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Minimum age
5
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Maximum age
11
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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
1. Background pharmacologic LMT, nutraceuticals or over-the-counter (OTC) therapies known to affect lipids, at a dose/regimen that has not been stable for at least 4 weeks (8 weeks for PCSK9 inhibitors) before the screening visit and patient is unwilling to enter the run-in period
2. For patients entering Part A, unable to temporarily discontinue apheresis from the baseline visit through the week 4 visit
3. Receiving lipid apheresis, a setting (if applicable) and schedule that has not been stable for approximately 8 weeks before the screening visit or an apheresis schedule that is not anticipated to be stable over the duration of the treatment period (48 weeks).
4. Plasmapheresis within 8 weeks of the screening visit, or plans to undergo plasmapheresis during Part A or Part B
5. Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins
6. Newly diagnosed (within 3 months prior to randomization visit) diabetes mellitus or poorly controlled diabetes as defined in the protocol
Note: Other protocol-defined criteria apply
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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
29/06/2020
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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
30/05/2023
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Sample size
Target
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Accrual to date
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Final
20
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Recruitment in Australia
Recruitment state(s)
NSW
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Recruitment hospital [1]
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Regeneron Research Center - Westmead
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Recruitment postcode(s) [1]
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2145 - Westmead
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Delaware
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Country [2]
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United States of America
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State/province [2]
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Florida
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Country [3]
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United States of America
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State/province [3]
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Kansas
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Country [4]
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United States of America
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State/province [4]
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Massachusetts
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Country [5]
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United States of America
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State/province [5]
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Pennsylvania
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Country [6]
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United States of America
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State/province [6]
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Utah
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Country [7]
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Austria
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State/province [7]
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Vienna
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Country [8]
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Netherlands
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State/province [8]
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Amsterdam
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Country [9]
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Taiwan
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State/province [9]
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Taipei
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Country [10]
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Ukraine
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State/province [10]
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Kyiv
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Regeneron Pharmaceuticals
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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 primary objective for Part A of the study is to assess the pharmacokinetics (PK) of evinacumab in pediatric patients with homozygous familial hypercholesterolemia (HoFH). The primary objective for Part B of the study is to demonstrate a reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab in pediatric (5 to 11 years of age) patients with HoFH. The secondary objective for Part A of the study is to evaluate the safety and tolerability of evinacumab administered intravenous (IV) in pediatric patients with HoFH. The secondary objectives for Part B of the study are: * To evaluate the effect of evinacumab on other lipid parameters (ie, apolipoprotein B (Apo B), non-high-density lipoprotein cholesterol (non-HDL-C), total cholesterol (TC), lipoprotein a \[Lp(a)\]) in pediatric patients with HoFH * To evaluate the safety and tolerability of evinacumab administered IV in pediatric patients with HoFH * To assess the PK of evinacumab in pediatric patients with HoFH * To assess the immunogenicity of evinacumab in pediatric patients with HoFH over time * To evaluate patient efficacy by mutation status
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Trial website
https://clinicaltrials.gov/study/NCT04233918
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Clinical Trial Management
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Address
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Regeneron Pharmaceuticals
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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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?
All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR), Analytic code
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When will data be available (start and end dates)?
Individual anonymized participant data will be considered for sharing once the indication has been approved by a regulatory body, if there is legal authority to share the data and there is not a reasonable likelihood of participant re-identification.
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Available to whom?
Qualified researchers may request access to anonymized patient level data or aggregate study data when Regeneron has received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency \[EMA\], Pharmaceuticals and Medical Devices Agency \[PMDA\], etc) for the product and indication, has the legal authority to share the data, and has made the study results publicly available (eg, scientific publication, scientific conference, clinical trial registry).
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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://vivli.org/
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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/18/NCT04233918/Prot_002.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/18/NCT04233918/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT04233918