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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03399786
Registration number
NCT03399786
Ethics application status
Date submitted
8/01/2018
Date registered
16/01/2018
Date last updated
18/05/2021
Titles & IDs
Public title
Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
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Scientific title
A Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
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Secondary ID [1]
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2017-001388-19
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Secondary ID [2]
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R1500-CL-1629
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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
Treatment: Drugs - Placebo
Experimental: evinacumab -
Experimental: Placebo -
Treatment: Drugs: evinacumab
IV administration of evinacumab
Treatment: Drugs: Placebo
IV administration of placebo
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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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Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 (Intent-to-Treat [ITT] Estimand)
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Assessment method [1]
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Percent change was calculated as 100x(calculated LDL-C value at Week 24 - calculated LDL-C value at baseline)/calculated LDL-C value at baseline. The baseline LDL-C value was the last calculated LDL-C value obtained before the first dose of double-blind-study drug. The calculated LDL-C at week 24 was the LDL-C value obtained within the week 24 efficacy analysis window, regardless of adherence to treatment and subsequent therapies (intent-to-treat \[ITT\] estimand). The ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [1]
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Week 24
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Secondary outcome [1]
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Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24 (ITT Estimand)
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Assessment method [1]
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Percent change in Apo B from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [1]
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Week 24
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Secondary outcome [2]
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Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24 (ITT Estimand)
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Assessment method [2]
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Percent change from baseline in non-HDL-C at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [2]
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Week 24
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Secondary outcome [3]
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Percent Change in Total Cholesterol (TC) From Baseline to Week 24 (ITT Estimand)
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Assessment method [3]
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Percent change in TC from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [3]
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Week 24
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Secondary outcome [4]
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Percentage of Participants With =30% Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 (ITT Estimand)
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Assessment method [4]
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Percentage of participants who achieved reduction in calculated LDL-C =30% at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [4]
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At Week 24
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Secondary outcome [5]
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Percentage of Participants With =50% Reduction in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) at Week 24 (ITT Estimand)
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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 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [5]
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At Week 24
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Secondary outcome [6]
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Absolute Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 (ITT Estimand)
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Assessment method [6]
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Absolute change in calculated LDL-C from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [6]
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Week 24
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Secondary outcome [7]
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Percentage of Participants Who Met United States (US) Apheresis Eligibility Criteria at Week 24 (ITT Estimand)
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Assessment method [7]
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US apheresis eligibility criteria included participants who had inadequate response to diet and LMTs after 6 months of treatment and with functional Homozygous familial hypercholesterolemia (HoFH) or Heterozygous familial hypercholesterolemia (HeFH) (with 0-1 risk factor) with LDL-C = 300 mg/dL (7.77 mmol/L). Percentage of participants who met US apheresis eligibility criteria at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [7]
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At Week 24
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Secondary outcome [8]
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Percentage of Participants With Low-Density Lipoprotein Cholesterol (LDL-C) <100 Milligrams Per Deciliter (mg/dL) (2.59 Millimoles Per Liter [mmol/L]) at Week 24 (ITT Estimand)
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Assessment method [8]
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Percentage of participants with LDL-C value \<100 mg/dL (2.59 mmol/L) in the DBTP at Week 24 was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analysed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [8]
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At Week 24
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Secondary outcome [9]
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Percentage of Participants Who Met European Union (EU) Apheresis Eligibility Criteria at Week 24 (ITT Estimand)
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Assessment method [9]
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EU apheresis eligibility criteria included participants who had inadequate response to diet and Lipid modifying therapies (LMTs) after 3 months of treatment, Primary prevention: Participants with Familial hypercholesterolemia (FH) with LDL-C \>160 mg/dL (4.2 mmol/L) and Cardiovascular (CV) events in close relatives. Secondary prevention: Participants with progressive CV events with LDL-C \> 120 to 130 mg/dL (3.1-3.4 mmol/L). Percentage of participants who met EU apheresis eligibility criteria at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [9]
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At Week 24
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Secondary outcome [10]
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Percentage of Participants With Calculated Low-Density Lipoprotein Cholesterol (LDL-C) <70 mg/dL (1.81 mmol/L) at Week 24 (ITT Estimand)
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Assessment method [10]
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Percentage of participants with LDL-C \<70 mg/dL (1.81 mmol/L) at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [10]
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At Week 24
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Secondary outcome [11]
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Percent Change in Fasting Triglycerides (TG) From Baseline to Week 24 (ITT Estimand)
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Assessment method [11]
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Percent change from baseline in TG at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [11]
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Week 24
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Secondary outcome [12]
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Percent Change in Lipoprotein A (Lp[a]) From Baseline to Week 24 (ITT Estimand)
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Assessment method [12]
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Percent change in Lp(a) from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [12]
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Week 24
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Secondary outcome [13]
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Absolute Change in Apolipoprotein B (Apo B) From Baseline to Week 24 (ITT Estimand)
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Assessment method [13]
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Absolute change in Apo B from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [13]
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Week 24
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Secondary outcome [14]
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Absolute Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24 (ITT Estimand)
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Assessment method [14]
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Absolute change in non-HDL-C from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [14]
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Week 24
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Secondary outcome [15]
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Absolute Change in Total Cholesterol (TC) From Baseline to Week 24 (ITT Estimand)
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Assessment method [15]
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Absolute change in TC from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [15]
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Week 24
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Secondary outcome [16]
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Percent Change in Apolipoprotein CIII (Apo CIII) From Baseline to Week 24 (ITT Estimand)
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Assessment method [16]
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Percent change in Apo CIII from baseline at Week 24 in the DBTP was reported; value obtained regardless of adherence to study treatment and subsequent therapies (ITT estimand). ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
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Timepoint [16]
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Week 24
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Eligibility
Key inclusion criteria
Key
1. Diagnosis of functional HoFH
2. If undergoing LDL apheresis, must have initiated LDL apheresis at least 3 months prior to screening and must have been on a stable weekly or every other week schedule and/or stable settings for at least 8 weeks
3. Willing to consistently maintain his/her usual low fat or heart-healthy diet for the duration of the study
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Minimum age
12
Years
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Maximum age
No limit
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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. LDL-C level <70 mg/dL (1.81 mmol/L) at the screening visit
2. Background medical Lipid Modifying Therapy (LMT) (if applicable) that has not been stable before the screening visit
3. Lipid-apheresis schedule /apheresis settings (if applicable) that have not been stable for at least 8 weeks before the screening visit
4. Use of nutraceuticals or over-the-counter therapies known to affect lipids, at a dose/amount that has not been stable for at least 4 weeks prior to the screening visit
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 (HbA1c >9%) diabetes
7. History of a MI, unstable angina leading to hospitalization, coronary artery bypass graft surgery, percutaneous coronary intervention, uncontrolled cardiac arrhythmia, carotid surgery or stenting, stroke, transient ischemic attack, valve replacement surgery, carotid revascularization, endovascular procedure or surgical intervention for peripheral vascular disease within 3 months prior to the screening visit
8. Pregnant or breastfeeding women
9. Sexually active women of child bearing potential (WOCBP), who are unwilling to practice a highly effective birth control method prior to the initial dose, during the study, and for 24 weeks after the last dose of study drug
10. Men who are sexually active with women of child bearing potential (WOCBP) and are unwilling to consistently use condoms during the study drug treatment period and for 24 weeks after the last dose of study drug regardless of vasectomy status
Note: Other protocol defined inclusion/exclusion criteria may apply.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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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
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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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
18/01/2018
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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
17/03/2020
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Sample size
Target
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Accrual to date
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Final
65
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Recruitment in Australia
Recruitment state(s)
NSW,WA
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Recruitment hospital [1]
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Regeneron Research Site - Camperdown
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Recruitment hospital [2]
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Regeneron Research Site - Perth
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Recruitment postcode(s) [1]
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2050 - Camperdown
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Recruitment postcode(s) [2]
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6000 - Perth
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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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Florida
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United States of America
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State/province [2]
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Massachusetts
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United States of America
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State/province [3]
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New York
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United States of America
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Ohio
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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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Oregon
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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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Texas
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Country [7]
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Austria
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State/province [7]
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Innsbruck
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Canada
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State/province [8]
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Quebec
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France
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Cedex
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France
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State/province [10]
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Marseille
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Greece
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State/province [11]
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Ioannina
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Country [12]
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Greece
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State/province [12]
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Athens
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Country [13]
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Italy
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State/province [13]
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Napoli
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Country [14]
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Japan
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State/province [14]
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Fukuoka
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Country [15]
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Japan
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State/province [15]
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Hyogo
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Country [16]
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Japan
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State/province [16]
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Ishikawa
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Country [17]
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Japan
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State/province [17]
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Osaka
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Country [18]
0
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Netherlands
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State/province [18]
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Amsterdam
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Country [19]
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Netherlands
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State/province [19]
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Rotterdam
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Country [20]
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South Africa
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State/province [20]
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Johannesburg
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Country [21]
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Ukraine
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State/province [21]
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Ivano-Frankivs'k
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Country [22]
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Ukraine
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State/province [22]
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Kharkiv
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Country [23]
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Ukraine
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State/province [23]
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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 of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab intravenously (IV) in comparison to placebo after 24 weeks in patients with homozygous familial hypercholesterolemia (HoFH). The secondary objectives of the study are to evaluate the effect of evinacumab IV on other lipid parameters, evaluate the effect of evinacumab on LDL-C goal attainment, assess the effect of evinacumab on eligibility for apheresis (using German and US apheresis criteria), evaluate the safety and tolerability of evinacumab in patients with HoFH, assess the pharmacokinetics (PK) of evinacumab in patients with HoFH and evaluate the potential development of anti-evinacumab antibodies.
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Trial website
https://clinicaltrials.gov/study/NCT03399786
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Trial related presentations / publications
Raal FJ, Rosenson RS, Reeskamp LF, Hovingh GK, Kastelein JJP, Rubba P, Ali S, Banerjee P, Chan KC, Gipe DA, Khilla N, Pordy R, Weinreich DM, Yancopoulos GD, Zhang Y, Gaudet D; ELIPSE HoFH Investigators. Evinacumab for Homozygous Familial Hypercholesterolemia. N Engl J Med. 2020 Aug 20;383(8):711-720. doi: 10.1056/NEJMoa2004215.
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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
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/86/NCT03399786/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/86/NCT03399786/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/NCT03399786
Download to PDF