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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03409744
Registration number
NCT03409744
Ethics application status
Date submitted
8/01/2018
Date registered
24/01/2018
Date last updated
30/04/2024
Titles & IDs
Public title
Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
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Scientific title
An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
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Secondary ID [1]
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2017-003170-13
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Secondary ID [2]
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R1500-CL-1719
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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 -
Treatment: Drugs: evinacumab
Intravenous (IV) administration
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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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Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Up to Week 216
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Assessment method [1]
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The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
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Timepoint [1]
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Up to 216 weeks
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Secondary outcome [1]
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Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) Over Time
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Assessment method [1]
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The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
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Timepoint [1]
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Up to 120 weeks
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Secondary outcome [2]
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Absolute Change in LDL-C Over Time
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Assessment method [2]
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The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
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Timepoint [2]
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Up to 120 weeks
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Secondary outcome [3]
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Percent Change in Apolipoprotein B (Apo B) Over Time
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Assessment method [3]
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The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
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Timepoint [3]
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Up to 120 weeks
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Secondary outcome [4]
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Absolute Change in Apo B Over Time
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Assessment method [4]
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The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
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Timepoint [4]
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Up to 120 weeks
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Secondary outcome [5]
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Percent Change in Non-High-Density Lipoprotein Cholesterol (HDL-C) Over Time
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Assessment method [5]
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The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
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Timepoint [5]
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Up to 120 weeks
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Secondary outcome [6]
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Absolute Change in Non-HDL-C Over Time
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Assessment method [6]
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The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
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Timepoint [6]
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Up to 120 weeks
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Secondary outcome [7]
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Percent Change in Total Cholesterol (TC) Over Time
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Assessment method [7]
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The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
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Timepoint [7]
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Up to 120 weeks
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Secondary outcome [8]
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Absolute Change in TC Over Time
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Assessment method [8]
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The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
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Timepoint [8]
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Up to 120 weeks
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Secondary outcome [9]
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Percent Change in Triglycerides (TGs) Over Time
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Assessment method [9]
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The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
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Timepoint [9]
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Up to 120 weeks
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Secondary outcome [10]
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Absolute Change in TGs Over Time
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Assessment method [10]
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The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
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Timepoint [10]
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Up to 120 weeks
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Eligibility
Key inclusion criteria
Key
1. Completion of the parent study in which they participated
2. Able to understand and complete study-related questionnaires
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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. Significant protocol deviation in the parent study based on the investigator's
judgment, such as non-compliance by the patient
2. Concomitant medications that have not been stable prior to the baseline visit
3. Adverse event leading to permanent discontinuation from parent study
4. Any new condition or worsening of an existing condition, which in the opinion of the
investigator would make the patient unsuitable for enrollment, or could interfere with
the patient participating in or completing the study
5. Member of the clinical site study team and/or his/her immediate family
6. Pregnant or breastfeeding women
7. Women of childbearing potential who are unwilling to practice highly effective
contraception prior to the initial dose/start of the first treatment, during the
study, and for at least 24 weeks after the last dose of study drug
8. Men who are sexually active with women of childbearing potential and are unwilling to
use the following forms of medically acceptable birth control during the study drug
treatment period and for 24 weeks after the last injection of study drug: vasectomy
with medical assessment of surgical success OR consistent use of a condom.
Note: Other protocol defined Inclusion/Exclusion criteria apply
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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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
13/03/2018
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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
13/04/2023
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Sample size
Target
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Accrual to date
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Final
116
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Recruitment in Australia
Recruitment state(s)
NSW,WA
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Recruitment hospital [1]
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Clinical Trial Site - Camperdown
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Recruitment hospital [2]
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Clinical Trial 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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California
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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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Massachusetts
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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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New York
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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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Ohio
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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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Oregon
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Country [7]
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United States of America
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Pennsylvania
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Country [8]
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United States of America
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State/province [8]
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Texas
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Country [9]
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Austria
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State/province [9]
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Wien
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Country [10]
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Austria
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State/province [10]
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Innsbruck
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Canada
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State/province [11]
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Quebec
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Country [12]
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Czechia
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State/province [12]
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Katerinská
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Country [13]
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France
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State/province [13]
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Cedex 13
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Country [14]
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France
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State/province [14]
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Cedex 5
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Country [15]
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France
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State/province [15]
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Cedex
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Country [16]
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Greece
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State/province [16]
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Athens
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Country [17]
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Greece
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State/province [17]
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Ioannina
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Country [18]
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Italy
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State/province [18]
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Campania
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Country [19]
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Italy
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State/province [19]
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Rome
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Country [20]
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Japan
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State/province [20]
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Aichi
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Country [21]
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Japan
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State/province [21]
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Fukuoka
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Country [22]
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Japan
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State/province [22]
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Hyogo
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Japan
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State/province [23]
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Ishikawa
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Country [24]
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Japan
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State/province [24]
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Osaka
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Country [25]
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Netherlands
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State/province [25]
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Amsterdam
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Country [26]
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South Africa
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State/province [26]
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Gauteng
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Country [27]
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South Africa
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State/province [27]
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Western Cape
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Country [28]
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Ukraine
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State/province [28]
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Ivano-Frankivsk
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Country [29]
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Ukraine
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State/province [29]
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Kharkiv
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Country [30]
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Ukraine
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State/province [30]
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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 objectives of the study are:
- To evaluate the long-term safety and tolerability of evinacumab in patients with
Homozygous Familial Hypercholesterolemia (HoFH)
- To evaluate the long-term safety and tolerability of evinacumab in adolescent patients
with HoFH
The secondary objectives of the study are:
- To evaluate the effect of evinacumab on lipid parameters in patients with HoFH
- To evaluate the effect of evinacumab on lipid parameters in adolescent patients with
HoFH
- To evaluate the potential development of anti-evinacumab antibodies
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03409744
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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
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03409744
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