Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

April 4, 2025 updated by: Regeneron Pharmaceuticals

An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

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

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
116

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Camperdown, New South Wales, Australia, 2050
        • Clinical Trial Site
    • Western Australia
      • Perth, Western Australia, Australia, 6000
        • Clinical Trial Site
  • Austria
      • Innsbruck, Austria, 6020
        • Regeneron Study Site
    • Wien
      • Vienna, Wien, Austria, 1090
        • Regeneron Study Site
  • Canada
      • Quebec, Canada, G1V 4W2
        • Clinical Trial Site
    • Quebec
      • Chicoutimi, Quebec, Canada, G7H 7K9
        • Clinical Trial Site
  • Czechia
    • Kateřinská
      • Praha 2, Kateřinská, Czechia, 121 08
        • Regeneron Study Site
  • France
    • Cedex
      • Dijon, Cedex, France, 21079
        • Clinical Trial Site
    • Cedex 13
      • Paris, Cedex 13, France, 75651
        • Clinical Trial Site
    • Cedex 5
      • Marseille, Cedex 5, France, 13385
        • Clinical Trial Site
  • Greece
      • Ioannina, Greece, 45500
        • Clinical Trial Site
    • Athens
      • Kallithea, Athens, Greece, 17674
        • Regeneron Study Site
  • Italy
    • Campania
      • Napoli, Campania, Italy, 80131
        • Clinical Trial Site
    • Rome
      • Roma, Rome, Italy, 161
        • Clinical Trial Site
  • Japan
    • Aichi
      • Obu, Aichi, Japan, 474-8710
        • Clinical Trial Site
    • Fukuoka
      • Kurume, Fukuoka, Japan, 830-8522
        • Regeneron Study Site
    • Hyogo
      • Nishinomiya, Hyogo, Japan, 662-0918
        • Regeneron Study Site
    • Ishikawa
      • Kanazawa, Ishikawa, Japan, 920-8641
        • Regeneron Study Site
    • Osaka
      • Kita-ku, Osaka, Japan, 530-0001
        • Regeneron Study Site
      • Suita, Osaka, Japan, 564-8565
        • Regeneron Study Site
      • Suita, Osaka, Japan, 565-0871
        • Regeneron Study Site
  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
        • Clinical Trial Site
  • South Africa
    • Gauteng
      • Johannesburg, Gauteng, South Africa, 2000
        • Regeneron Study Site
    • Western Cape
      • Cape Town, Western Cape, South Africa, 7925
        • Regeneron Study Site
  • Ukraine
      • Ivano-Frankivsk, Ukraine, 76075
        • Clinical Trial Site
      • Kharkiv, Ukraine, 61039
        • Clinical Trial Site #1
      • Kharkiv, Ukraine, 61176
        • Clinical Trial Site #2
      • Kyiv, Ukraine, 02660
        • Clinical Trial Site
      • Kyiv, Ukraine, 3680
        • Rgeneron Study Site
  • United States
    • California
      • Los Angeles, California, United States, 90048
        • Clinical Trial Site
    • Florida
      • Boca Raton, Florida, United States, 33434
        • Clinical Trial Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Clinical Trial Site
    • New York
      • New York, New York, United States, 10029
        • Clinical Trial Site
    • Ohio
      • Cincinnati, Ohio, United States, 45227
        • Clinical Trial Site
    • Oregon
      • Portland, Oregon, United States, 97239
        • Clinical Trial Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Clinical Trial Site
    • Texas
      • Dallas, Texas, United States, 75226
        • Clinical Trial Site
      • Dallas, Texas, United States, 75390
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

8 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  1. Completion of the parent study in which they participated
  2. Able to understand and complete study-related questionnaires

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: evinacumab
Drug: evinacumab
Intravenous (IV) administration
Other Names:
  • REGN1500
  • EVKEEZA®

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Up to Week 216
Time Frame: Up to 216 weeks
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.
Up to 216 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) Over Time
Time Frame: Up to 120 weeks
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.
Up to 120 weeks
Absolute Change in LDL-C Over Time
Time Frame: Up to 120 weeks
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.
Up to 120 weeks
Percent Change in Apolipoprotein B (Apo B) Over Time
Time Frame: Up to 120 weeks
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.
Up to 120 weeks
Absolute Change in Apo B Over Time
Time Frame: Up to 120 weeks
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.
Up to 120 weeks
Percent Change in Non-High-Density Lipoprotein Cholesterol (HDL-C) Over Time
Time Frame: Up to 120 weeks
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.
Up to 120 weeks
Absolute Change in Non-HDL-C Over Time
Time Frame: Up to 120 weeks
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.
Up to 120 weeks
Percent Change in Total Cholesterol (TC) Over Time
Time Frame: Up to 120 weeks
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.
Up to 120 weeks
Absolute Change in TC Over Time
Time Frame: Up to 120 weeks
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.
Up to 120 weeks
Percent Change in Triglycerides (TGs) Over Time
Time Frame: Up to 120 weeks
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.
Up to 120 weeks
Absolute Change in TGs Over Time
Time Frame: Up to 120 weeks
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.
Up to 120 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Regeneron Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Clinical Trial Management, Regeneron Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 13, 2018

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 13, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 13, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 8, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 17, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 24, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 8, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 4, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • R1500-CL-1719
  • 2017-003170-13 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Homozygous Familial Hypercholesterolemia

Clinical Trials on evinacumab

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings