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

May 1, 2023 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

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

116

Phase

  • 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

Ages Eligible for Study

10 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
Intervention / Treatment
Experimental: evinacumab
Drug: evinacumab
Intravenous (IV) administration
Other Names:
  • REGN1500
  • EVKEEZA®

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence and severity of treatment-emergent adverse events (TEAEs)
Time Frame: Up to 192 weeks
Up to 192 weeks

Secondary Outcome Measures

Outcome Measure
Time Frame
Percent change in Low-Density Lipoprotein Cholesterol (LDL-C) over time
Time Frame: Up to 192 weeks
Up to 192 weeks
Absolute change in LDL-C over time
Time Frame: Up to 192 weeks
Up to 192 weeks
Percent change in Apolipoprotein B (Apo B) over time
Time Frame: Up to 192 weeks
Up to 192 weeks
Absolute change in Apo B over time
Time Frame: Up to 192 weeks
Up to 192 weeks
Percent change in non-High-Density Lipoprotein Cholesterol (HDL-C) over time
Time Frame: Up to 192 weeks
Up to 192 weeks
Absolute change in non-HDL-C over time
Time Frame: Up to 192 weeks
Up to 192 weeks
Percent change in Total Cholesterol (TC) over time
Time Frame: Up to 192 weeks
Up to 192 weeks
Absolute change in TC over time
Time Frame: Up to 192 weeks
Up to 192 weeks
Percent change in Triglycerides (TGs) over time
Time Frame: Up to 192 weeks
Up to 192 weeks
Absolute change in TGs over time
Time Frame: Up to 192 weeks
Up to 192 weeks
Presence of anti-evinacumab antibodies
Time Frame: Up to 192 weeks
Up to 192 weeks

Collaborators and Investigators

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

Sponsor

Regeneron Pharmaceuticals

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)

March 13, 2018

Primary Completion (Actual)

April 13, 2023

Study Completion (Actual)

April 13, 2023

Study Registration Dates

First Submitted

January 8, 2018

First Submitted That Met QC Criteria

January 17, 2018

First Posted (Actual)

January 24, 2018

Study Record Updates

Last Update Posted (Actual)

May 3, 2023

Last Update Submitted That Met QC Criteria

May 1, 2023

Last Verified

April 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID 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.

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