Skip to main content
OpenTrials
Completed

NCT Number: NCT03409744

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

Completed

Looking for future studies?

Notify Me

Key information

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Clinical Trial Site, Camperdown, New South Wales, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

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

Key Exclusion Criteria:

  • Significant protocol deviation in the parent study based on the investigator's judgment, such as non-compliance by the patient
  • Concomitant medications that have not been stable prior to the baseline visit
  • Adverse event leading to permanent discontinuation from parent study
  • 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
  • Member of the clinical site study team and/or his/her immediate family
  • Pregnant or breastfeeding women
  • 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
  • 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

Treatment and study plan

Evinacumab

Drug

Intravenous (IV) administration

Other names: REGN1500, EVKEEZA®

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

  8. 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.

  9. 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.

  10. 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.

Sponsors and collaborators

Lead sponsor

Regeneron Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2018
Primary completion
2023
Study completion
2023
First posted
Jan 24, 2018
Registry last updated
Apr 8, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.