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

NCT03409744 | Completed Phase 3 Results DMC FDA Drug

• 2 other identifiers: R1500-CL-17192017-003170-13
• Study Type: interventional
• Target: 116
• Locations: 11 countries
• Sites: 36

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

Conditions

Homozygous Familial Hypercholesterolemia

Keywords

HoFH

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Up to Week 216

  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 Outcomes (10)

• Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) Over Time

  Up to 120 weeks

• Absolute Change in LDL-C Over Time

  Up to 120 weeks

• Percent Change in Apolipoprotein B (Apo B) Over Time

  Up to 120 weeks

• Absolute Change in Apo B Over Time

  Up to 120 weeks

• Percent Change in Non-High-Density Lipoprotein Cholesterol (HDL-C) Over Time

  Up to 120 weeks

Study Arms (1)

evinacumab

EXPERIMENTAL

Drug: evinacumab

Interventions

evinacumab DRUG

Intravenous (IV) administration

Also known as: REGN1500, EVKEEZA®

evinacumab

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Regeneron Pharmaceuticals: lead

Study Sites (38)

Clinical Trial Site

Los Angeles, California, 90048, United States

Location

Clinical Trial Site

Boca Raton, Florida, 33434, United States

Location

Clinical Trial Site

Boston, Massachusetts, 02114, United States

Location

Clinical Trial Site

New York, New York, 10029, United States

Location

Clinical Trial Site

Cincinnati, Ohio, 45227, United States

Location

Clinical Trial Site

Portland, Oregon, 97239, United States

Location

Clinical Trial Site

Philadelphia, Pennsylvania, 19104, United States

Location

Clinical Trial Site

Dallas, Texas, 75226, United States

Location

Clinical Trial Site

Dallas, Texas, 75390, United States

Location

Clinical Trial Site

Camperdown, New South Wales, 2050, Australia

Location

Clinical Trial Site

Perth, Western Australia, 6000, Australia

Location

Regeneron Study Site

Vienna, Vienna, 1090, Austria

Location

Regeneron Study Site

Innsbruck, 6020, Austria

Location

Clinical Trial Site

Chicoutimi, Quebec, G7H 7K9, Canada

Location

Clinical Trial Site

Québec, G1V 4W2, Canada

Location

Regeneron Study Site

Prague, Kateřinská, 121 08, Czechia

Location

Clinical Trial Site

Paris, Cedex 13, 75651, France

Location

Clinical Trial Site

Marseille, Cedex 5, 13385, France

Location

Clinical Trial Site

Dijon, Cedex, 21079, France

Location

Regeneron Study Site

Kallithea, Athens, 17674, Greece

Location

Clinical Trial Site

Ioannina, 45500, Greece

Location

Clinical Trial Site

Napoli, Campania, 80131, Italy

Location

Clinical Trial Site

Roma, Rome, 161, Italy

Location

Clinical Trial Site

Ōbu, Aichi-ken, 474-8710, Japan

Location

Regeneron Study Site

Kurume, Fukuoka, 830-8522, Japan

Location

Regeneron Study Site

Nishinomiya, Hyōgo, 662-0918, Japan

Location

Regeneron Study Site

Kanazawa, Ishikawa-ken, 920-8641, Japan

Location

Regeneron Study Site

Kita-ku, Osaka, 530-0001, Japan

Location

Regeneron Study Site

Suita, Osaka, 564-8565, Japan

Location

Regeneron Study Site

Suita, Osaka, 565-0871, Japan

Location

Clinical Trial Site

Amsterdam, 1105 AZ, Netherlands

Location

Regeneron Study Site

Johannesburg, Gauteng, 2000, South Africa

Location

Regeneron Study Site

Cape Town, Western Cape, 7925, South Africa

Location

Clinical Trial Site

Ivano-Frankivsk, 76075, Ukraine

Location

Clinical Trial Site #1

Kharkiv, 61039, Ukraine

Location

Clinical Trial Site #2

Kharkiv, 61176, Ukraine

Location

Clinical Trial Site

Kyiv, 02660, Ukraine

Location

Rgeneron Study Site

Kyiv, 3680, Ukraine

Location

Related Links

• A Plain Language Summary is available on TrialSummaries.com

MeSH Terms

Conditions

Homozygous Familial Hypercholesterolemia

Interventions

evinacumab

Condition Hierarchy (Ancestors)

Hyperlipoproteinemia Type II; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hyperlipoproteinemias; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Clinical Trials Administrator
• Organization: Regeneron Pharmaceuticals, Inc.

clinicaltrials@regeneron.com

844-734-6643

Study Officials

• Clinical Trial Management

  Regeneron Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 8, 2018
• First Posted: January 24, 2018
• Study Start: March 13, 2018
• Primary Completion: April 13, 2023
• Study Completion: April 13, 2023
• Last Updated: April 8, 2025
• Results First Posted: April 30, 2024

Record last verified: 2025-04

Locations

NL (1); CA (2); ZA (2); GR (2); AU (2); IT (2); FR (3); UA (5); US (9); CZ (1); JP (7)