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Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03399786
Recruitment Status : Completed
First Posted : January 16, 2018
Last Update Posted : June 1, 2020
Sponsor:
Information provided by (Responsible Party):
Regeneron Pharmaceuticals

Brief Summary:
The primary objective of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab intravenously (IV) in comparison to placebo after 24 weeks in patients with homozygous familial hypercholesterolemia (HoFH). The secondary objectives of the study are to evaluate the effect of evinacumab IV on other lipid parameters, evaluate the effect of evinacumab on LDL-C goal attainment, assess the effect of evinacumab on eligibility for apheresis (using German and US apheresis criteria), evaluate the safety and tolerability of evinacumab in patients with HoFH, assess the pharmacokinetics (PK) of evinacumab in patients with HoFH and evaluate the potential development of anti-evinacumab antibodies.

Condition or disease Intervention/treatment Phase
Homozygous Familial Hypercholesterolemia Drug: evinacumab Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 65 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
Actual Study Start Date : January 18, 2018
Actual Primary Completion Date : June 10, 2019
Actual Study Completion Date : March 17, 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: evinacumab Drug: evinacumab
IV administration of evinacumab
Other Name: REGN1500

Experimental: Placebo Drug: Placebo
IV administration of placebo




Primary Outcome Measures :
  1. Percent change in calculated LDL-C from baseline to week 24 [ Time Frame: Week 24 ]

Secondary Outcome Measures :
  1. Percent change in Apolipoprotein B (Apo B) from baseline to week 24 [ Time Frame: Week 24 ]
  2. Percent change in non-High-density lipoprotein cholesterol (HDL-C) from baseline to week 24 [ Time Frame: Week 24 ]
  3. Percent change in Total cholesterol (TC) from baseline to week 24 [ Time Frame: Week 24 ]
  4. Proportion of patients with ≥30% reduction in calculated LDL-C [ Time Frame: Week 24 ]
  5. Proportion of patients with ≥50% reduction in calculated LDL-C [ Time Frame: Week 24 ]
  6. Proportion of patients with LDL-C <100 mg/dL [2.59 mmol/L] [ Time Frame: Week 24 ]
  7. Change in calculated LDL-C from baseline to week 24 [ Time Frame: Week 24 ]
  8. Proportion of patients who meet European Union (EU) apheresis eligibility criteria (see German Apheresis Working Group) [ Time Frame: Week 24 ]
  9. Proportion of patients who meet United States (US) apheresis eligibility criteria (see US [National Lipid Association] Lipid Apheresis Criteria) [ Time Frame: Week 24 ]
  10. Percent change in Triglyceride (TG) from baseline to week 24 [ Time Frame: Week 24 ]
  11. Change in Apo B from baseline to week 24 [ Time Frame: Week 24 ]
  12. Change in non-HDL-C from baseline to week 24 [ Time Frame: Week 24 ]
  13. Change in TC from baseline to week 24 [ Time Frame: Week 24 ]
  14. Percent change in lipoprotein a [Lp(a)] from baseline to week 24 [ Time Frame: Week 24 ]
  15. Proportion of patients with LDL-C <70 mg/dL [1.81 mmol/L] [ Time Frame: Week 24 ]
  16. Percent change in apolipoprotein CIII (Apo CIII) from baseline to week 24 [ Time Frame: Week 24 ]
  17. Total evinacumab concentration in serum [ Time Frame: Week 24 ]
  18. Incidence of anti-drug antibody (ADA) [ Time Frame: Week 24 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  1. Diagnosis of functional HoFH
  2. If undergoing LDL apheresis, must have initiated LDL apheresis at least 3 months prior to screening and must have been on a stable weekly or every other week schedule and/or stable settings for at least 8 weeks
  3. Willing to consistently maintain his/her usual low fat or heart-healthy diet for the duration of the study

Key Exclusion Criteria:

  1. LDL-C level <70 mg/dL (1.81 mmol/L) at the screening visit
  2. Background medical Lipid Modifying Therapy (LMT) (if applicable) that has not been stable before the screening visit
  3. Lipid-apheresis schedule /apheresis settings (if applicable) that have not been stable for at least 8 weeks before the screening visit
  4. Use of nutraceuticals or over-the-counter therapies known to affect lipids, at a dose/amount that has not been stable for at least 4 weeks prior to the screening visit
  5. Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins
  6. Newly diagnosed (within 3 months prior to randomization visit) diabetes mellitus or poorly controlled (HbA1c >9%) diabetes
  7. History of a MI, unstable angina leading to hospitalization, coronary artery bypass graft surgery, percutaneous coronary intervention, uncontrolled cardiac arrhythmia, carotid surgery or stenting, stroke, transient ischemic attack, valve replacement surgery, carotid revascularization, endovascular procedure or surgical intervention for peripheral vascular disease within 3 months prior to the screening visit
  8. Pregnant or breastfeeding women
  9. Sexually active women of child bearing potential (WOCBP), who are unwilling to practice a highly effective birth control method prior to the initial dose, during the study, and for 24 weeks after the last dose of study drug
  10. Men who are sexually active with women of child bearing potential (WOCBP) and are unwilling to consistently use condoms during the study drug treatment period and for 24 weeks after the last dose of study drug regardless of vasectomy status

Note: Other protocol defined inclusion/exclusion criteria may apply.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03399786


Locations
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United States, Florida
Regeneron Research Site
Boca Raton, Florida, United States, 33434
United States, Massachusetts
Regeneron Research Site
Boston, Massachusetts, United States, 02114
United States, New York
Regeneron Research Site
New York, New York, United States, 10029
United States, Ohio
Regeneron Research Site
Cincinnati, Ohio, United States, 45227
United States, Oregon
Regeneron Research Site
Portland, Oregon, United States, 97239
United States, Texas
Regeneron Research Site
Dallas, Texas, United States, 78226
Australia, New South Wales
Regeneron Research Site
Camperdown, New South Wales, Australia, 2050
Australia, Western Australia
Regeneron Research Site
Perth, Western Australia, Australia, 6000
Austria
Regeneron Research Site
Innsbruck, Austria, 6020
Canada, Quebec
Regeneron Research Site
Chicoutimi, Quebec, Canada, G7H 7K9
Regeneron Research Site
Québec, Quebec, Canada, G1V 4W2
France
Regeneron Research Site
Paris, Cedex, France, 75651
Regeneron Research Site
Marseille, France, 13385
Greece
Regeneron Research Site
Ioánnina, Ioannina, Greece, 45500
Regeneron Research Site
Athens, Greece, 17674
Italy
Regeneron Research Site # 2
Napoli, Italy, 80131
Japan
Regeneron Research Site
Kurume, Fukuoka, Japan, 830-8522
Regeneron Research Site
Nishinomiya, Hyogo, Japan, 662-0918
Regeneron Research Site
Kanazawa, Ishikawa, Japan, 920-8641
Regeneron Research Site #3
Suita, Osaka, Japan, 565-0871
Regeneron Research Site
Suita, Osaka, Japan, 565-8565
Regeneron Research Site
Osaka, Japan, 530-0001
Netherlands
Regeneron Research Site
Amsterdam, Netherlands, 1105 AZ
Regeneron Research Site
Rotterdam, Netherlands, 3045 PM
South Africa
Regeneron Research Site
Parktown, Johannesburg, South Africa, 2000
Ukraine
Regeneron Research Site
Ivano-Frankivs'k, Ukraine, 76075
Regeneron Research Site
Kharkiv, Ukraine, 61039
Regeneron Research Site #2
Kharkiv, Ukraine, 61176
Regeneron Research Site #2
Kyiv, Ukraine, 02660
Regeneron Research Site
Kyiv, Ukraine, 03680
Sponsors and Collaborators
Regeneron Pharmaceuticals
Investigators
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Study Director: Clinical Trial Management Regeneron Pharmaceuticals
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03399786    
Other Study ID Numbers: R1500-CL-1629
2017-001388-19 ( EudraCT Number )
First Posted: January 16, 2018    Key Record Dates
Last Update Posted: June 1, 2020
Last Verified: May 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Regeneron Pharmaceuticals:
HoFH
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type II
Hypercholesterolemia
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias