ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 20 of 73 for:    Recruiting, Not yet recruiting, Available Studies | "Hypercholesterolemia"

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03399786
Recruitment Status : Recruiting
First Posted : January 16, 2018
Last Update Posted : June 25, 2018
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

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 57 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
Estimated Primary Completion Date : April 5, 2020
Estimated Study Completion Date : April 5, 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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years and older   (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. Sexually active men who are unwilling to use forms of medically acceptable birth control during the study drug treatment period and for 24 weeks after the last dose of study drug

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


Contacts
Contact: Clinical Trials Administrator 844-734-6643 clinicaltrials@regeneron.com

  Show 38 Study Locations
Sponsors and Collaborators
Regeneron Pharmaceuticals
Investigators
Study Director: Clinical Trial Management Regeneron Pharmaceuticals

Responsible Party: Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03399786     History of Changes
Other Study ID Numbers: R1500-CL-1629
2017-001388-19 ( EudraCT Number )
First Posted: January 16, 2018    Key Record Dates
Last Update Posted: June 25, 2018
Last Verified: June 2018

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:
Hypercholesterolemia
Hyperlipoproteinemia Type II
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias