Study of the Safety and Efficacy of REGN727/SAR236553 in Patients With HeFH Hypercholesterolemia

August 20, 2015 updated by: Regeneron Pharmaceuticals

A Randomized, Double-Blind, Placebo-Controlled, 12-Week Study of the Safety and Efficacy of REGN727 in Patients With Heterozygous Familial Hypercholesterolemia

The purpose of this study is to assess the efficacy and safety of REGN727/SAR236553 in participants diagnosed with heterozygous familial hypercholesterolemia (heFH)

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
77

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

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

  • Canada
    • Manitoba
      • Winnipeg, Manitoba, Canada
    • Ontario
      • London, Ontario, Canada
    • Quebec
      • Chicoutimi, Quebec, Canada
      • Montreal, Quebec, Canada
      • St. Foy, Quebec, Canada
  • United States
    • Alabama
      • Huntsville, Alabama, United States
    • California
      • Mission Viejo, California, United States
      • Newport Beach, California, United States
    • Connecticut
      • Bridgeport, Connecticut, United States
    • Florida
      • Jacksonville, Florida, United States
      • Miami, Florida, United States
      • Port Orange, Florida, United States
    • Illinois
      • Chicago, Illinois, United States
    • Kansas
      • Kansas City, Kansas, United States
    • Maine
      • Auburn, Maine, United States
      • Biddeford, Maine, United States
    • Missouri
      • St. Louis, Missouri, United States
    • New Hampshire
      • Concord, New Hampshire, United States
    • North Carolina
      • Durham, North Carolina, United States
    • Ohio
      • Cincinnati, Ohio, United States
    • Tennessee
      • Nashville, Tennessee, United States
    • Texas
      • Houston, Texas, United States

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Must meet the World Health Organization criteria for heFH
  2. Participants must be on a stable statin dose, with or without ezetimibe, for at least 6 weeks before screening
  3. Serum LDL-C levels ≥ 100 mg/dL at screening
  4. Willing to follow the NCEP ATPIII TLC diet, or an equivalent diet plan, starting at screening and continuing until the last study visit
  5. A negative urine/serum pregnancy test at each screening visit and start of the study, for women of childbearing potential

Key Exclusion Criteria:

  1. Participants with homozygous FH (clinically or by previous genotyping)

  2. Use of a medication (other than a statin or EZE) to alter serum lipids within 42 days (6 weeks) before screening including, but not limited to:

    • Fibrates
    • Niacin (>500 mg/day)
    • Omega-3 fatty acids (>1000 mg/day of DHA/EPA)
    • Bile acid resins

  3. Use of nutraceuticals or OTC medications that may alter lipid levels that are not stable for at least 6 weeks before screening and are not planned to remain constant throughout the study. Examples include:

    • Omega-3 fatty acids (≤1000 mg/day of DHA/EPA)
    • Niacin (≤500 mg/day)
    • Plant stanols, such as found in Benecol, flax seed oil, psyllium
    • Red yeast rice
  4. Disorders known to influence lipid levels, such as nephrotic syndrome, significant liver disease, Cushing's disease, untreated hypothyroidism (patients on stable thyroid replacement for at least 12 weeks before the full screening visit, who are metabolically euthyroid by thyroid-stimulating hormone (TSH) testing are allowed)
  5. Use of thyroid medications (except for replacement therapy which has been stable for at least 12 weeks before the full screening visit)
  6. Fasting serum TG >350 mg/dL screening
  7. LDL apheresis within 12 months before screening

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: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Placebo Comparator: Placebo
Placebo SC injection Q2W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks.
Drug: Placebo
Placebo two SC injections in the abdomen only.
Experimental: Alirocumab 150 mg Q4W
Alirocumab 150 mg SC injection Q4W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks.
Drug: Alirocumab
Alirocumab two SC injections in the abdomen only.
Other Names:
  • REGN727/SAR236553
Experimental: Alirocumab 200 mg Q4W
Alirocumab 200 mg SC injection Q4W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks.
Drug: Alirocumab
Alirocumab two SC injections in the abdomen only.
Other Names:
  • REGN727/SAR236553
Experimental: Alirocumab 300 mg Q4W
Alirocumab 300 mg SC injection Q4W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks.
Drug: Alirocumab
Alirocumab two SC injections in the abdomen only.
Other Names:
  • REGN727/SAR236553
Experimental: Alirocumab 150 mg Q2W
Alirocumab 150 mg SC injection Q2W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks.
Drug: Alirocumab
Alirocumab two SC injections in the abdomen only.
Other Names:
  • REGN727/SAR236553

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Percent Change From Baseline in Calculated LDL-C at Week 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12 (LOCF)
Calculated LDL-C values were obtained using the Friedewald formula. Baseline adjusted least squares (LS) means and standard errors were estimated using an analysis of covariance (ANCOVA) model including available post-baseline data on treatment from first investigational medicinal product (IMP) injection up to 21 days after last IMP injection (on-treatment analysis). Missing Week 12 data were imputed by last observation carried forward [LOCF] method.
From Baseline to Week 12 (LOCF)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Absolute Change From Baseline in Calculated LDL-C at Week 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12 (LOCF)
Calculated LDL-C value was obtained from Friedewald formula. Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
From Baseline to Week 12 (LOCF)
Percentage of Participants Achieving Calculated LDL-C <100 mg/dL (2.59 mmol/L) at Week 12 - On-treatment Analysis
Time Frame: Week 12 (LOCF)
Calculated LDL-C value was obtained from Friedewald formula.
Week 12 (LOCF)
Percentage of Participants Achieving LDL-C < 70 mg/dL (1.81 mmol/L) at Week 12 - On-treatment Analysis
Time Frame: Week 12 (LOCF)
Calculated LDL-C value was obtained from Friedewald formula.
Week 12 (LOCF)
Percent Change From Baseline in Total Cholesterol at Week 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12 (LOCF)
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in Total Cholesterol at Week 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12 (LOCF)
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
From Baseline to Week 12 (LOCF)
Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Week 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12 (LOCF)
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint..
From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in HDL-C at Week 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12 (LOCF)
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
From Baseline to Week 12 (LOCF)
Percent Change From Baseline in Triglycerides at Week 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12 (LOCF)
Since the assumptions of normal distribution and equality of variances were not verified for the lipid parameter, percent changes were expressed as median (interquartile range)
From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in Triglycerides at Week at 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12 (LOCF)
Since the assumptions of normal distribution and equality of variances were not verified for the lipid parameters, percent changes were expressed as median (interquartile range)
From Baseline to Week 12 (LOCF)
Percent Change From Baseline in Non-HDL-C at Week 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
From Baseline to Week 12
Absolute Change From Baseline in Non-HDL-C at Week 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12 (LOCF)
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
From Baseline to Week 12 (LOCF)
Percent Change From Baseline in Apo Lipoprotein B (Apo-B) at Week 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12 (LOCF)
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in Apo-B at Week 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
From Baseline to Week 12
Percent Change From Baseline in Apolipoprotein - A1 (Apo-A1) at Week 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12 (LOCF)
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in Apo-A1 at Week 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12 (LOCF)
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
From Baseline to Week 12 (LOCF)
Absolute Change in the Ratio ApoB/ApoA-1 From Baseline to Week 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12
Adjusted LS means and standard errors were estimated using the same ANCOVA model as for primary endpoint.
From Baseline to Week 12
Percent Change From Baseline in Lipoprotein(a) at Week 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12 (LOCF)
Since the assumptions of normal distribution and equality of variances were not verified for the lipid parameters, percent changes were expressed as median (interquartile range)
From Baseline to Week 12 (LOCF)
Absolute Change From Baseline in Lipoprotein(a) at Week 12 - On-treatment Analysis
Time Frame: From Baseline to Week 12 (LOCF)
Since the assumptions of normal distribution and equality of variances were not verified for the lipid parameter, percent changes were expressed as median (interquartile range)
From Baseline to Week 12 (LOCF)

Collaborators and Investigators

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

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Regeneron Pharmaceuticals

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Sanofi

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 1, 2011

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 1, 2011

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 1, 2011

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 23, 2010

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 23, 2010

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 24, 2010

Study Record Updates

Last Update Posted (Estimate)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 22, 2015

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 20, 2015

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2015

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • R727-CL-1003

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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