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OpenTrials
Completed

NCT Number: NCT01266876

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

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

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Key information

Conditions

Hypercholesterolemia Dyslipidemias Hyperlipidemias Lipid Metabolism Disorders Metabolic Diseases Nutritional and Metabolic Diseases

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Winnipeg, Manitoba, Canada

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Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

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

Key Exclusion Criteria:

  • Participants with homozygous FH (clinically or by previous genotyping)
  • 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
  • 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
  • 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)
  • Use of thyroid medications (except for replacement therapy which has been stable for at least 12 weeks before the full screening visit)
  • Fasting serum TG >350 mg/dL screening
  • LDL apheresis within 12 months before screening

Treatment and study plan

Alirocumab

Drug

Alirocumab two SC injections in the abdomen only.

Other names: REGN727/SAR236553

Placebo

Drug

Placebo two SC injections in the abdomen only.

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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..

  7. 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.

  8. 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)

  9. 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)

  10. 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.

  11. 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.

  12. 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.

  13. 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.

  14. 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.

  15. 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.

  16. 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.

  17. 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)

  18. 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)

Sponsors and collaborators

Lead sponsor

Regeneron Pharmaceuticals

Industry

Collaborators

  • Sanofi

Registry information

Official study title

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

Important dates

Study start
2011
Primary completion
2011
Study completion
2011
First posted
Dec 24, 2010
Registry last updated
Sep 22, 2015

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.

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