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

This study has been completed.
Sponsor:
Collaborator:
Sanofi
Information provided by (Responsible Party):
Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01266876
First received: December 23, 2010
Last updated: March 15, 2012
Last verified: March 2012
  Purpose

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


Condition Intervention Phase
Hypercholesterolemia
Biological: REGN727 (SAR236553)
Other: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, 12-Week Study of the Safety and Efficacy of REGN727 in Patients With Heterozygous Familial Hypercholesterolemia

Resource links provided by NLM:


Further study details as provided by Regeneron Pharmaceuticals:

Primary Outcome Measures:
  • Percent change of low-density lipoprotein cholesterol (LDL-C) from baseline to week 12 [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Percent change in LDL-C from baseline to each visit [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Immunogenicity of repeated SC doses of REGN727 throughout the course of the study [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Pharmacokinetic (PK) profile of multiple doses of REGN727. [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Safety and tolerability of multiple doses of REGN727 [ Time Frame: 12 weeks ] [ Designated as safety issue: Yes ]

Enrollment: 77
Study Start Date: January 2011
Study Completion Date: November 2011
Primary Completion Date: November 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Group 1 Biological: REGN727 (SAR236553)
Dose 1 every 4 weeks
Experimental: Group 2 Biological: REGN727 (SAR236553)
Dose 2 every 4 weeks
Experimental: Group 3 Biological: REGN727 (SAR236553)
Dose 3 every 4 weeks
Experimental: Group 4 Biological: REGN727 (SAR236553)
Dose 3 every 2 weeks
Placebo Comparator: Group 5 Other: Placebo
Every 2 weeks

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Must meet the WHO criteria for heFH
  2. Patients 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. Patients 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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01266876

  Show 25 Study Locations
Sponsors and Collaborators
Regeneron Pharmaceuticals
Sanofi
Investigators
Study Director: Dan Gipe, MD Regeneron Pharmaceuticals
  More Information

No publications provided by Regeneron Pharmaceuticals

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01266876     History of Changes
Other Study ID Numbers: R727-CL-1003
Study First Received: December 23, 2010
Last Updated: March 15, 2012
Health Authority: United States: Food and Drug Administration
Canada: Health Canada

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

ClinicalTrials.gov processed this record on July 31, 2014