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

December 23, 2010
March 15, 2012
January 2011
November 2011   (final data collection date for primary outcome measure)
Percent change of low-density lipoprotein cholesterol (LDL-C) from baseline to week 12 [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT01266876 on ClinicalTrials.gov Archive Site
  • 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 ]
Same as current
Not Provided
Not Provided
 
Study of the Safety and Efficacy of REGN727(SAR236553) in Patients With HeFH Hypercholesterolemia
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 patients diagnosed with heterozygous familial hypercholesterolemia (heFH)

Not Provided
Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Hypercholesterolemia
  • Biological: REGN727 (SAR236553)
    Dose 1 every 4 weeks
  • Biological: REGN727 (SAR236553)
    Dose 2 every 4 weeks
  • Biological: REGN727 (SAR236553)
    Dose 3 every 4 weeks
  • Biological: REGN727 (SAR236553)
    Dose 3 every 2 weeks
  • Other: Placebo
    Every 2 weeks
  • Experimental: Group 1
    Intervention: Biological: REGN727 (SAR236553)
  • Experimental: Group 2
    Intervention: Biological: REGN727 (SAR236553)
  • Experimental: Group 3
    Intervention: Biological: REGN727 (SAR236553)
  • Experimental: Group 4
    Intervention: Biological: REGN727 (SAR236553)
  • Placebo Comparator: Group 5
    Intervention: Other: Placebo
Stein EA, Gipe D, Bergeron J, Gaudet D, Weiss R, Dufour R, Wu R, Pordy R. Effect of a monoclonal antibody to PCSK9, REGN727/SAR236553, to reduce low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolaemia on stable statin dose with or without ezetimibe therapy: a phase 2 randomised controlled trial. Lancet. 2012 Jul 7;380(9836):29-36. Epub 2012 May 26.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
77
November 2011
November 2011   (final data collection date for primary outcome measure)

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
Both
18 Years to 75 Years
No
Contact information is only displayed when the study is recruiting subjects
United States,   Canada,   Netherlands
 
NCT01266876
R727-CL-1003
Yes
Regeneron Pharmaceuticals
Regeneron Pharmaceuticals
Sanofi
Study Director: Dan Gipe, MD Regeneron Pharmaceuticals
Regeneron Pharmaceuticals
March 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP