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A Study to Determine if REGN727(SAR236553) Will Have an Effect of Circulating Lipids in Patients With Gain-of-function (GOF) Mutations in Their PCSK9 Gene

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Sanofi
Information provided by (Responsible Party):
Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01604824
First received: May 22, 2012
Last updated: January 23, 2013
Last verified: January 2013
History of Changes
  Purpose

The purpose of this study is to assess the pharmacodynamic (PD) effect of REGN727 on serum low-density lipoprotein cholesterol (LDL-C) during 14 weeks of subcutaneously (SC) administered REGN727 in patients with autosomal dominant hypercholesterolemia (ADH) and gain-of-function mutations (GOFm) in 1 or both alleles of the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene.


Condition Intervention Phase
Hypercholesterolemia
 Drug: REGN727 (SAR236553)
 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2 Pilot Study With a Randomized Double-Blind Treatment Phase to Evaluate the Pharmacodynamics and Safety of REGN727 in Patients With Autosomal Dominant Hypercholesterolemia and Gain-of-Function Mutations in 1 or Both Alleles of the PCSK9 Gene

Resource links provided by NLM:

MedlinePlus related topics: Cholesterol
U.S. FDA Resources

Further study details as provided by Regeneron Pharmaceuticals:

Primary Outcome Measures:
  • Percent change in serum LDL-C [ Time Frame: Day 1(baseline) to Day 15 ] [ Designated as safety issue: No ]
    Percent change in Low density lipoprotein cholesterol (LDL-C) from day 1 (baseline) to day 15 for group A compared to group B.


Secondary Outcome Measures:
  • Number of TEAEs [ Time Frame: Day 1 to Day 155 ] [ Designated as safety issue: Yes ]
    Incidence and severity of treatment-emergent adverse events (TEAEs) in patients treated with REGN727 reported between the first dose of REGN727 (day 1) and the end-of-study (EOS) visit (day 155)

  • Percent change in ApoB100 [ Time Frame: Day 1 to Day 15 ] [ Designated as safety issue: No ]
    Percent change in Apolipoprotein (Apo) B100 from day 1 (baseline) to day 15 for group A compared to group B

  • Percent change in non-HDL-C [ Time Frame: Day 1 to Day 15 ] [ Designated as safety issue: No ]
    Percent change in non-HDL-C (High Density Lipoprotein Cholesterol) from day 1 (baseline) to day 15 for group A compared to group B

  • Percent change in total cholesterol [ Time Frame: Day 1 to Day 15 ] [ Designated as safety issue: No ]
    Percent change in total cholesterol from day 1 (baseline) to day 15 for group A compared to group B


Enrollment: 13
Study Start Date: September 2012
Estimated Study Completion Date: July 2013
Estimated Primary Completion Date: July 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Group A
Regimen 1
 Drug: REGN727 (SAR236553)
Patients in Group A will receive REGN727 (SAR236553) or placebo as per Regimen 1
Experimental: Group B
Regimen 2
 Drug: REGN727 (SAR236553)
Patients in Group B will receive REGN727 (SAR236553) or placebo as per Regimen 2

  Eligibility

Ages Eligible for Study:   18 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Inclusion criteria include, but are not limited to the following:

  1. Between the ages of 18 and 70 years, inclusive
  2. Plasma LDL-Cholesterol levels greater than or equal to 70 mg/dL at the screening visit

Exclusion Criteria:

Exclusion criteria include, but are not limited to the following:

  1. Serum Triglycerides greater than 350 mg/dL at the screening visit
  2. Known to be positive for human immunodeficiency virus (HIV), hepatitis B virus, or hepatitis C virus
  3. Pregnant or breast-feeding women.
  4. Sexually active man or woman of childbearing potential who is unwilling to practice adequate contraception during the study
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01604824

Locations
United States, Utah
Salt Lake City, Utah, United States
France
Lille, Cedex, France
Nante, Cedex, France
Paris, France
Sponsors and Collaborators
Regeneron Pharmaceuticals
Sanofi
Investigators
Study Director: Clinical Trial Management Regeneron Pharmaceuticals
  More Information

No publications provided

Responsible Party: Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01604824     History of Changes
Other Study ID Numbers: R727-CL-1018
Study First Received: May 22, 2012
Last Updated: January 23, 2013
Health Authority: United States: Food and Drug Administration
France: Ministry of Health

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 May 16, 2013