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

NCT ID: NCT01266876

Last Updated: 2015-09-22

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

77 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-01-31

Study Completion Date

2011-11-30

Brief Summary

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

Detailed Description

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Conditions

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Hypercholesterolemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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Placebo

Placebo SC injection Q2W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo two SC injections in the abdomen only.

Alirocumab 150 mg Q4W

Alirocumab 150 mg SC injection Q4W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks.

Group Type EXPERIMENTAL

Alirocumab

Intervention Type DRUG

Alirocumab two SC injections in the abdomen only.

Alirocumab 200 mg Q4W

Alirocumab 200 mg SC injection Q4W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks.

Group Type EXPERIMENTAL

Alirocumab

Intervention Type DRUG

Alirocumab two SC injections in the abdomen only.

Alirocumab 300 mg Q4W

Alirocumab 300 mg SC injection Q4W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks.

Group Type EXPERIMENTAL

Alirocumab

Intervention Type DRUG

Alirocumab two SC injections in the abdomen only.

Alirocumab 150 mg Q2W

Alirocumab 150 mg SC injection Q2W added to stable statin regimen with or without concomitant ezetimibe for 12 weeks.

Group Type EXPERIMENTAL

Alirocumab

Intervention Type DRUG

Alirocumab two SC injections in the abdomen only.

Interventions

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Alirocumab

Alirocumab two SC injections in the abdomen only.

Intervention Type DRUG

Placebo

Placebo two SC injections in the abdomen only.

Intervention Type DRUG

Other Intervention Names

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REGN727/SAR236553

Eligibility Criteria

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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

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sanofi

INDUSTRY

Sponsor Role collaborator

Regeneron Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trial Management

Role: STUDY_DIRECTOR

Regeneron Pharmaceuticals

Locations

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Port Orange, Florida, United States

Site Status

Chicago, Illinois, United States

Site Status

Kansas City, Kansas, United States

Site Status

Auburn, Maine, United States

Site Status

Biddeford, Maine, United States

Site Status

St Louis, Missouri, United States

Site Status

Concord, New Hampshire, United States

Site Status

Durham, North Carolina, United States

Site Status

Cincinnati, Ohio, United States

Site Status

Nashville, Tennessee, United States

Site Status

Houston, Texas, United States

Site Status

Winnipeg, Manitoba, Canada

Site Status

London, Ontario, Canada

Site Status

Chicoutimi, Quebec, Canada

Site Status

Montreal, Quebec, Canada

Site Status

Saint Foy, Quebec, Canada

Site Status

Huntsville, Alabama, United States

Site Status

Mission Viejo, California, United States

Site Status

Newport Beach, California, United States

Site Status

Bridgeport, Connecticut, United States

Site Status

Jacksonville, Florida, United States

Site Status

Miami, Florida, United States

Site Status

Countries

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United States Canada

References

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Leiter LA, Tinahones FJ, Karalis DG, Bujas-Bobanovic M, Letierce A, Mandel J, Samuel R, Jones PH. Alirocumab safety in people with and without diabetes mellitus: pooled data from 14 ODYSSEY trials. Diabet Med. 2018 Dec;35(12):1742-1751. doi: 10.1111/dme.13817. Epub 2018 Oct 9.

Reference Type DERIVED
PMID: 30183102 (View on PubMed)

Defesche JC, Stefanutti C, Langslet G, Hopkins PN, Seiz W, Baccara-Dinet MT, Hamon SC, Banerjee P, Kastelein JJP. Efficacy of alirocumab in 1191 patients with a wide spectrum of mutations in genes causative for familial hypercholesterolemia. J Clin Lipidol. 2017 Nov-Dec;11(6):1338-1346.e7. doi: 10.1016/j.jacl.2017.08.016. Epub 2017 Sep 4.

Reference Type DERIVED
PMID: 28964736 (View on PubMed)

Toth PP, Hamon SC, Jones SR, Martin SS, Joshi PH, Kulkarni KR, Banerjee P, Hanotin C, Roth EM, McKenney JM. Effect of alirocumab on specific lipoprotein non-high-density lipoprotein cholesterol and subfractions as measured by the vertical auto profile method: analysis of 3 randomized trials versus placebo. Lipids Health Dis. 2016 Feb 13;15:28. doi: 10.1186/s12944-016-0197-4.

Reference Type DERIVED
PMID: 26872608 (View on PubMed)

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. doi: 10.1016/S0140-6736(12)60771-5. Epub 2012 May 26.

Reference Type DERIVED
PMID: 22633824 (View on PubMed)

Other Identifiers

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R727-CL-1003

Identifier Type: -

Identifier Source: org_study_id

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