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

Regeneron Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Hypercholesterolemia

Treatments: Drug: Alirocumab; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT01266876

R727-CL-1003

Details and patient eligibility

About

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

Enrollment: 77 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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

Key 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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77 participants in 5 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo

Alirocumab 150 mg Q4W

Experimental group

Description:

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

Treatment:

Drug: Alirocumab

Alirocumab 200 mg Q4W

Experimental group

Description:

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

Treatment:

Drug: Alirocumab

Alirocumab 300 mg Q4W

Experimental group

Description:

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

Treatment:

Drug: Alirocumab

Alirocumab 150 mg Q2W

Experimental group

Description:

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

Treatment:

Drug: Alirocumab