Open-Label Extension of Study R727-CL-1003 (NCT01266876) to Evaluate the Long-Term Safety and Efficacy of Alirocumab (REGN727) in Participants With Heterozygous Familial Hypercholesterolemia (HeFH)

Regeneron Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Hypercholesterolemia; Heterozygous Familial Hypercholesterolemia

Treatments: Drug: Alirocumab; Drug: Placebo Matched to Alirocumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT01576484

R727-CL-1032

Details and patient eligibility

About

The primary objective of the study was to assess the long-term safety and tolerability of alirocumab in patients with heFH who were receiving concomitant treatment with hydroxymethyl glutaryl-coenzyme A (HMG-CoA) reductase inhibitors (statins), with or without other lipid-modifying therapies (LMTs).

Enrollment: 58 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

1. Prior participation in and the successful completion of the R727-CL-1003 study (NCT01266876).
2. Patients must be on a stable daily statin regimen for at least 3 weeks before prior to entry into the study
3. A negative urine pregnancy at the screening/baseline visit for women of childbearing potential

Key Exclusion Criteria:

1. Reported a drug-related serious adverse event (SAE) or drug-related clinical or laboratory adverse event (AE) in the R727-CL-1003 study that resulted in early termination or withdrawal
2. Significant protocol deviation in R727-CL-1003, such as non-compliance by the investigator or patient
3. Low-density lipoprotein (LDL) apheresis within 12 months before the screening/baseline visit

Note: Other exclusion criteria applied

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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58 participants in 2 patient groups, including a placebo group

Placebo Matched to Alirocumab

Placebo Comparator group

Description:

Participants who received placebo in parent study (NCT01576484), has received a subcutaneous injection of placebo matched to alirocumab every 2 weeks for 4 years in this study.

Treatment:

Drug: Placebo Matched to Alirocumab

Alirocumab 150 mg

Experimental group

Description:

Participants who received alirocumab in parent study (NCT01576484), has received a subcutaneous injection of alirocumab 150 milligram (mg) every 2 weeks for 4 years in this study.

Treatment:

Drug: Alirocumab