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)

NCT01576484 | Completed Phase 2 Results DMC

• 1 other identifier: R727-CL-1032
• Study Type: interventional
• Target: 58
• Locations: 2 countries
• Sites: 14

Brief Summary

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).

Conditions

Hypercholesterolemia; Heterozygous Familial Hypercholesterolemia

Outcome Measures

Primary Outcomes (1)

• Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Death

  An AE was defined as any untoward medical occurrence in a participant administered a study drug which may or may not have a causal relationship with the study drug. Treatment- emergent adverse events (TEAEs) were defined as AEs that developed or worsened or became serious during on- treatment period (time from the first dose of study drug to the last dose of study drug plus 70 days). A serious adverse event (SAE) was defined as any untoward medical occurrence that resulted in any of the following outcomes: death, life- threatening, required initial or prolonged in-patient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event. Any TEAE included participants with both serious and non-serious AEs.

  Baseline (Day 1 of current study) to end of study (Week 218)

Secondary Outcomes (19)

• Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline in the Current Study to Week 24

  Baseline (current study) to Week 24

• Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) From Baseline in Current Study to Week 12

  Baseline (current study) up to Week 12

• Percent Change in Apolipoprotein (Apo) B, Non-High Density Lipoprotein Cholesterol (HDL-C), and Total Cholesterol From Baseline in Current Study to Week 24

  Baseline(current study) up to Week 24

• Percent Change in Apolipoprotein (Apo) B, Non-High Density Lipoprotein Cholesterol (HDL-C), and Total Cholesterol From Baseline in Current Study to Week 12

  Baseline (current study) up to Week 12

• Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) From Baseline in Current Study to Week 52

  Baseline (current study) up to Week 52

Study Arms (2)

Placebo Matched to Alirocumab

PLACEBO COMPARATOR

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.

Drug: Placebo Matched to Alirocumab

Alirocumab 150 mg

EXPERIMENTAL

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.

Drug: Alirocumab

Interventions

Alirocumab DRUG

Alirocumab was supplied in a pre-filled syringe and administered subcutaneously (SC) in the abdomen, thigh, or outer upper arm; REGN727(SAR236553) is an anti-PCSK9 (proprotein convertase subtilisin/kexin type 9) antibody

Also known as: REGN727, SAR236553, PRALUENT

Alirocumab 150 mg

Placebo Matched to Alirocumab DRUG

Placebo matched to alirocumab was supplied in a pre-filled syringe and administered subcutaneously (SC) in the abdomen, thigh, or outer upper arm; REGN727(SAR236553) is an anti-PCSK9 (proprotein convertase subtilisin/kexin type 9) antibody

Placebo Matched to Alirocumab

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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
• Significant protocol deviation in R727-CL-1003, such as non-compliance by the investigator or patient
• Low-density lipoprotein (LDL) apheresis within 12 months before the screening/baseline visit

Sponsors & Collaborators

• Regeneron Pharmaceuticals: lead
• Sanofi: collaborator

Study Sites (14)

Unknown Facility

Mission Viejo, California, United States

Location

Unknown Facility

Newport Beach, California, United States

Location

Unknown Facility

Thousand Oaks, California, United States

Location

Unknown Facility

Miami, Florida, United States

Location

Unknown Facility

Port Orange, Florida, United States

Location

Unknown Facility

Kansas City, Kansas, United States

Location

Unknown Facility

Auburn, Maine, United States

Location

Unknown Facility

St Louis, Missouri, United States

Location

Unknown Facility

Durham, North Carolina, United States

Location

Unknown Facility

Cincinnati, Ohio, United States

Location

Unknown Facility

Houston, Texas, United States

Location

Unknown Facility

Chicoutimi, Quebec, Canada

Location

Unknown Facility

Montreal, Quebec, Canada

Location

Unknown Facility

Sainte-Foy, Quebec, Canada

Location

Related Publications (1)

• Dufour R, Bergeron J, Gaudet D, Weiss R, Hovingh GK, Qing Z, Yang F, Andisik M, Torri A, Pordy R, Gipe DA. Open-label therapy with alirocumab in patients with heterozygous familial hypercholesterolemia: Results from three years of treatment. Int J Cardiol. 2017 Feb 1;228:754-760. doi: 10.1016/j.ijcard.2016.11.046. Epub 2016 Nov 9.

  PMID: 27886619 DERIVED

MeSH Terms

Conditions

Hypercholesterolemia

Interventions

alirocumab

Condition Hierarchy (Ancestors)

Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Clinical Trial Administrator
• Organization: Regeneron Pharmaceuticals, Inc.

clinicaltrials@regeneron.com

844-734-6643

Study Officials

• Clinical Trial Management

  Regeneron Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 27, 2012
• First Posted: April 12, 2012
• Study Start: February 28, 2012
• Primary Completion: December 22, 2016
• Study Completion: December 22, 2016
• Last Updated: August 5, 2020
• Results First Posted: August 5, 2020

Record last verified: 2020-07

Locations

US (11); CA (3)