Study of REGN1500 in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

NCT02265952 | Completed Phase 2 Results

• 2 other identifiers: R1500-CL-13312016-000411-32
• Study Type: interventional
• Target: 9
• Locations: 3 countries
• Sites: 5

Brief Summary

This is an open-label, single-arm study to assess the reduction of low-density lipoprotein cholesterol (LDL-C) by REGN1500 in patients with homozygous familial hypercholesterolemia (HoFH).

Conditions

Homozygous Familial Hypercholesterolemia

Outcome Measures

Primary Outcomes (1)

• Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 0) to Week 4 in the Main Study Period

  Percent change was reported for low-density lipoprotein cholesterol (LDL-C) from baseline (week 0) up to week 4. LDL-C was measured using conventional units mg/dL. The efficacy analysis set included all participants in the safety analysis set (SAF) who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

  Baseline (Week 0) up to Week 4

Secondary Outcomes (21)

• Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 0) to Week 4 in the Main Study Period

  Baseline (Week 0) up to Week 4

• Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Week 2 to Week 4 in the Main Study Period

  Week 2 to Week 4

• Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Week 2 to Week 4 in the Main Study Period

  Week 2 to Week 4

• Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 0) Over Time in the Main Study Period

  Baseline (Week 0) up to Week 26

• Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 0) Over Time in the Main Study Period

  Baseline (Week 0) up to Week 26

Study Arms (1)

Open-label

EXPERIMENTAL

Open-label REGN1500

Drug: REGN1500 250 mg SC/15 mg/kg IV/450 mg SC

Interventions

REGN1500 250 mg SC/15 mg/kg IV/450 mg SC DRUG

Participants received single dose of REGN1500 subcutaneous (SC) injection of 250 milligrams (mg) at Week 0 (Day 1), followed by single dose of REGN1500 intravenous (IV) injection of 15 milligrams per kilogram (mg/kg) at Week 2 (Day 15) and then followed by 4 doses of REGN1500 SC injection of 450 mg once weekly starting from Week 12 (Day 85). Only the first 2 enrolled participants received 4 weekly REGN1500 450 mg SC doses at weeks 12, 13, 14, and 15 per the protocol. This dose regimen was removed under protocol amendment 4. Participants were followed for a period of 24 weeks (through Week 26 \[Day 183\]) after the last dose of study drug in the main study period.

Open-label

Eligibility Criteria

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

You may qualify if:

• Men and women ≥18 years of age at the time of the screening visit
• Diagnosis of homozygous familial hypercholesterolemia (HoFH)
• Willing to consistently maintain usual diet for the duration of the study

You may not qualify if:

• Background medical lipid modifying therapy that has not been stable for at least 4 weeks (6 weeks for fibrates) prior to the screening visit
• Having undergone lipid apheresis within 4 weeks prior to the screening visit
• Use of another investigational drug or therapy within 30 days or at least 5 half-lives (whichever is longer) prior to the screening visit
• Previous participation in any clinical trial of REGN1500

Sponsors & Collaborators

• Regeneron Pharmaceuticals: lead

Study Sites (5)

Unknown Facility

Los Angeles, California, United States

Location

Unknown Facility

Philadelphia, Pennsylvania, United States

Location

Unknown Facility

Dallas, Texas, United States

Location

Unknown Facility

Chicoutimi, Quebec, Canada

Location

Unknown Facility

Amsterdam, Netherlands

Location

Related Publications (2)

• Gaudet D, Gipe DA, Pordy R, Ahmad Z, Cuchel M, Shah PK, Chyu KY, Sasiela WJ, Chan KC, Brisson D, Khoury E, Banerjee P, Gusarova V, Gromada J, Stahl N, Yancopoulos GD, Hovingh GK. ANGPTL3 Inhibition in Homozygous Familial Hypercholesterolemia. N Engl J Med. 2017 Jul 20;377(3):296-297. doi: 10.1056/NEJMc1705994. No abstract available.

  PMID: 28723334 BACKGROUND

• Banerjee P, Chan KC, Tarabocchia M, Benito-Vicente A, Alves AC, Uribe KB, Bourbon M, Skiba PJ, Pordy R, Gipe DA, Gaudet D, Martin C. Functional Analysis of LDLR (Low-Density Lipoprotein Receptor) Variants in Patient Lymphocytes to Assess the Effect of Evinacumab in Homozygous Familial Hypercholesterolemia Patients With a Spectrum of LDLR Activity. Arterioscler Thromb Vasc Biol. 2019 Nov;39(11):2248-2260. doi: 10.1161/ATVBAHA.119.313051. Epub 2019 Oct 3.

  PMID: 31578082 DERIVED

MeSH Terms

Conditions

Homozygous Familial Hypercholesterolemia

Interventions

evinacumab

Condition Hierarchy (Ancestors)

Hyperlipoproteinemia Type II; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hyperlipoproteinemias; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Limitations and Caveats

At the time patients completed the main study, the open-label extension (OLE) study was not yet open to enrollment. Therefore, there was a lag of between 168 days to 586 days between the end of the main study and the start of the OLE study.

Results Point of Contact

• Title: Clinical Trial Information
• 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: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 10, 2014
• First Posted: October 16, 2014
• Study Start: February 4, 2015
• Primary Completion: November 21, 2016
• Study Completion: July 23, 2018
• Last Updated: December 9, 2019
• Results First Posted: December 9, 2019

Record last verified: 2019-12

Locations

US (3); CA (1); NL (1)