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

NCT ID: NCT02265952

Last Updated: 2019-12-09

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

9 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-02-04

Study Completion Date

2018-07-23

Brief Summary

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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).

Detailed Description

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Conditions

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Homozygous Familial Hypercholesterolemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Open-label

Open-label REGN1500

Group Type EXPERIMENTAL

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

Intervention Type 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.

Interventions

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

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.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

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

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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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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Los Angeles, California, United States

Site Status

Philadelphia, Pennsylvania, United States

Site Status

Dallas, Texas, United States

Site Status

Chicoutimi, Quebec, Canada

Site Status

Amsterdam, , Netherlands

Site Status

Countries

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United States Canada Netherlands

References

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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.

Reference Type BACKGROUND
PMID: 28723334 (View on PubMed)

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.

Reference Type DERIVED
PMID: 31578082 (View on PubMed)

Other Identifiers

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2016-000411-32

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

R1500-CL-1331

Identifier Type: -

Identifier Source: org_study_id