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

Primary Purpose

Homozygous Familial Hypercholesterolemia

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

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

About this trial

This is an interventional treatment trial for Homozygous Familial Hypercholesterolemia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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

Exclusion Criteria:

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

Note: The information listed above is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial therefore not all inclusion/ exclusion criteria are listed.

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Open-label

Arm Description

Open-label REGN1500

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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

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

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

Absolute change in low-density lipoprotein cholesterol (LDL-C) from week 2 to week 4 was reported. The efficacy analysis set included all participants in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

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

Percent change was reported in low-density lipoprotein cholesterol (LDL-C) from week 2 to week 4. LDL-C was measured using conventional units mg/dL. The efficacy analysis set included all participants in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

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

Absolute change was reported in low-density lipoprotein cholesterol (LDL-C) from baseline (week 0) up to week 26. 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.

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

Percent change was reported in low-density lipoprotein cholesterol (LDL-C) from baseline (week 0) up to week 26. LDL-C was measured using conventional units mg/dL. The efficacy analysis set included all participants in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period

Absolute change in low-density lipoprotein cholesterol (LDL-C) from baseline (week 26) up to week 214 was reported. The efficacy analysis set included all participants in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period. (ET= Early Termination; EOS = End of Study)

Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period

Percent change was reported in low-density lipoprotein cholesterol (LDL-C) from baseline (week 26) to week 214 in the OLE period. LDL-C was measured using conventional units mg/dL. The efficacy analysis set included all participants in the SAF who had at least 1 post-baseline measure of the lipid panel in the main study period. (ET= Early Termination; EOS = End of Study)

Absolute Change in Apolipoprotein (Apo B), Non-High-Density Lipoprotein Cholesterol (Non-HDL-C), Total Cholesterol (Total-C), and Lipoprotein(a) (Lp[a]) From Baseline (Week 0) up to Week 26 in the Main Study Period

Absolute change was reported for Apo B, Non-HDL-C, Total-C, and Lp(a) from baseline (week 0) up to Week 26. The efficacy analysis set included all participants in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

Percent Change in Apolipoprotein (Apo B), Non-High-Density Lipoprotein Cholesterol (Non-HDL-C), Total Cholesterol (Total-C), and Lipoprotein(a) (Lp[a]) From Baseline (Week 0) up to Week 26 in the Main Study Period

Percent change was reported for Apo B, Non-HDL-C, Total-C, and Lp(a) from baseline (week 0) up to week 26. Apo B, Non-HDL-C, Total-C, and Lp(a) were measured using conventional units mg/dL. The efficacy analysis set included all participants in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

Absolute changein Apo B, Non-HDL-C, Total-C, and Lp(a) from baseline to week 214 in open label extension (OLE) period. The efficacy analysis set included all participants in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period. (ET = Early Termination; EOS = End of Study)

Percent change was reported in Apo B, Non-HDL-C, Total-C, and Lp(a) from baseline (week 26) up to week 214 in OLE Period. Apo B, Non-HDL-C, Total-C, and Lp(a) were measured using conventional units mg/dL. The efficacy analysis set included all participants in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period. (ET= Early Termination; EOS = End of Study)

Percentage of Participants Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 25% From Baseline (Week 0) in the Main Study Period

Percentage of participants who achieved reduction in low-density lipoprotein cholesterol (LDL-C) of greater than or equal to (≥) 25 percent (%) from baseline in the main study period was reported. The efficacy analysis set included all participants in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

Percentage of Participants Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 25% From Baseline (Week 26) in the Open Label Extension (OLE) Period

Percentage of participants who achieved a reduction in low-density lipoprotein cholesterol (LDL-C) of ≥ 25% from baseline (week 26) to week 214 was reported. The efficacy analysis set included all participants in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

Percentage of Participants Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 50% From Baseline (Week 0) in the Main Study Period

Percentage of participants who achieved a reduction in low-density lipoprotein cholesterol (LDL-C) of ≥ 50% from baseline (week 0) to week 26 was reported. The efficacy analysis set included all participants in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

Percentage of Participants Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 50% From Baseline (Week 26) in the Open Label Extension (OLE) Period

Percentage of participants who achieved reduction in low-density lipoprotein cholesterol (LDL-C) of ≥ 50% from baseline (week 26) in the OLE period was reported. The efficacy analysis set included all participants in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

Absolute Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) From Baseline (Week 0) Over Time in the Main Study Period

Absolute change was reported in HDL-C, TG, and Apo A-1 from baseline (week 0) up to week 26. The efficacy analysis set included all participants in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period.

Percent Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) From Baseline (Week 0) Over Time in the Main Study Period

Percent change was reported in HDL-C, TG, and Apo A-1 from baseline (week 0) up to week 26. HDL-C, TG, and Apo A-1 were measured using conventional units mg/dL. The efficacy analysis set included all participants in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

Percent Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period

Percent change was reported in HDL-C, TG and Apo A-1 from baseline (week 26) up to week 214. HDL-C, TG and Apo A-1 were measured using conventional units mg/dL. The efficacy analysis set included all participants in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period. (ET= Early Termination; EOS = End of Study)

Absolute Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period

Absolute change was reported in HDL-C, TG and Apo A-1 from baseline (week 26) up to week 214. The efficacy analysis set included all participants in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period. (ET= Early Termination; EOS = End of Study)

Absolute Change in Apolipoprotein CIII (Apo CIII) From Baseline (Week 0) Over Time in the Main Study Period

Absolute change was reported in Apo CIII from baseline (week 0) up to week 16. The efficacy analysis set included all participants in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

Percent Change in Apolipoprotein CIII (Apo CIII) From Baseline (Week 0) Over Time in the Main Study Period

Percent change was reported in Apo CIII from baseline (week 0) up to week 16. Apo CIII was measured using conventional units mg/dL. The efficacy analysis set included all participants in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period.

Full Information

NCT ID

NCT02265952

First Posted

October 10, 2014

Last Updated

December 6, 2019

Sponsor

Regeneron Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT02265952

Brief Title

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

Official Title

An Open-Label, Single-Arm, Proof-of-Concept Study to Evaluate the Safety and Efficacy of Single and Multiple Doses of REGN1500 in Patients With Homozygous Familial Hypercholesterolemia

Study Type

Interventional

2. Study Status

Record Verification Date

December 2019

Overall Recruitment Status

Completed

Study Start Date

February 4, 2015 (Actual)

Primary Completion Date

November 21, 2016 (Actual)

Study Completion Date

July 23, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Regeneron Pharmaceuticals

4. Oversight

Data Monitoring Committee

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Homozygous Familial Hypercholesterolemia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

9 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Open-label

Arm Type

Experimental

Arm Description

Open-label REGN1500

Intervention Type

Drug

Intervention Name(s)

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

Intervention Description

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.

Primary Outcome Measure Information:

Title

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

Description

Time Frame

Baseline (Week 0) up to Week 4

Secondary Outcome Measure Information:

Title

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

Description

Time Frame

Baseline (Week 0) up to Week 4

Title

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

Description

Time Frame

Week 2 to Week 4

Title

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

Description

Time Frame

Week 2 to Week 4

Title

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

Description

Time Frame

Baseline (Week 0) up to Week 26

Title

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

Description

Time Frame

Baseline (Week 0) up to Week 26

Title

Absolute Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period

Description

Time Frame

Baseline (Week 26) up to Week 214

Title

Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period

Description

Time Frame

Baseline (Week 26) up to Week 214

Title

Description

Time Frame

Baseline (Week 0) up to Week 26

Title

Description

Time Frame

Baseline (Week 0) up to Week 26

Title

Description

Time Frame

Baseline (Week 26) up to Week 214

Title

Description

Time Frame

Baseline (Week 26) up to Week 214

Title

Percentage of Participants Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 25% From Baseline (Week 0) in the Main Study Period

Description

Time Frame

Baseline (Week 0) up to Week 26

Title

Percentage of Participants Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 25% From Baseline (Week 26) in the Open Label Extension (OLE) Period

Description

Time Frame

Baseline (Week 26) to Week 214

Title

Percentage of Participants Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 50% From Baseline (Week 0) in the Main Study Period

Description

Time Frame

Baseline (Week 0) to Week 26

Title

Percentage of Participants Who Achieved Reduction in Low-Density Lipoprotein Cholesterol (LDL-C) of ≥ 50% From Baseline (Week 26) in the Open Label Extension (OLE) Period

Description

Time Frame

Baseline (Week 26) up to Week 214

Title

Absolute Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) From Baseline (Week 0) Over Time in the Main Study Period

Description

Time Frame

Baseline (Week 0) up to Week 26

Title

Percent Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) From Baseline (Week 0) Over Time in the Main Study Period

Description

Time Frame

Baseline (Week 0) up to Week 26

Title

Percent Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period

Description

Time Frame

Baseline (Week 26) up to Week 214

Title

Absolute Change in High-Density Lipoprotein Cholesterol (HDL-C), Triglycerides (TG), and Apolipoprotein A-1 (Apo A-1) From Baseline (Week 26) to Week 214 in the Open Label Extension (OLE) Period

Description

Time Frame

Baseline (Week 26) up to Week 214

Title

Absolute Change in Apolipoprotein CIII (Apo CIII) From Baseline (Week 0) Over Time in the Main Study Period

Description

Time Frame

Baseline (Week 0) up to Week 16

Title

Percent Change in Apolipoprotein CIII (Apo CIII) From Baseline (Week 0) Over Time in the Main Study Period

Description

Time Frame

Baseline (Week 0) to Week 16

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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 Exclusion Criteria: 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 Note: The information listed above is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial therefore not all inclusion/ exclusion criteria are listed.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Clinical Trial Management

Organizational Affiliation

Regeneron Pharmaceuticals

Official's Role

Study Director

Facility Information:

City

Los Angeles

State/Province

California

Country

United States

City

Philadelphia

State/Province

Pennsylvania

Country

United States

City

Dallas

State/Province

Texas

Country

United States

City

Chicoutimi

State/Province

Quebec

Country

Canada

City

Amsterdam

Country

Netherlands

12. IPD Sharing Statement

Citations:

PubMed Identifier

28723334

Citation

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.

Results Reference

background

PubMed Identifier

31578082

Citation

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.

Results Reference

derived

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

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