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Clinical Trial Results:
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

Summary
EudraCT number	2016-000411-32
Trial protocol	NL
Global end of trial date	23 Jul 2018

Results information
Results version number	v1(current)
This version publication date	07 Aug 2019
First version publication date	07 Aug 2019
Other versions

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

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Sponsor protocol code

R1500-CL-1331

ISRCTN number

US NCT number

NCT02265952

WHO universal trial number (UTN)

Sponsor organisation name

Regeneron Pharmaceuticals, Inc.

Sponsor organisation address

777 Old Saw Mill River Road, Tarrytown, United States, 10591

Public contact

Clinical Trial Information, Regeneron Pharmaceuticals, Inc., clinicaltrials@regeneron.com

Scientific contact

Clinical Trial Information, Regeneron Pharmaceuticals, Inc., clinicaltrials@regeneron.com

Is trial part of an agreed paediatric investigation plan (PIP)

Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Analysis stage

Final

Date of interim/final analysis

09 Aug 2018

Is this the analysis of the primary completion data?

Global end of trial reached?

Yes

Global end of trial date

23 Jul 2018

Was the trial ended prematurely?

Main objective of the trial

The purpose of this study was to assess the reduction of low-density lipoprotein cholesterol (LDL-C) by REGN1500 in subjects with Homozygous Familial Hypercholesterolemia (HoFH).

Protection of trial subjects

This study was conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki and that are consistent with the International Conference on Harmonisation (ICH) guidelines for Good Clinical Practice (GCP) and applicable regulatory requirements.

Background therapy

Evidence for comparator

Actual start date of recruitment

04 Feb 2015

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Number of subjects enrolled per country

Country: Number of subjects enrolled

Canada: 3

Country: Number of subjects enrolled

United States: 4

Country: Number of subjects enrolled

Netherlands: 2

Worldwide total number of subjects

EEA total number of subjects

Number of subjects enrolled per age group

In utero

Preterm newborn - gestational age < 37 wk

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

Adolescents (12-17 years)

Adults (18-64 years)

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

The study was conducted at five sites in United States, Canada and the Netherlands between 04 Feb 2015 and 23 Jul 2018. A total of 12 subjects were screened in the study. Out of 12 subjects, 9 subjects received the study treatment.

Screening details

This study consisted of 2 screening periods: 1st for main study and 2nd for open label extension (OLE) period. Subjects on stable background medical lipid-modifying therapy for at least 4 weeks prior to screening, or who didn't undergo lipid apheresis within 4 weeks prior to screening visit, were directly entered into a 2-week screening period.

Period 1 title

Main Study Period

Is this the baseline period?

Yes

Allocation method

Non-randomised - controlled

Blinding used

Not blinded

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

Arm description

Subjects 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 subjects 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. Subjects 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.

Arm type

Experimental

Investigational medicinal product name

REGN1500

Investigational medicinal product code

REGN1500

Other name

Evinacumab

Pharmaceutical forms

Concentrate for solution for injection/infusion

Routes of administration

Intravenous use, Subcutaneous use

Dosage and administration details

Subjects received REGN1500 SC in the abdomen/ IV infusion in the main study period.

Number of subjects in period 1	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Started	9
Completed	9

Period 2 title

Open Label Extension (OLE) Period

Is this the baseline period?

Allocation method

Non-randomised - controlled

Blinding used

Not blinded

REGN1500 300 mg SC/20 mg/kg IV

Arm description

Subjects received REGN1500 a SC injection of 300 mg at Week 26 (Day 183), 27(Day 190), 28 (Day 197) and 29 (Day 204) followed by IV injection of 20 mg/kg at Week 38 (Day 267) and every 12 weeks starting at Week 58 (Day 407) through Week 178 (Day 1247) in open-label extension period. Subjects were to be followed for a period of 24 weeks (through Week 214 [Day 1499]) after the last dose of study drug in the OLE treatment period.

Arm type

Experimental

Investigational medicinal product name

REGN1500

Investigational medicinal product code

REGN1500

Other name

Evinacumab

Pharmaceutical forms

Concentrate for solution for injection/infusion

Routes of administration

Intravenous use, Subcutaneous use

Dosage and administration details

Subjects received REGN1500 SC in the abdomen/ IV infusion in the open-label treatment period.

Number of subjects in period 2 ^[1]	REGN1500 300 mg SC/20 mg/kg IV
Started	8
Completed	0
Not completed	8
Physician decision	7
Consent withdrawn by subject	1

Notes
[1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
Justification: One subject chose not to enroll in OLE period.

Baseline characteristics

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Reporting group title

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

Reporting group description

Reporting group values	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC	Total
Number of subjects	9	9
Age categorical
Units: Subjects
< 45	7	7
≥ 45 to < 65	2	2
≥ 65 to < 75	0	0
< 75	0	0
Age continuous
Units: years
arithmetic mean (standard deviation)	35.9 ± 9.06	-
Gender categorical
Units: Subjects
Female	4	4
Male	5	5
Ethnicity
Units: Subjects
Hispanic or Latino	3	3
Not Hispanic or Latino	6	6
Not Reported	0	0
Race
Units: Subjects
White	7	7
Black or African American	0	0
Asian	0	0
American Indian or Alaska Native	0	0
Native Hawaiian or Other Pacific Islander	0	0
Other	0	0
Not Reported	2	2
Baseline Low-Density Lipoprotein Cholesterol (LDL-C)
Units: Milligram per Deciliter (mg/dL)
arithmetic mean (standard deviation)	376.0 ± 240.85	-
Baseline High-Density Lipoprotein Cholesterol (HDL-C)
Units: mg/dL
arithmetic mean (standard deviation)	38.8 ± 14.24	-
Baseline Non-High-Density Lipoprotein Cholesterol (Non-HDL-C)
Units: mg/dL
arithmetic mean (standard deviation)	392.1 ± 246.41	-
Baseline Apolipoprotein B (Apo B)
Units: mg/dL
arithmetic mean (standard deviation)	226.1 ± 131.70	-
Baseline Apolipoprotein CIII (Apo CIII)
Units: mg/dL
arithmetic mean (standard deviation)	7.976 ± 3.3747	-
Baseline Triglyceride (TG)
Units: mg/dL
arithmetic mean (standard deviation)	80.0 ± 41.20	-
Baseline Lipoprotein(a) (Lp[a])
Units: mg/dL
arithmetic mean (standard deviation)	154.9 ± 109.25	-
Baseline Total Cholesterol (Total -C)
Units: mg/dL
arithmetic mean (standard deviation)	430.9 ± 235.60	-
Baseline Apolipoprotein A-1 (Apo A-1)
Units: mg/dL
arithmetic mean (standard deviation)	110.4 ± 23.74	-

End points

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Reporting group title

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

Reporting group description

Reporting group title

REGN1500 300 mg SC/20 mg/kg IV

Reporting group description

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

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End point title

Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0) to Week 4 in the Main Study Period ^[1]

End point description

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 subjects 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.

End point type

Primary

End point timeframe

Baseline (Week 0) up to Week 4

Notes
[1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: No sensitivity analysis was completed for this study.

End point values	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Number of subjects analysed	9
Units: Percent Change
arithmetic mean (standard deviation)	-49.17 ± 23.136

No statistical analyses for this end point

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

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End point title

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

End point description

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 subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

End point type

Secondary

End point timeframe

Baseline (Week 0) up to Week 4

End point values	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Number of subjects analysed	9
Units: mg/dL
arithmetic mean (standard deviation)	-157.34 ± 89.590

No statistical analyses for this end point

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

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End point title

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

End point description

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

End point type

Secondary

End point timeframe

Week 2 to Week 4

End point values	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Number of subjects analysed	9
Units: Milligram per Deciliter (mg/dL)
arithmetic mean (standard deviation)	-63.63 ± 50.970

No statistical analyses for this end point

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

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End point title

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

End point description

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 subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

End point type

Secondary

End point timeframe

Week 2 to Week 4

End point values	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Number of subjects analysed	9
Units: Percent Change
arithmetic mean (standard deviation)	-30.05 ± 18.503

No statistical analyses for this end point

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

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End point title

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

End point description

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 subjects 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. Here, "n" signifies those subjects who were evaluable for this endpoint at a given time point.

End point type

Secondary

End point timeframe

Baseline (Week 0) up to Week 26

End point values	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Number of subjects analysed	9
Units: mg/dL
arithmetic mean (standard deviation)
Change at Day 4 (n= 9)	-39.60 ± 41.677
Change at Week 1 (n= 9)	-67.59 ± 46.041
Change at Week 2 (n= 9)	-93.71 ± 86.504
Change at Week 3 (n= 9)	-129.97 ± 90.533
Change at Week 4 (n= 9)	-157.34 ± 89.590
Change at Week 5 (n= 8)	-179.18 ± 101.260
Change at Week 6 (n= 9)	-183.41 ± 111.313
Change at Week 8 (n= 9)	-176.72 ± 118.441
Change at Week 10 (n= 9)	-151.19 ± 78.164
Change at Week 12 (n= 9)	-109.40 ± 74.108
Change at Week 13 (n= 2)	-170.15 ± 105.288
Change at Week 14 (n= 9)	-104.16 ± 78.954
Change at Week 15 (n= 2)	-186.15 ± 119.147
Change at Week 16 (n= 9)	-97.29 ± 179.016
Change at Week 17 (n= 2)	-174.20 ± 103.803
Change at Week 18 (n= 7)	-62.11 ± 167.004
Change at Week 20 (n= 2)	-155.25 ± 79.832
Change at Week 22 (n= 7)	-41.37 ± 169.432
Change at Week 23 (n= 2)	-155.85 ± 78.984
Change at Week 25 (n= 2)	-87.90 ± 23.900
Change at Week 26 (n= 6)	-23.98 ± 152.794

No statistical analyses for this end point

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

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End point title

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

End point description

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 subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for this endpoint at a given time point.

End point type

Secondary

End point timeframe

Baseline (Week 0) up to Week 26

End point values	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Number of subjects analysed	9
Units: Percent Change
arithmetic mean (standard deviation)
Percent change at Day 4 (n= 9)	-12.76 ± 11.086
Percent change at Week 1 (n= 9)	-24.00 ± 21.078
Percent change at Week 2 (n= 9)	-30.13 ± 24.402
Percent change at Week 3 (n= 9)	-41.38 ± 24.986
Percent change at Week 4 (n= 9)	-49.17 ± 23.136
Percent change at Week 5 (n= 8)	-46.88 ± 14.507
Percent change at Week 6 (n= 9)	-52.13 ± 14.768
Percent change at Week 8 (n= 9)	-51.59 ± 17.910
Percent change at Week 10 (n= 9)	-45.61 ± 13.839
Percent change at Week 12 (n= 9)	-36.54 ± 19.281
Percent change at Week 13 (n= 2)	-39.84 ± 10.753
Percent change at Week 14 (n= 9)	-33.02 ± 18.538
Percent change at Week 15 (n= 2)	-43.38 ± 12.814
Percent change at Week 16 (n= 9)	-24.98 ± 25.441
Percent change at Week 17 (n= 2)	-40.99 ± 9.950
Percent change at Week 18 (n= 7)	-13.72 ± 28.424
Percent change at Week 20 (n= 2)	-37.17 ± 5.503
Percent change at Week 22 (n= 7)	-9.50 ± 38.456
Percent change at Week 23 (n= 2)	-37.37 ± 5.217
Percent change at Week 25 (n= 2)	-22.12 ± 2.537
Percent change at Week 26 (n= 6)	-10.27 ± 25.806

No statistical analyses for this end point

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

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End point title

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

End point description

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 subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for the endpoint at a given time point. (ET= Early Termination; EOS = End of Study)

End point type

Secondary

End point timeframe

Baseline (Week 26) up to Week 214

End point values	REGN1500 300 mg SC/20 mg/kg IV
Number of subjects analysed	8
Units: mg/dL
arithmetic mean (standard deviation)
Change at Week 26 (n= 8)	261.34 ± 178.792
Change at Week 27 (n= 8)	-58.73 ± 56.992
Change at Week 28 (n= 8)	-88.16 ± 83.196
Change at Week 29 (n= 8)	-116.20 ± 102.219
Change at Week 30 (n= 7)	-87.80 ± 108.905
Change at Week 31 (n= 8)	-144.99 ± 130.764
Change at Week 32 (n= 8)	-159.16 ± 128.072
Change at Week 34 (n= 8)	-128.38 ± 108.331
Change at Week 36 (n= 8)	-105.94 ± 89.990
Change at Week 38 (n= 8)	-79.98 ± 97.851
Change at Week 39 (n= 6)	-138.28 ± 123.133
Change at Week 40 (n= 6)	-127.13 ± 122.808
Change at Week 41 (n= 6)	-163.28 ± 137.365
Change at Week 42 (n= 6)	-142.65 ± 126.647
Change at Week 44 (n= 8)	-140.36 ± 109.275
Change at Week 46 (n= 8)	-123.25 ± 112.503
Change at Week 50 (n= 8)	-106.61 ± 99.458
Change at Week 54 (n= 8)	-13.38 ± 177.649
Change at Week 58 (n= 6)	-48.27 ± 93.443
Change at Week 70 (n= 4)	-117.93 ± 145.917
Change at Week 82 (n= 4)	-63.88 ± 64.738
Change at Week 94 (n= 1)	-369.90 ± 99999
Change at Week 106 (n= 1)	-290.00 ± 99999
Change at ET (n= 8)	-9.78 ± 64.930
Change at EOS (n= 3)	95.63 ± 147.100

No statistical analyses for this end point

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

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End point title

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

End point description

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 subjects in the SAF who had at least 1 post-baseline measure of the lipid panel in the main study period. Here "n" signifies those subjects who were evaluable for this endpoint at a given time point. (ET= Early Termination; EOS = End of Study)

End point type

Secondary

End point timeframe

Baseline (Week 26) up to Week 214

End point values	REGN1500 300 mg SC/20 mg/kg IV
Number of subjects analysed	8
Units: Percent Change
arithmetic mean (standard deviation)
Baseline (n= 8)	261.34 ± 178.792
Percent change at Week 27 (n= 8)	-22.49 ± 16.520
Percent change at Week 28 (n= 8)	-36.74 ± 20.931
Percent change at Week 29 (n= 8)	-44.35 ± 20.817
Percent change at Week 30 (n= 7)	-37.37 ± 31.297
Percent change at Week 31 (n= 8)	-50.74 ± 20.147
Percent change at Week 32 (n= 8)	-60.03 ± 18.502
Percent change at Week 34 (n= 8)	-53.03 ± 22.130
Percent change at Week 36 (n= 8)	-47.90 ± 21.623
Percent change at Week 38 (n= 8)	-37.05 ± 25.341
Percent change at Week 39 (n= 6)	-59.83 ± 22.220
Percent change at Week 40 (n= 6)	-53.90 ± 20.970
Percent change at Week 41 (n= 6)	-66.28 ± 18.555
Percent change at Week 42 (n= 6)	-59.59 ± 18.950
Percent change at Week 44 (n= 8)	-57.64 ± 18.599
Percent change at Week 46 (n= 8)	-48.74 ± 19.717
Percent change at Week 50 (n= 8)	-42.31 ± 23.194
Percent change at Week 54 (n= 8)	-13.60 ± 44.339
Percent change at Week 58 (n= 6)	-12.86 ± 27.645
Percent change at Week 70 (n= 4)	-38.61 ± 36.151
Percent change at Week 82 (n= 4)	-24.68 ± 30.789
Percent change at Week 94 (n= 1)	-81.67 ± 99999
Percent change at Week 106 (n= 1)	-64.03 ± 99999
Percent change at ET (n= 8)	-17.65 ± 28.813
Percent change at EOS (n= 3)	19.59 ± 40.976

No statistical analyses for this end point

Secondary: 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

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End point title

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

End point description

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 subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for this endpoint at a given time point.

End point type

Secondary

End point timeframe

Baseline (Week 0) up to Week 26

End point values	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Number of subjects analysed	9
Units: mg/dL
arithmetic mean (standard deviation)
Apo B: Change at Week 2 (n = 9)	-53.0 ± 56.29
Apo B: Change at Week 3 (n = 9)	-81.0 ± 54.97
Apo B: Change at Week 4 (n = 9)	-96.3 ± 56.01
Apo B: Change at Week 5 (n = 8)	-104.6 ± 61.43
Apo B: Change at Week 6 (n = 9)	-100.7 ± 71.12
Apo B: Change at Week 8 (n = 9)	-96.8 ± 67.36
Apo B: Change at Week 12 (n = 9)	-60.0 ± 63.61
Apo B: Change at Week 14 (n = 9)	-61.7 ± 64.18
Apo B: Change at Week 16 (n = 9)	-62.6 ± 98.77
Apo B: Change at Week 18 (n = 7)	-28.6 ± 80.58
Apo B: Change at Week 20 (n = 2)	-114.0 ± 67.88
Apo B: Change at Week 22 (n = 7)	-22.7 ± 77.82
Apo B: Change at Week 23 (n = 2)	-119.0 ± 79.20
Apo B: Change at Week 26 (n = 6)	-4.7 ± 68.52
Non-HDL-C: Change at Day 4 (n = 9)	-44.61 ± 42.322
Non-HDL-C: Change at Week 1 (n = 9)	-72.16 ± 46.480
Non-HDL-C: Change at Week 2 (n = 9)	-97.12 ± 88.026
Non-HDL-C: Change at Week 3 (n = 9)	-138.09 ± 91.574
Non-HDL-C: Change at Week 4 (n = 9)	-165.64 ± 93.130
Non-HDL-C: Change at Week 5 (n = 8)	-187.46 ± 104.826
Non-HDL-C: Change at Week 6 (n = 9)	-191.20 ± 116.337
Non-HDL-C: Change at Week 8 (n = 9)	-182.98 ± 121.701
Non-HDL-C: Change at Week 10 (n = 9)	-156.98 ± 81.636
Non-HDL-C: Change at Week 12 (n = 9)	-116.20 ± 77.582
Non-HDL-C: Change at Week 13 (n = 2)	-180.25 ± 116.178
Non-HDL-C: Change at Week 14 (n = 9)	-108.58 ± 83.773
Non-HDL-C: Change at Week 15 (n = 2)	-196.85 ± 135.270
Non-HDL-C: Change at Week 16 (n = 9)	-99.58 ± 186.796
Non-HDL-C: Change at Week 17 (n = 2)	-185.40 ± 114.834
Non-HDL-C: Change at Week 18 (n = 7)	-60.41 ± 169.805
Non-HDL-C: Change at Week 20 (n = 2)	-161.90 ± 85.843
Non-HDL-C: Change at Week 22 (n = 7)	-41.16 ± 175.121
Non-HDL-C: Change at Week 23 (n = 2)	-163.25 ± 85.065
Non-HDL-C: Change at Week 25 (n = 2)	-92.20 ± 29.981
Non-HDL-C: Change at Week 26 (n = 6)	-25.47 ± 156.948
Total-C: Change at Day 4 (n = 9)	-50.36 ± 41.860
Total-C: Change at Week 1 (n = 9)	-81.99 ± 46.513
Total-C: Change at Week 2 (n = 9)	-109.03 ± 86.365
Total-C: Change at Week 3 (n = 9)	-154.10 ± 86.927
Total-C: Change at Week 4 (n = 9)	-180.79 ± 87.304
Total-C: Change at Week 5 (n = 8)	-199.11 ± 98.202
Total-C: Change at Week 6 (n = 9)	-207.63 ± 107.187
Total-C: Change at Week 8 (n = 9)	-195.18 ± 113.993
Total-C: Change at Week 10 (n = 9)	-167.87 ± 76.914
Total-C: Change at Week 12 (n = 9)	-122.94 ± 76.104
Total-C: Change at Week 13 (n = 2)	-188.45 ± 113.349
Total-C: Change at Week 14 (n = 9)	-114.68 ± 84.082
Total-C: Change at Week 15 (n = 2)	-205.60 ± 136.047
Total-C: Change at Week 16 (n = 9)	-105.24 ± 186.103
Total-C: Change at Week 17 (n = 2)	-195.00 ± 111.157
Total-C: Change at Week 18 (n = 7)	-63.27 ± 166.911
Total-C: Change at Week 20 (n = 2)	-170.45 ± 78.135
Total-C: Change at Week 22 (n = 7)	-41.30 ± 174.597
Total-C: Change at Week 23 (n = 2)	-167.55 ± 80.539
Total-C: Change at Week 25 (n = 2)	-90.15 ± 26.234
Total-C: Change at Week 26 (n = 6)	-30.77 ± 156.736
Lp(a): Change at Week 2 (n = 9)	-22.4 ± 30.18
Lp(a): Change at Week 3 (n = 9)	-20.8 ± 30.11
Lp(a): Change at Week 4 (n = 9)	-21.1 ± 28.29
Lp(a): Change at Week 5 (n = 8)	-30.1 ± 24.91
Lp(a): Change at Week 6 (n = 9)	-25.0 ± 31.40
Lp(a): Change at Week 8 (n = 9)	-28.9 ± 23.81
Lp(a): Change at Week 12 (n = 9)	-20.9 ± 16.30
Lp(a): Change at Week 14 (n = 9)	-22.3 ± 21.01
Lp(a): Change at Week 16 (n = 9)	-26.9 ± 38.97
Lp(a): Change at Week 18 (n = 7)	-29.9 ± 29.27
Lp(a): Change at Week 20 (n = 2)	-19.0 ± 46.67
Lp(a): Change at Week 22 (n = 7)	-24.3 ± 37.53
Lp(a): Change at Week 23 (n = 2)	-12.5 ± 19.09
Lp(a): Change at Week 26 (n = 6)	-16.3 ± 37.90

No statistical analyses for this end point

Secondary: 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

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End point title

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

End point description

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 subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for this endpoint at a given time point.

End point type

Secondary

End point timeframe

Baseline (Week 0) up to Week 26

End point values	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Number of subjects analysed	9
Units: Percent Change
arithmetic mean (standard deviation)
Apo B: Percent change at Week 2 (n = 9)	-24.38 ± 20.959
Apo B: Percent change at Week 3 (n = 9)	-38.63 ± 22.114
Apo B: Percent change at Week 4 (n = 9)	-45.89 ± 18.222
Apo B: Percent change at Week 5 (n = 8)	-42.28 ± 13.247
Apo B: Percent change at Week 6 (n = 9)	-43.12 ± 14.716
Apo B: Percent change at Week 8 (n = 9)	-42.66 ± 14.475
Apo B: Percent change at Week 12 (n = 9)	-29.47 ± 21.736
Apo B: Percent change at Week 14 (n = 9)	-27.42 ± 18.418
Apo B: Percent change at Week 16 (n = 9)	-21.60 ± 24.970
Apo B: Percent change at Week 18 (n = 7)	-7.63 ± 24.347
Apo B: Percent change at Week 20 (n = 2)	-39.32 ± 6.481
Apo B: Percent change at Week 22 (n = 7)	-8.07 ± 30.444
Apo B: Percent change at Week 23 (n = 2)	-40.29 ± 10.089
Apo B: Percent change at Week 26 (n = 6)	-2.03 ± 25.320
Non-HDL-C: Percent change at Day 4 (n = 9)	-13.61 ± 10.721
Non-HDL-C: Percent change at Week 1 (n = 9)	-24.12 ± 20.083
Non-HDL-C: Percent change at Week 2 (n = 9)	-29.61 ± 23.406
Non-HDL-C: Percent change at Week 3 (n = 9)	-41.62 ± 23.969
Non-HDL-C: Percent change at Week 4 (n = 9)	-48.91 ± 22.257
Non-HDL-C: Percent change at Week 5 (n = 8)	-46.61 ± 14.172
Non-HDL-C: Percent change at Week 6 (n = 9)	-51.49 ± 14.344
Non-HDL-C: Percent change at Week 8 (n = 9)	-50.65 ± 17.231
Non-HDL-C: Percent change at Week 10 (n = 9)	-44.80 ± 13.148
Non-HDL-C: Percent change at Week 12 (n = 9)	-36.40 ± 18.711
Non-HDL-C: Percent change at Week 13 (n = 2)	-39.84 ± 11.313
Non-HDL-C: Percent change at Week 14 (n = 9)	-32.71 ± 18.214
Non-HDL-C: Percent change at Week 15 (n = 2)	-43.09 ± 14.482
Non-HDL-C: Percent change at Week 16 (n = 9)	-24.22 ± 25.899
Non-HDL-C: Percent change at Week 17 (n = 2)	-41.21 ± 10.455
Non-HDL-C: Percent change at Week 18 (n = 7)	-12.70 ± 27.798
Non-HDL-C: Percent change at Week 20 (n = 2)	-36.71 ± 5.471
Non-HDL-C: Percent change at Week 22 (n = 7)	-9.43 ± 37.102
Non-HDL-C: Percent change at Week 23 (n = 2)	-37.10 ± 5.138
Non-HDL-C: Percent change at Week 25 (n = 2)	-21.86 ± 1.676
Non-HDL-C: Percent change at Week 26 (n = 6)	-9.99 ± 25.292
Total-C: Percent change at Day 4 (n = 9)	-13.18 ± 8.932
Total-C: Percent change at Week 1 (n = 9)	-23.23 ± 16.602
Total-C: Percent change at Week 2 (n = 9)	-29.14 ± 20.307
Total-C: Percent change at Week 3 (n = 9)	-40.81 ± 21.011
Total-C: Percent change at Week 4 (n = 9)	-46.95 ± 19.083
Total-C: Percent change at Week 5 (n = 8)	-45.43 ± 12.909
Total-C: Percent change at Week 6 (n = 9)	-50.68 ± 13.327
Total-C: Percent change at Week 8 (n = 9)	-48.29 ± 15.556
Total-C: Percent change at Week 10 (n = 9)	-42.15 ± 10.858
Total-C: Percent change at Week 12 (n = 9)	-33.48 ± 16.136
Total-C: Percent change at Week 13 (n = 2)	-38.98 ± 10.525
Total-C: Percent change at Week 14 (n = 9)	-29.66 ± 15.759
Total-C: Percent change at Week 15 (n = 2)	-42.01 ± 14.356
Total-C: Percent change at Week 16 (n = 9)	-23.51 ± 25.208
Total-C: Percent change at Week 17 (n = 2)	-40.59 ± 9.468
Total-C: Percent change at Week 18 (n = 7)	-12.65 ± 26.105
Total-C: Percent change at Week 20 (n = 2)	-36.28 ± 3.862
Total-C: Percent change at Week 22 (n = 7)	-7.73 ± 33.729
Total-C: Percent change at Week 23 (n = 2)	-35.50 ± 4.662
Total-C: Percent change at Week 25 (n = 2)	-19.82 ± 1.459
Total-C: Percent change at Week 26 (n = 6)	-10.05 ± 22.903
Lp(a): Percent change at Week 2 (n = 9)	-11.64 ± 20.323
Lp(a): Percent change at Week 3 (n = 9)	-12.25 ± 19.350
Lp(a): Percent change at Week 4 (n = 9)	-10.85 ± 24.465
Lp(a): Percent change at Week 5 (n = 8)	-23.79 ± 17.908
Lp(a): Percent change at Week 6 (n = 9)	-17.12 ± 23.680
Lp(a): Percent change at Week 8 (n = 9)	-17.24 ± 17.764
Lp(a): Percent change at Week 12 (n = 9)	-13.09 ± 12.614
Lp(a): Percent change at Week 14 (n = 9)	-13.54 ± 10.151
Lp(a): Percent change at Week 16 (n = 9)	-16.93 ± 24.795
Lp(a): Percent change at Week 18 (n = 7)	-19.61 ± 21.686
Lp(a): Percent change at Week 20 (n = 2)	-19.72 ± 32.894
Lp(a): Percent change at Week 22 (n = 7)	-10.37 ± 29.230
Lp(a): Percent change at Week 23 (n = 2)	-10.62 ± 15.373
Lp(a): Percent change at Week 26 (n = 6)	3.46 ± 39.400

No statistical analyses for this end point

Secondary: 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 26) to Week 214 in the Open Label Extension (OLE) Period

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End point title

End point description

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 subjects in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for this endpoint at a given time point. (ET = Early Termination; EOS = End of Study)

End point type

Secondary

End point timeframe

Baseline (Week 26) up to Week 214

End point values	REGN1500 300 mg SC/20 mg/kg IV
Number of subjects analysed	8
Units: mg/dL
arithmetic mean (standard deviation)
Apo B: Change at Week 30 (n= 7)	-48.1 ± 61.41
Apo B: Change at Week 34 (n= 8)	-65.9 ± 58.96
Apo B: Change at Week 38 (n= 8)	-40.9 ± 56.34
Apo B: Change at Week 42 (n= 6)	-79.5 ± 71.19
Apo B: Change at Week 44 (n= 8)	-78.6 ± 58.93
Apo B: Change at Week 50 (n= 8)	-57.8 ± 50.82
Apo B: Change at Week 70 (n= 4)	-60.3 ± 78.24
Apo B: Change at Week 82 (n= 4)	-38.0 ± 35.66
Apo B: Change at Week 94 (n= 1)	-212.0 ± 99999
Apo B: Change at Week 106 (n= 1)	-173.0 ± 99999
Apo B: Change at ET (n= 8)	-3.5 ± 42.88
Apo B: Change at EOS (n= 3)	65.0 ± 100.53
Non- HDL-C: Change at Week 27 (n= 8)	-65.06 ± 61.756
Non- HDL-C: Change at Week 28 (n= 8)	-95.65 ± 87.499
Non- HDL-C: Change at Week 29 (n= 8)	-122.94 ± 106.296
Non- HDL-C: Change at Week 30 (n= 7)	-94.33 ± 113.286
Non- HDL-C: Change at Week 31 (n= 8)	-152.98 ± 135.977
Non- HDL-C: Change at Week 32 (n= 8)	-166.94 ± 133.990
Non- HDL-C: Change at Week 34 (n= 8)	-133.69 ± 112.247
Non- HDL-C: Change at Week 36 (n= 8)	-111.06 ± 93.635
Non- HDL-C: Change at Week 38 (n= 8)	-84.16 ± 101.210
Non- HDL-C: Change at Week 39 (n= 6)	-146.35 ± 128.368
Non- HDL-C: Change at Week 40 (n= 6)	-134.50 ± 128.198
Non- HDL-C: Change at Week 41 (n= 6)	-171.25 ± 143.692
Non- HDL-C:Change at Week 42 (n= 6)	-150.28 ± 132.214
Non- HDL-C: Change at Week 44 (n= 8)	-149.00 ± 114.152
Non- HDL-C: Change at Week 46 (n= 8)	-129.99 ± 116.770
Non- HDL-C: Change at Week 50 (n= 8)	-110.83 ± 104.353
Non- HDL-C: Change at Week 54 (n= 8)	-14.79 ± 178.797
Non- HDL-C: Change at Week 58 (n= 6)	-49.13 ± 97.212
Non- HDL-C: Change at Week 70 (n= 4)	-121.83 ± 152.887
Non- HDL-C: Change at Week 82 (n= 4)	-69.00 ± 66.715
Non- HDL-C: Change at Week 94 (n= 1)	-388.10 ± 99999
Non- HDL-C: Change at Week 106 (n= 1)	-303.10 ± 99999
Non- HDL-C: Change at ET (n= 8)	-12.45 ± 64.513
Non- HDL-C: Change at EOS (n= 3)	102.07 ± 153.301
Total-C: Change at Week 27 (n= 8)	-73.80 ± 60.737
Total-C: Change at Week 28 (n= 8)	-107.78 ± 83.051
Total-C: Change at Week 29 (n= 8)	-134.33 ± 102.667
Total-C: Change at Week 30 (n= 7)	-107.61 ± 109.689
Total-C: Change at Week 31 (n= 8)	-166.85 ± 130.925
Total-C: Change at Week 32 (n= 8)	-181.09 ± 130.048
Total-C: Change at Week 34 (n= 8)	-148.90 ± 106.534
Total-C: Change at Week 36 (n= 8)	-125.14 ± 89.065
Total-C: Change at Week 38 (n= 8)	-93.00 ± 99.558
Total-C: Change at Week 39 (n= 6)	-160.38 ± 125.452
Total-C: Change at Week 40 (n= 6)	-150.42 ± 124.695
Total-C: Change at Week 41 (n= 6)	-188.42 ± 141.249
Total-C: Change at Week 42 (n= 6)	-166.80 ± 128.247
Total-C: Change at Week 44 (n= 8)	-164.68 ± 110.222
Total-C: Change at Week 46 (n= 8)	-144.69 ± 110.682
Total-C: Change at Week 50 (n= 8)	-123.04 ± 99.299
Total-C: Change at Week 54 (n= 8)	-23.80 ± 180.982
Total-C: Change at Week 58 (n= 6)	-54.45 ± 97.160
Total-C: Change at Week 70 (n= 4)	-132.55 ± 152.664
Total-C: Change at Week 82 (n= 4)	-75.43 ± 67.802
Total-C: Change at Week 94 (n= 1)	-392.30 ± 99999
Total-C: Change at Week 106 (n= 1)	-303.10 ± 99999
Total-C: Change at ET (n= 8)	-20.38 ± 68.809
Total-C: Change at EOS (n= 3)	100.40 ± 144.828
Lp(a): Change at Week 30 (n= 7)	-9.0 ± 27.59
Lp(a): Change at Week 34 (n= 8)	-10.8 ± 24.00
Lp(a): Change at Week 38 (n= 8)	-16.0 ± 19.70
Lp(a): Change at Week 42 (n= 6)	-0.8 ± 41.36
Lp(a): Change at Week 44 (n= 8)	1.5 ± 37.73
Lp(a): Change at Week 50 (n= 8)	11.6 ± 48.23
Lp(a): Change at Week 70 (n= 4)	36.8 ± 77.92
Lp(a): Change at Week 82 (n= 4)	-5.3 ± 27.04
Lp(a): Change at Week 94 (n= 1)	-18.0 ± 99999
Lp(a): Change at Week 106 (n= 1)	39.0 ± 99999
Lp(a): Change at Week ET (n= 8)	11.6 ± 26.72
Lp(a): Change at EOS (n= 3)	18.3 ± 18.01

No statistical analyses for this end point

Secondary: 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 26) to Week 214 in the Open Label Extension (OLE) Period

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End point title

End point description

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 subjects in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period. Here "n" signifies the subjects who were evaluable for this endpoint at a given time point. (ET= Early Termination; EOS = End of Study)

End point type

Secondary

End point timeframe

Baseline (Week 26) up to Week 214

End point values	REGN1500 300 mg SC/20 mg/kg IV
Number of subjects analysed	8
Units: Percent change
arithmetic mean (standard deviation)
Apo B: Percent change at Week 30 (n= 7)	-30.59 ± 25.991
Apo B: Percent change at Week 34 (n= 8)	-41.27 ± 19.573
Apo B: Percent change at Week 38 (n= 8)	-29.02 ± 25.029
Apo B: Percent change at Week 42 (n= 6)	-50.90 ± 17.497
Apo B: Percent change at Week 44 (n= 8)	-48.59 ± 16.201
Apo B: Percent change at Week 50 (n= 8)	-36.68 ± 19.620
Apo B: Percent change at Week 70 (n= 4)	-33.44 ± 26.773
Apo B: Percent change at Week 82 (n= 4)	-24.87 ± 17.148
Apo B: Percent change at Week 94 (n= 1)	-77.09 ± 99999
Apo B: Percent change at Week 106 (n= 1)	-62.91 ± 99999
Apo B: Percent change at ET (n= 8)	-11.94 ± 25.708
Apo B: Percent change at EOS (n= 3)	22.60 ± 38.988
Non- HDL-C: Percent change at Week 27 (n= 8)	-23.62 ± 15.342
Non- HDL-C: Percent change at Week 28 (n= 8)	-37.22 ± 19.207
Non- HDL-C: Percent change at Week 29 (n= 8)	-44.30 ± 19.766
Non- HDL-C: Percent change at Week 30 (n= 7)	-37.90 ± 29.133
Non- HDL-C: Percent change at Week 31 (n= 8)	-50.59 ± 19.285
Non- HDL-C: Percent change at Week 32 (n= 8)	-59.09 ± 18.200
Non- HDL-C: Percent change at Week 34 (n= 8)	-51.55 ± 21.112
Non- HDL-C: Percent change at Week 36 (n= 8)	-46.62 ± 20.797
Non- HDL-C: Percent change at Week 38 (n= 8)	-36.10 ± 24.473
Non- HDL-C: Percent change at Week 39 (n= 6)	-59.25 ± 21.381
Non- HDL-C: Percent change at Week 40 (n= 6)	-53.24 ± 20.135
Non- HDL-C: Percent change at Week 41 (n= 6)	-65.07 ± 18.375
Non- HDL-C: Percent change at Week 42 (n= 6)	-58.92 ± 18.287
Non- HDL-C: Percent change at Week 44 (n= 8)	-57.03 ± 17.675
Non- HDL-C: Percent change at Week 46 (n= 8)	-48.27 ± 18.674
Non- HDL-C: Percent change at Week 50 (n= 8)	-41.16 ± 22.284
Non- HDL-C: Percent change at Week 54 (n= 8)	-12.93 ± 42.412
Non- HDL-C: Percent change at Week 58 (n= 6)	-12.28 ± 26.060
Non- HDL-C: Percent change at Week 70 (n= 4)	-37.68 ± 35.771
Non- HDL-C: Percent change at Week 82 (n= 4)	-26.16 ± 27.153
Non- HDL-C: Percent change at Week 94 (n= 1)	-81.52 ± 99999
Non- HDL-C: Percent change at Week 106 (n= 1)	-63.66 ± 99999
Non- HDL-C: Percent change at ET (n= 8)	-17.65 ± 27.339
Non- HDL-C: Percent change at EOS (n= 3)	19.98 ± 40.313
Total-C: Percent change at Week 27 (n= 8)	-22.24 ± 11.566
Total-C: Percent change at Week 28 (n= 8)	-34.78 ± 14.984
Total-C: Percent change at Week 29 (n= 8)	-40.57 ± 14.335
Total-C: Percent change at Week 30 (n= 7)	-36.37 ± 23.550
Total-C: Percent change at Week 31 (n= 8)	-48.83 ± 16.944
Total-C: Percent change at Week 32 (n= 8)	-55.05 ± 16.624
Total-C: Percent change at Week 34 (n= 8)	-48.67 ± 18.123
Total-C: Percent change at Week 36 (n= 8)	-44.16 ± 19.175
Total-C: Percent change at Week 38 (n= 8)	-33.29 ± 22.145
Total-C: Percent change at Week 39 (n= 6)	-54.40 ± 19.803
Total-C: Percent change at Week 40 (n= 6)	-50.13 ± 18.316
Total-C: Percent change at Week 41 (n= 6)	-60.87 ± 17.966
Total-C: Percent change at Week 42 (n= 6)	-54.88 ± 15.886
Total-C: Percent change at Week 44 (n= 8)	-53.41 ± 16.231
Total-C: Percent change at Week 46 (n= 8)	-46.06 ± 16.855
Total-C: Percent change at Week 50 (n= 8)	-39.04 ± 19.318
Total-C: Percent change at Week 54 (n= 8)	-12.82 ± 38.323
Total-C: Percent change at Week 58 (n= 6)	-13.78 ± 21.976
Total-C: Percent change at Week 70 (n= 4)	-36.63 ± 32.371
Total-C: Percent change at Week 82 (n= 4)	-23.09 ± 19.171
Total-C: Percent change at Week 94 (n= 1)	-77.50 ± 99999
Total-C: Percent change at Week 106 (n= 1)	-59.88 ± 99999
Total-C: Percent change at ET (n= 8)	-16.76 ± 25.085
Total-C: Percent change at EOS (n= 3)	18.20 ± 33.900
Lp(a): Percent change at Week 30 (n= 7)	-3.57 ± 24.610
Lp(a): Percent change at Week 34 (n= 8)	-6.43 ± 21.281
Lp(a): Percent change at Week 38 (n= 8)	-9.50 ± 14.429
Lp(a): Percent change at Week 42 (n= 6)	-4.76 ± 24.227
Lp(a): Percent change at Week 44 (n= 8)	-5.13 ± 25.377
Lp(a): Percent change at Week 50 (n= 8)	1.02 ± 29.756
Lp(a): Percent change at Week 70 (n= 4)	15.80 ± 36.280
Lp(a): Percent change at Week 82 (n= 4)	-3.77 ± 16.168
Lp(a): Percent change at Week 94 (n= 1)	-11.92 ± 99999
Lp(a): Percent change at Week 106 (n= 1)	25.83 ± 99999
Lp(a): Percent change at ET (n= 8)	9.96 ± 21.597
Lp(a): Percent change at EOS (n= 3)	40.51 ± 39.616

No statistical analyses for this end point

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

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End point title

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

End point description

Percentage of subjects 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 subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

End point type

Secondary

End point timeframe

Baseline (Week 0) up to Week 26

End point values	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Number of subjects analysed	9
Units: Percentage of subjects
number (not applicable)	100

No statistical analyses for this end point

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

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End point title

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

End point description

Percentage of subjects 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 subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

End point type

Secondary

End point timeframe

Baseline (Week 26) to Week 214

End point values	REGN1500 300 mg SC/20 mg/kg IV
Number of subjects analysed	8
Units: Percentage of Subjects
number (not applicable)	100

No statistical analyses for this end point

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

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End point title

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

End point description

Percentage of subjects 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 subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

End point type

Secondary

End point timeframe

Baseline (Week 0) to Week 26

End point values	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Number of subjects analysed	9
Units: Percentage of subjects
number (not applicable)	77.8

No statistical analyses for this end point

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

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End point title

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

End point description

Percentage of subjects 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 subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.

End point type

Secondary

End point timeframe

Baseline (Week 26) up to Week 214

End point values	REGN1500 300 mg SC/20 mg/kg IV
Number of subjects analysed	8
Units: Percentage of subjects
number (not applicable)	87.5

No statistical analyses for this end point

Secondary: 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

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End point 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

End point description

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 subjects in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for this endpoint at a given time point.

End point type

Secondary

End point timeframe

Baseline (Week 0) up to Week 26

End point values	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Number of subjects analysed	9
Units: mg/dL
arithmetic mean (standard deviation)
HDL-C: Change at Day 4 (n = 9)	-5.81 ± 4.789
HDL-C: Change at Week 1 (n = 9)	-9.92 ± 6.578
HDL-C: Change at Week 2 (n = 9)	-11.99 ± 7.351
HDL-C: Change at Week 3 (n = 9)	-16.02 ± 10.737
HDL-C: Change at Week 4 (n = 9)	-15.07 ± 10.792
HDL-C: Change at Week 5 (n = 8)	-11.66 ± 8.442
HDL-C: Change at Week 6 (n = 9)	-16.46 ± 11.868
HDL-C: Change at Week 8 (n = 9)	-12.06 ± 11.334
HDL-C: Change at Week 10 (n = 9)	-10.94 ± 8.215
HDL-C: Change at Week 12 (n = 9)	-6.82 ± 6.811
HDL-C: Change at Week 13 (n = 2)	-8.10 ± 2.970
HDL-C: Change at Week 14 (n = 9)	-6.18 ± 4.205
HDL-C: Change at Week 15 (n = 2)	-8.70 ± 0.566
HDL-C: Change at Week 16 (n = 9)	-5.54 ± 5.785
HDL-C: Change at Week 17 (n = 2)	-9.40 ± 3.818
HDL-C: Change at Week 18 (n = 7)	-2.93 ± 7.435
HDL-C: Change at Week 20 (n = 2)	-8.70 ± 8.202
HDL-C: Change at Week 22 (n = 7)	-0.30 ± 6.943
HDL-C: Change at Week 23 (n = 2)	-4.00 ± 4.384
HDL-C: Change at Week 25 (n = 2)	2.50 ± 3.818
HDL-C: Change at Week 26 (n = 6)	-5.30 ± 5.266
TG: Change at Day 4 (n = 9)	-25.17 ± 18.545
TG: Change at Week 1 (n = 9)	-21.41 ± 17.603
TG: Change at Week 2 (n = 9)	-15.83 ± 15.908
TG: Change at Week 3 (n = 9)	-39.73 ± 31.977
TG: Change at Week 4 (n = 9)	-40.81 ± 30.277
TG: Change at Week 5 (n = 8)	-42.04 ± 28.887
TG: Change at Week 6 (n = 9)	-38.73 ± 35.120
TG: Change at Week 8 (n = 9)	-30.37 ± 26.383
TG: Change at Week 10 (n = 9)	-28.79 ± 21.095
TG: Change at Week 12 (n = 9)	-33.23 ± 32.901
TG: Change at Week 13 (n = 2)	-49.15 ± 56.922
TG: Change at Week 14 (n = 9)	-22.11 ± 35.348
TG: Change at Week 15 (n = 2)	-52.20 ± 82.590
TG: Change at Week 16 (n = 9)	-11.20 ± 48.136
TG: Change at Week 17 (n = 2)	-55.35 ± 58.195
TG: Change at Week 18 (n = 7)	9.77 ± 24.537
TG: Change at Week 20 (n = 2)	-33.65 ± 33.729
TG: Change at Week 22 (n = 7)	1.90 ± 33.916
TG: Change at Week 23 (n = 2)	-36.30 ± 31.254
TG: Change at Week 25 (n = 2)	-23.45 ± 34.436
TG: Change at Week 26 (n = 6)	-5.87 ± 28.748
Apo A-1: Change at Week 2 (n = 9)	-28.6 ± 18.64
Apo A-1: Change at Week 3 (n = 9)	-43.3 ± 22.54
Apo A-1: Change at Week 4 (n = 9)	-43.4 ± 16.88
Apo A-1: Change at Week 5 (n = 8)	-35.8 ± 17.53
Apo A-1: Change at Week 6 (n = 9)	-40.7 ± 19.86
Apo A-1: Change at Week 8 (n = 9)	-33.6 ± 20.64
Apo A-1: Change at Week 12 (n = 9)	-17.2 ± 20.04
Apo A-1: Change at Week 14 (n = 9)	-17.7 ± 11.97
Apo A-1: Change at Week 16 (n = 9)	-11.3 ± 16.50
Apo A-1: Change at Week 18 (n = 7)	-4.9 ± 17.72
Apo A-1: Change at Week 20 (n = 2)	-24.0 ± 8.49
Apo A-1: Change at Week 22 (n = 7)	1.9 ± 15.42
Apo A-1: Change at Week 23 (n = 2)	-23.0 ± 8.49
Apo A-1: Change at Week 26 (n = 6)	-10.8 ± 13.83

No statistical analyses for this end point

Secondary: 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

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End point 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

End point description

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 subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period. Here, "n" signifies the subjects who were evaluable for this endpoint at a given time point.

End point type

Secondary

End point timeframe

Baseline (Week 0) up to Week 26

End point values	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Number of subjects analysed	9
Units: Percent Change
arithmetic mean (standard deviation)
HDL-C: Percent Change at Day 4 (n = 9)	-14.37 ± 10.830
HDL-C: Percent Change at Week 1 (n = 9)	-23.34 ± 10.783
HDL-C: Percent Change at Week 2 (n = 9)	-28.75 ± 8.458
HDL-C: Percent Change at Week 3 (n = 9)	-36.96 ± 16.495
HDL-C: Percent Change at Week 4 (n = 9)	-35.24 ± 16.212
HDL-C: Percent Change at Week 5 (n = 8)	-29.74 ± 14.240
HDL-C: Percent Change at Week 6 (n = 9)	-37.88 ± 15.864
HDL-C: Percent Change at Week 8 (n = 9)	-25.42 ± 23.199
HDL-C: Percent Change at Week 10 (n = 9)	-24.57 ± 22.409
HDL-C: Percent Change at Week 12 (n = 9)	-13.59 ± 23.189
HDL-C: Percent Change at Week 13 (n = 2)	-24.48 ± 6.243
HDL-C: Percent Change at Week 14 (n = 9)	-16.81 ± 14.822
HDL-C: Percent Change at Week 15 (n = 2)	-26.97 ± 4.895
HDL-C: Percent Change at Week 16 (n = 9)	-12.03 ± 16.026
HDL-C: Percent Change at Week 17 (n = 2)	-28.34 ± 8.393
HDL-C: Percent Change at Week 18 (n = 7)	-1.05 ± 24.171
HDL-C: Percent Change at Week 20 (n = 2)	-25.39 ± 22.187
HDL-C: Percent Change at Week 22 (n = 7)	3.78 ± 20.466
HDL-C: Percent Change at Week 23 (n = 2)	-11.56 ± 12.094
HDL-C: Percent Change at Week 25 (n = 2)	8.41 ± 12.698
HDL-C: Percent Change at Week 26 (n = 6)	-12.00 ± 11.109
TG: Percent Change at Day 4 (n = 9)	-29.61 ± 18.562
TG: Percent Change at Week 1 (n = 9)	-23.41 ± 20.150
TG: Percent Change at Week 2 (n = 9)	-17.19 ± 18.735
TG: Percent Change at Week 3 (n = 9)	-44.38 ± 19.223
TG: Percent Change at Week 4 (n = 9)	-46.57 ± 17.014
TG: Percent Change at Week 5 (n = 8)	-44.49 ± 16.583
TG: Percent Change at Week 6 (n = 9)	-40.03 ± 26.830
TG: Percent Change at Week 8 (n = 9)	-30.69 ± 24.800
TG: Percent Change at Week 10 (n = 9)	-31.40 ± 16.697
TG: Percent Change at Week 12 (n = 9)	-35.38 ± 25.014
TG: Percent Change at Week 13 (n = 2)	-35.11 ± 26.733
TG: Percent Change at Week 14 (n = 9)	-23.51 ± 28.335
TG: Percent Change at Week 15 (n = 2)	-27.77 ± 55.240
TG: Percent Change at Week 16 (n = 9)	-6.70 ± 40.326
TG: Percent Change at Week 17 (n = 2)	-42.09 ± 22.941
TG: Percent Change at Week 18 (n = 7)	23.13 ± 55.349
TG: Percent Change at Week 20 (n = 2)	-26.30 ± 11.945
TG: Percent Change at Week 22 (n = 7)	8.86 ± 43.819
TG: Percent Change at Week 23 (n = 2)	-30.58 ± 6.662
TG: Percent Change at Week 25 (n = 2)	-13.62 ± 21.582
TG: Percent Change at Week 26 (n = 6)	-1.60 ± 33.318
Apo A-1: Percent Change at Week 2 (n = 9)	-24.37 ± 12.794
Apo A-1: Percent Change at Week 3 (n = 9)	-36.47 ± 16.930
Apo A-1: Percent Change at Week 4 (n = 9)	-38.54 ± 8.906
Apo A-1: Percent Change at Week 5 (n = 8)	-31.91 ± 11.491
Apo A-1: Percent Change at Week 6 (n = 9)	-34.72 ± 12.342
Apo A-1: Percent Change at Week 8 (n = 9)	-29.03 ± 14.765
Apo A-1: Percent Change at Week 12 (n = 9)	-11.85 ± 25.176
Apo A-1: Percent Change at Week 14 (n = 9)	-17.65 ± 13.824
Apo A-1: Percent Change at Week 16 (n = 9)	-9.86 ± 14.372
Apo A-1: Percent Change at Week 18 (n = 7)	-1.16 ± 19.598
Apo A-1: Percent Change at Week 20 (n = 2)	-22.15 ± 7.965
Apo A-1: Percent Change at Week 22 (n = 7)	4.21 ± 17.857
Apo A-1: Percent Change at Week 23 (n = 2)	-21.22 ± 7.959
Apo A-1: Percent Change at Week 26 (n = 6)	-9.68 ± 11.250

No statistical analyses for this end point

Secondary: 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

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End point 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

End point description

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 subjects in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for the endpoint at a given time point. (ET= Early Termination; EOS = End of Study)

End point type

Secondary

End point timeframe

Baseline (Week 26) up to Week 214

End point values	REGN1500 300 mg SC/20 mg/kg IV
Number of subjects analysed	8
Units: Percent change
arithmetic mean (standard deviation)
HDL-C: Percent change at Week 27 (n= 8)	-21.47 ± 11.815
HDL-C: Percent change at Week 28 (n= 8)	-27.60 ± 11.485
HDL-C: Percent change at Week 29 (n= 8)	-26.51 ± 16.584
HDL-C: Percent change at Week 30 (n= 7)	-27.57 ± 14.566
HDL-C: Percent change at Week 31 (n= 8)	-32.81 ± 18.939
HDL-C: Percent change at Week 32 (n= 8)	-32.72 ± 14.684
HDL-C: Percent change at Week 34 (n= 8)	-34.60 ± 15.971
HDL-C: Percent change at Week 36 (n= 8)	-31.14 ± 18.695
HDL-C: Percent change at Week 38 (n= 8)	-18.57 ± 15.411
HDL-C: Percent change at Week 39 (n= 6)	-32.09 ± 16.062
HDL-C: Percent change at Week 40 (n= 6)	-36.25 ± 9.327
HDL-C: Percent change at Week 41 (n= 6)	-39.14 ± 13.804
HDL-C: Percent change at Week 42 (n= 6)	-38.31 ± 10.670
HDL-C: Percent change at Week 44 (n= 8)	-37.01 ± 12.049
HDL-C: Percent change at Week 46 (n= 8)	-32.77 ± 13.829
HDL-C: Percent change at Week 50 (n= 8)	-25.17 ± 17.957
HDL-C: Percent change at Week 54 (n= 8)	-20.16 ± 18.657
HDL-C: Percent change at Week 58 (n= 6)	-10.87 ± 14.194
HDL-C: Percent change at Week 70 (n= 4)	-24.67 ± 15.473
HDL-C: Percent change at Week 82 (n= 4)	-17.56 ± 10.574
HDL-C: Percent change at Week 94 (n= 1)	-13.95 ± 99999
HDL-C: Percent change at Week 106 (n= 1)	0.00 ± 99999
HDL-C: Percent change at ET (n= 8)	-15.08 ± 21.569
HDL-C: Percent change at EOS (n= 3)	-0.78 ± 27.385
Triglycerides: Percent change at Week 27 (n= 8)	-38.62 ± 26.036
Triglycerides: Percent change at Week 28 (n= 8)	-41.76 ± 27.247
Triglycerides: Percent change at Week 29 (n= 8)	-41.17 ± 18.721
Triglycerides: Percent change at Week 30 (n= 7)	-38.82 ± 23.649
Triglycerides: Percent change at Week 31 (n= 8)	-44.86 ± 28.212
Triglycerides: Percent change at Week 32 (n= 8)	-45.33 ± 23.671
Triglycerides: Percent change at Week 34 (n= 8)	-28.58 ± 23.643
Triglycerides: Percent change at Week 36 (n= 8)	-25.89 ± 36.606
Triglycerides: Percent change at Week 38 (n= 8)	-24.32 ± 25.386
Triglycerides: Percent change at Week 39 (n= 6)	-48.37 ± 26.457
Triglycerides: Percent change at Week 40 (n= 6)	-43.36 ± 26.527
Triglycerides: Percent change at Week 41 (n= 6)	-46.65 ± 25.386
Triglycerides: Percent change at Week 42 (n= 6)	-43.09 ± 29.631
Triglycerides: Percent change at Week 44 (n= 8)	-47.98 ± 35.076
Triglycerides: Percent change at Week 46 (n= 8)	-37.64 ± 26.234
Triglycerides: Percent change at Week 50 (n= 8)	-22.10 ± 27.247
Triglycerides: Percent change at Week 54 (n= 8)	-1.08 ± 30.518
Triglycerides: Percent change at Week 58 (n= 6)	3.72 ± 25.130
Triglycerides: Percent change at Week 70 (n= 4)	-22.28 ± 31.761
Triglycerides: Percent change at Week 82 (n= 4)	-36.47 ± 9.998
Triglycerides: Percent change at Week 94 (n= 1)	-76.36 ± 99999
Triglycerides: Percent change at Week 106 (n= 1)	-57.98 ± 99999
Triglycerides: Percent change at ET (n= 8)	-14.52 ± 25.892
Triglycerides: Percent change at EOS (n= 3)	30.98 ± 40.511
Apo A-1: Percent change at Week 30 (n= 7)	-27.20 ± 12.119
Apo A-1: Percent change at Week 34 (n= 8)	-29.05 ± 17.483
Apo A-1: Percent change at Week 38 (n= 8)	-11.79 ± 23.939
Apo A-1: Percent change at Week 42 (n= 6)	-35.47 ± 9.512
Apo A-1: Percent change at Week 44 (n= 8)	-34.28 ± 11.306
Apo A-1: Percent change at Week 50 (n= 8)	-21.01 ± 15.471
Apo A-1: Percent change at Week 70 (n= 4)	-25.28 ± 11.950
Apo A-1: Percent change at Week 82 (n= 4)	-18.98 ± 6.534
Apo A-1: Percent change at Week 94 (n= 1)	-22.45 ± 99999
Apo A-1: Percent change at Week 106 (n= 1)	3.06 ± 99999
Apo A-1: Percent change at ET (n= 8)	-9.63 ± 25.308
Apo A-1: Percent change at EOS (n= 3)	11.27 ± 25.093

No statistical analyses for this end point

Secondary: 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

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End point 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

End point description

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 subjects in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period. Here, "n" signifies those subjects who were evaluable for this endpoint at a given time point. (ET= Early Termination; EOS = End of Study)

End point type

Secondary

End point timeframe

Baseline (Week 26) up to Week 214

End point values	REGN1500 300 mg SC/20 mg/kg IV
Number of subjects analysed	8
Units: mg/dL
arithmetic mean (standard deviation)
HDL-C: Change at Week 27 (n= 8)	-8.78 ± 5.736
HDL-C: Change at Week 28 (n= 8)	-12.08 ± 7.244
HDL-C: Change at Week 29 (n= 8)	-11.44 ± 7.858
HDL-C: Change at Week 30 (n= 7)	-13.29 ± 9.270
HDL-C: Change at Week 31 (n= 8)	-13.94 ± 8.655
HDL-C: Change at Week 32 (n= 8)	-14.11 ± 8.163
HDL-C: Change at Week 34 (n= 8)	-15.10 ± 9.167
HDL-C: Change at Week 36 (n= 8)	-14.14 ± 10.426
HDL-C: Change at Week 38 (n= 8)	-8.89 ± 7.513
HDL-C: Change at Week 39 (n= 6)	-14.02 ± 8.023
HDL-C: Change at Week 40 (n= 6)	-15.90 ± 7.171
HDL-C: Change at Week 41 (n= 6)	-17.17 ± 8.262
HDL-C: Change at Week 42 (n= 6)	-16.65 ± 7.148
HDL-C: Change at Week 44 (n= 8)	-15.83 ± 8.167
HDL-C: Change at Week 46 (n= 8)	-14.85 ± 10.173
HDL-C: Change at Week 50 (n= 8)	-12.25 ± 10.985
HDL-C: Change at Week 54 (n= 8)	-9.06 ± 9.107
HDL-C: Change at Week 58 (n= 6)	-5.42 ± 6.058
HDL-C: Change at Week 70 (n= 4)	-10.83 ± 10.448
HDL-C: Change at Week 82 (n= 4)	-6.28 ± 2.655
HDL-C: Change at Week 94 (n= 1)	-4.20 ± 99999
HDL-C: Change at Week 106 (n= 1)	0.00 ± 99999
HDL-C: Percent change at ET (n= 8)	-7.86 ± 8.258
HDL-C: Change at EOS (n= 3)	-1.67 ± 10.698
Triglycerides: Change at Week 27 (n= 8)	-31.19 ± 28.590
Triglycerides: Change at Week 28 (n= 8)	-36.28 ± 30.630
Triglycerides: Change at Week 29 (n= 8)	-32.86 ± 25.188
Triglycerides: Change at Week 30 (n= 7)	-32.37 ± 30.475
Triglycerides: Change at Week 31 (n= 8)	-39.04 ± 30.169
Triglycerides: Change at Week 32 (n= 8)	-37.83 ± 30.808
Triglycerides: Change at Week 34 (n= 8)	-25.41 ± 26.559
Triglycerides: Change at Week 36 (n= 8)	-24.66 ± 28.139
Triglycerides: Change at Week 38 (n= 8)	-21.13 ± 25.190
Triglycerides: Change at Week 39 (n= 6)	-40.27 ± 32.803
Triglycerides: Change at Week 40 (n= 6)	-37.30 ± 33.559
Triglycerides: Change at Week 41 (n= 6)	-39.37 ± 33.251
Triglycerides: Change at Week 42 (n= 6)	-37.62 ± 33.724
Triglycerides: Change at Week 44 (n= 8)	-42.25 ± 32.682
Triglycerides: Change at Week 46 (n= 8)	-31.53 ± 28.209
Triglycerides: Change at Week 50 (n= 8)	-20.00 ± 27.720
Triglycerides: Change at Week 54 (n= 8)	-6.40 ± 26.869
Triglycerides: Change at Week 58 (n= 6)	-3.53 ± 21.753
Triglycerides: Change at Week 70 (n= 4)	-21.23 ± 35.614
Triglycerides: Change at Week 82 (n= 4)	-26.33 ± 21.457
Triglycerides: Change at Week 94 (n= 1)	-88.50 ± 99999
Triglycerides: Change at Week 106 (n= 1)	-67.20 ± 99999
Triglycerides: Change at ET (n= 8)	-11.71 ± 21.343
Triglycerides: Change at EOS (n= 3)	30.67 ± 35.390
Apo A-1: Change at Week 30 (n= 7)	-34.4 ± 20.18
Apo A-1: Change at Week 34 (n= 8)	-35.1 ± 24.70
Apo A-1: Change at Week 38 (n= 8)	-18.0 ± 26.54
Apo A-1: Change at Week 42 (n= 6)	-43.7 ± 15.71
Apo A-1: Change at Week 44 (n= 8)	-39.4 ± 18.12
Apo A-1: Change at Week 50 (n= 8)	-26.9 ± 23.56
Apo A-1: Change at Week 70 (n= 4)	-30.3 ± 20.12
Apo A-1: Change at Week 82 (n= 4)	-21.0 ± 5.83
Apo A-1: Change at Week 94 (n= 1)	-22.0 ± 99999
Apo A-1: Change at Week 106 (n= 1)	3.0 ± 99999
Apo A-1: Change at ET (n= 8)	-15.6 ± 24.34
Apo A-1: Change at EOS (n= 3)	7.0 ± 24.98

No statistical analyses for this end point

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

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End point title

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

End point description

Absolute change was reported in Apo CIII from baseline (week 0) up to week 16. The efficacy analysis set included all subjects in the SAF who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period. Here, "n" signifies the subjects who were evaluable for this endpoint at a given time point.

End point type

Secondary

End point timeframe

Baseline (Week 0) up to Week 16

End point values	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Number of subjects analysed	9
Units: mg/dL
arithmetic mean (standard deviation)
Apo CIII: Change at Day 4 (n = 9)	-3.729 ± 1.6905
Apo CIII: Change at Week 1 (n = 9)	-4.296 ± 1.9829
Apo CIII: Change at Week 2 (n = 9)	-4.301 ± 2.2542
Apo CIII: Change at Week 5 (n = 8)	-6.000 ± 2.8541
Apo CIII: Change at Week 6 (n = 1)	-8.380 ± 99999
Apo CIII: Change at Week 12 (n = 9)	-3.772 ± 2.8443
Apo CIII: Change at Week 14 (n = 9)	-3.866 ± 3.7327
Apo CIII: Change at Week 16 (n = 9)	-2.757 ± 4.4877

No statistical analyses for this end point

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

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End point title

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

End point description

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 subjects in the SAF who had baseline and at least one post-baseline measure of the lipid panel in the main study period. Here, "n" signifies the subjects who were evaluable for this endpoint at a given time point.

End point type

Secondary

End point timeframe

Baseline (Week 0) to Week 16

End point values	REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Number of subjects analysed	9
Units: Percent Change
arithmetic mean (standard deviation)
Apo CIII: Percent Change at Day 4 (n = 9)	-46.59 ± 22.129
Apo CIII: Percent Change at Week 1 (n = 9)	-55.43 ± 18.541
Apo CIII: Percent Change at Week 2 (n = 9)	-56.57 ± 24.815
Apo CIII: Percent Change at Week 5 (n = 8)	-75.44 ± 9.667
Apo CIII: Percent Change at Week 6 (n = 1)	-91.48 ± 99999
Apo CIII: Percent Change at Week 12 (n = 9)	-49.07 ± 25.517
Apo CIII: Percent Change at Week 14 (n = 9)	-40.67 ± 44.674
Apo CIII: Percent Change at Week 16 (n = 9)	-28.30 ± 54.676

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

All adverse events (AEs) were collected from signature of the informed consent form up to the final visit regardless of seriousness or relationship to investigational product.

Adverse event reporting additional description

During the main study period, the safety analysis set (SAF) was defined as all enrolled subjects who received at least 1 dose or part of a dose. For OLE, the SAF was defined as all enrolled subjects who received at least 1 dose or part of a dose in the OLE period. The SAF in both study periods was based on the treatment received (as treated).

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

17.0

Reporting group title

OLE Period: REGN1500 300 mg SC/20 mg/kg IV

Reporting group description

Reporting group title

Main Study Period: REGN1500 250 mg SC/15 mg/kg IV/450 mg SC

Reporting group description

Serious adverse events	OLE Period: REGN1500 300 mg SC/20 mg/kg IV	Main Study Period: REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Total subjects affected by serious adverse events
subjects affected / exposed	2 / 8 (25.00%)	1 / 9 (11.11%)
number of deaths (all causes)	0	0
number of deaths resulting from adverse events	0	0
Cardiac disorders
Coronary artery disease
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Coronary artery stenosis
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Respiratory, thoracic and mediastinal disorders
Bronchospasm
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 5%

Non-serious adverse events	OLE Period: REGN1500 300 mg SC/20 mg/kg IV	Main Study Period: REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Total subjects affected by non serious adverse events
subjects affected / exposed	8 / 8 (100.00%)	9 / 9 (100.00%)
Vascular disorders
Flushing
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	2
General disorders and administration site conditions
Chest discomfort
subjects affected / exposed	1 / 8 (12.50%)	1 / 9 (11.11%)
occurrences all number	1	1
Chest pain
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1
Fatigue
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Injection site haematoma
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Influenza like illness
subjects affected / exposed	2 / 8 (25.00%)	2 / 9 (22.22%)
occurrences all number	2	3
Injection site haemorrhage
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	2
Local swelling
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Oedema peripheral
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Pyrexia
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Immune system disorders
Food allergy
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	2	0
Respiratory, thoracic and mediastinal disorders
Bronchospasm
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Cough
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Dyspnoea
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1
Epistaxis
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1
Oropharyngeal pain
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1
Rhinalgia
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1
Upper-Airway cough syndrome
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Psychiatric disorders
Insomnia
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Investigations
Blood creatine phosphokinase increased
subjects affected / exposed	1 / 8 (12.50%)	1 / 9 (11.11%)
occurrences all number	2	3
Crystal urine present
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1
Liver function test abnormal
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1
Transaminases increased
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Injury, poisoning and procedural complications
Crush injury
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Contusion
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Procedural hypotension
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1
Thermal burn
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1
Nervous system disorders
Dizziness
subjects affected / exposed	1 / 8 (12.50%)	1 / 9 (11.11%)
occurrences all number	1	1
Headache
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1
Memory impairment
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Blood and lymphatic system disorders
Anaemia
subjects affected / exposed	1 / 8 (12.50%)	1 / 9 (11.11%)
occurrences all number	1	1
Eye disorders
Corneal opacity
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1
Eye inflammation
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Gastrointestinal disorders
Abdominal pain
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	2	0
Diarrhoea
subjects affected / exposed	4 / 8 (50.00%)	1 / 9 (11.11%)
occurrences all number	6	2
Faeces discoloured
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1
Nausea
subjects affected / exposed	3 / 8 (37.50%)	4 / 9 (44.44%)
occurrences all number	5	6
Vomiting
subjects affected / exposed	1 / 8 (12.50%)	1 / 9 (11.11%)
occurrences all number	1	1
Skin and subcutaneous tissue disorders
Alopecia
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Ecchymosis
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1
Generalised erythema
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Pruritus allergic
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Rash
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	3	0
Renal and urinary disorders
Renal failure chronic
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Musculoskeletal and connective tissue disorders
Back pain
subjects affected / exposed	4 / 8 (50.00%)	4 / 9 (44.44%)
occurrences all number	8	6
Muscle spasms
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1
Musculoskeletal pain
subjects affected / exposed	2 / 8 (25.00%)	2 / 9 (22.22%)
occurrences all number	3	2
Myalgia
subjects affected / exposed	0 / 8 (0.00%)	2 / 9 (22.22%)
occurrences all number	0	2
Neck pain
subjects affected / exposed	1 / 8 (12.50%)	1 / 9 (11.11%)
occurrences all number	1	1
Pain in extremity
subjects affected / exposed	1 / 8 (12.50%)	1 / 9 (11.11%)
occurrences all number	1	1
Infections and infestations
Gastroenteritis
subjects affected / exposed	1 / 8 (12.50%)	1 / 9 (11.11%)
occurrences all number	1	2
Nasopharyngitis
subjects affected / exposed	3 / 8 (37.50%)	2 / 9 (22.22%)
occurrences all number	4	3
Influenza
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Pharyngitis
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1
Rhinitis
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Sinusitis
subjects affected / exposed	2 / 8 (25.00%)	1 / 9 (11.11%)
occurrences all number	2	2
Upper respiratory tract infection
subjects affected / exposed	2 / 8 (25.00%)	0 / 9 (0.00%)
occurrences all number	3	0
Urinary tract infection
subjects affected / exposed	1 / 8 (12.50%)	0 / 9 (0.00%)
occurrences all number	1	0
Metabolism and nutrition disorders
Hypokalaemia
subjects affected / exposed	0 / 8 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1

More information

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Were there any global substantial amendments to the protocol? Yes

05 May 2014

The purpose of this amendment was to include sampling for, and assessment of, low-density lipoprotein receptor (LDLR) function.

01 Oct 2014

The purpose of this amendment was to: Allow up to 4 patients who are taking stable, background lomitapide to be enrolled; Allow patients with a known history of HIV to be enrolled. Inclusion of these patients will be dependent on their ability to meet entry criteria for CD4 count, viral load, absence of opportunistic infections or tuberculosis, and stability of their antiretroviral therapy; Require more stringent (double-barrier) contraception; Move the required DNA sample for confirmation of diagnosis of homozygous familial hypercholesterolemia from visit 2 (baseline) to visit 1 (screening); Add review of inclusion/exclusion criteria at visit 2 (baseline); Increase the time after the final date of the clinical study report that sub-study samples may be stored and used for research purposes from up to 10 years to up to 15 years; Remove the 1-day window from visit 2 (baseline); Update exposure data from the ongoing phase 1 study; Clarify the end of treatment and end of study visits for patients who prematurely discontinue from the study; Make minor editorial changes

24 Feb 2015

The purpose of this amendment was to: Exclude women of childbearing potential from eligibility in the study, in agreement with the Food and Drug Administration (FDA), based on safety findings from a pilot embryo-fetal toxicity study with REGN1500; Include measurement of follicle-stimulating hormone (FSH) at screening; Remove urine pregnancy testing; Allow for patients to be rescreened on a case-by-case basis; Allow for replacement of patients who discontinued prior to visit 4

02 Nov 2015

The purpose of this amendment was to: Remove the 4 weekly 450 mg SC doses; Extend the duration of follow-up/observation period to 24 weeks after the last dose of study drug to ensure patients are monitored for the duration of drug exposure; Allow women of childbearing potential to participate in the study; Update exclusion criteria; Update prohibited and permitted concomitant medications

14 May 2016

The purpose of this amendment was to: Add an open-label extension (OLE) period; Update the protocol with new clinical and preclinical data; Revise the PK and ADA variables; Revise the statistical hypothesis and justification of sample size; Increase the number and region of the study sites; Remove the 250 mg/kg subcutaneous (SC) dose from the main period of the study; Include a possible interim analysis; Allow the required DNA sample to be collected at visit 1a or visit 1; Revise the secondary endpoints to reflect the removal of the 4 weekly 450 mg SC doses that was implemented in Amendment 4; Clarify pharmacokinetic (PK) sampling times in the main study period; Clarify anti-drug antibody (ADA) sampling; Update definition of baseline and clarify use of data from unscheduled assessments; Update the safety section of the protocol with the new language in the updated protocol template; State that serum pregnancy testing in the main period of the study will only be performed in women of childbearing potential (WOCBP); Make miscellaneous editorial/formatting revisions

Were there any global interruptions to the trial? No

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

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.

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Clinical trials

Clinical Trial Results: 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

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Secondary: 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

Secondary: 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

Secondary: 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

Secondary: 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

Secondary: 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 26) to Week 214 in the Open Label Extension (OLE) Period

Secondary: 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 26) to Week 214 in the Open Label Extension (OLE) Period

Secondary: 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 26) to Week 214 in the Open Label Extension (OLE) Period

Secondary: 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 26) to Week 214 in the Open Label Extension (OLE) Period

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

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

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

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

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

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

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

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

Secondary: 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

Secondary: 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

Secondary: 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

Secondary: 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

Secondary: 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

Secondary: 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

Secondary: 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

Secondary: 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

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

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

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

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

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

Clinical Trial Results:
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