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Trial record 2 of 22 for:    ALN-PCSsc

A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH) (ORION-2)

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ClinicalTrials.gov Identifier: NCT02963311
Recruitment Status : Completed
First Posted : November 15, 2016
Results First Posted : May 18, 2020
Last Update Posted : May 18, 2020
Sponsor:
Information provided by (Responsible Party):
The Medicines Company

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Homozygous Familial Hypercholesterolemia
Interventions Drug: ALN-PCSSC
Drug: Standard of Care
Enrollment 9
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Inclisiran
Hide Arm/Group Description

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Period Title: Overall Study
Started 9
Completed 4
Not Completed 5
Reason Not Completed
Withdrawal by Subject             1
Screen Failure             4
Arm/Group Title Inclisiran
Hide Arm/Group Description

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Baseline Participants 4
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
<=18 years
0
   0.0%
Between 18 and 65 years
4
 100.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 4 participants
37.0  (13.04)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
Female
2
  50.0%
Male
2
  50.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
4
 100.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
4
 100.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 4 participants
Netherlands 1
South Africa 3
1.Primary Outcome
Title Percentage Change From Day 1 to Day 90 in LDL-C
Hide Description Due to the small number of subjects, and the fact that the data are not normally distributed, the data are presented as descriptive statistics with no inferential and limited summary statistics presented.
Time Frame Day 1, Day 90
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (95% Confidence Interval)
Unit of Measure: percent change
-12.26
(-43.75 to 19.24)
2.Primary Outcome
Title Percentage Change From Day 1 to Day 180 (or Final Visit) in LDL-C
Hide Description [Not Specified]
Time Frame Day 1, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (95% Confidence Interval)
Unit of Measure: percent change
-20.96
(-49.97 to 8.05)
3.Secondary Outcome
Title Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in LDL-C
Hide Description [Not Specified]
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: mg/dL
Day 90 -56.3  (115.18)
Day 180 -105.3  (116.44)
4.Secondary Outcome
Title Percentage Change From Day 1 to Day 60 and to Day 90 in PCSK9
Hide Description [Not Specified]
Time Frame Day 1, Day 60, Day 90
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: percent change
Day 60 -64.9  (18.33)
Day 90 -59.0  (16.46)
5.Secondary Outcome
Title Absolute Change From Day 1 to Day 60 and to Day 90 in PCSK9
Hide Description [Not Specified]
Time Frame Day 1, Day 60, Day 90
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: ng/mL
Day 60 -654.1  (564.67)
Day 90 -602.3  (559.61)
6.Secondary Outcome
Title Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Total Cholesterol
Hide Description [Not Specified]
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: percent change
Day 90 -13.9  (14.84)
Day 180 -19.8  (13.68)
7.Secondary Outcome
Title Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Total Cholesterol
Hide Description [Not Specified]
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: mg/dL
Day 90 -77.8  (103.41)
Day 180 -118.0  (103.52)
8.Secondary Outcome
Title Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Triglycerides
Hide Description [Not Specified]
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: percent change
Day 90 -21.0  (30.99)
Day 180 -20.13  (11.67)
9.Secondary Outcome
Title Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Triglycerides
Hide Description [Not Specified]
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: mg/dL
Day 90 -35.5  (38.76)
Day 180 -26.3  (24.64)
10.Secondary Outcome
Title Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in HDL-C
Hide Description [Not Specified]
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: percent change
Day 90 -12.1  (17.98)
Day 180 -19.8  (11.78)
11.Secondary Outcome
Title Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in HDL-C
Hide Description [Not Specified]
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: mg/dL
Day 90 -6.0  (9.13)
Day 180 -9.5  (4.73)
12.Secondary Outcome
Title Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Non-HDL-C
Hide Description [Not Specified]
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: percent change
Day 90 -13.9  (14.78)
Day 180 -19.7  (13.93)
13.Secondary Outcome
Title Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Non-HDL-C
Hide Description [Not Specified]
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: mg/dL
Day 90 -71.8  (97.70)
Day 180 -108.5  (99.38)
14.Secondary Outcome
Title Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in VLDL-C
Hide Description [Not Specified]
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: percent change
Day 90 8.5  (65.28)
Day 180 55.5  (131.92)
15.Secondary Outcome
Title Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in VLDL-C
Hide Description [Not Specified]
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: mg/dL
Day 90 -15.5  (28.41)
Day 180 -3.3  (37.32)
16.Secondary Outcome
Title Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Apolipoprotein A1
Hide Description [Not Specified]
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: percent change
Day 90 -8.3  (12.95)
Day 180 -14.2  (14.44)
17.Secondary Outcome
Title Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Apolipoprotein A1
Hide Description [Not Specified]
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: mg/dL
Day 90 -11.5  (17.52)
Day 180 -19.8  (19.00)
18.Secondary Outcome
Title Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Apolipoprotein B
Hide Description [Not Specified]
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: percent change
Day 90 -26.6  (14.98)
Day 180 -25.0  (14.50)
19.Secondary Outcome
Title Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Apolipoprotein B
Hide Description [Not Specified]
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: mg/dL
Day 90 -95.3  (78.16)
Day 180 -86.3  (72.02)
20.Secondary Outcome
Title Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Lipoprotein-a
Hide Description The reported percent change value is the per participant calculated Mean.
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: percent change
Day 90 -3.5  (15.70)
Day 180 -11.8  (15.15)
21.Secondary Outcome
Title Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Lipoprotein-a
Hide Description [Not Specified]
Time Frame Day 1, Day 90, Day 180 (or Final Visit)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Inclisiran
Hide Arm/Group Description:

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Overall Number of Participants Analyzed 4
Mean (Standard Deviation)
Unit of Measure: nmol/L
Day 90 13.5  (21.92)
Day 180 -23.8  (52.73)
Time Frame 18 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Inclisiran
Hide Arm/Group Description

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Inclisiran: Inclisiran is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.

Standard of Care: Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

All-Cause Mortality
Inclisiran
Affected / at Risk (%)
Total   0/4 (0.00%)    
Hide Serious Adverse Events
Inclisiran
Affected / at Risk (%) # Events
Total   1/4 (25.00%)    
Cardiac disorders   
Angina unstable * 1  1/4 (25.00%)  1
1
Term from vocabulary, MedDRA 10.0
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Inclisiran
Affected / at Risk (%) # Events
Total   3/4 (75.00%)    
Cardiac disorders   
Angina unstable * 1  1/4 (25.00%) 
Gastrointestinal disorders   
Abdominal pain upper * 1  1/4 (25.00%) 
General disorders   
Influenza like illness * 1  1/4 (25.00%) 
Non-cardiac chest pain * 1  1/4 (25.00%) 
Infections and infestations   
Cystitis * 1  1/4 (25.00%) 
Nasopharyngitis * 1  1/4 (25.00%) 
Injury, poisoning and procedural complications   
Limb injury * 1  1/4 (25.00%) 
Road traffic accident * 1  1/4 (25.00%) 
Musculoskeletal and connective tissue disorders   
Musculoskeletal pain * 1  1/4 (25.00%) 
Pain in extremity * 1  1/4 (25.00%) 
Nervous system disorders   
Paraesthesia * 1  1/4 (25.00%) 
Respiratory, thoracic and mediastinal disorders   
Dyspnoea * 1  1/4 (25.00%) 
Oropharyngeal pain * 1  1/4 (25.00%) 
Skin and subcutaneous tissue disorders   
Acne * 1  1/4 (25.00%) 
1
Term from vocabulary, MedDRA 10.0
*
Indicates events were collected by non-systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Frank Bosley, Vice-President, Regulatory Operaions
Organization: The Medicines Company
Phone: 9732906016
EMail: frank.bosley@themedco.com
Layout table for additonal information
Responsible Party: The Medicines Company
ClinicalTrials.gov Identifier: NCT02963311    
Other Study ID Numbers: MDCO-PCS-16-02
First Submitted: November 10, 2016
First Posted: November 15, 2016
Results First Submitted: October 23, 2019
Results First Posted: May 18, 2020
Last Update Posted: May 18, 2020