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Efficacy and Safety of Gemcabene in Patients With Homozygous Familial Hypercholesterolemia on Stable, Lipid-Lowering Therapy (COBALT-1)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02722408
Recruitment Status : Completed
First Posted : March 30, 2016
Results First Posted : June 25, 2020
Last Update Posted : June 25, 2020
Sponsor:
Information provided by (Responsible Party):
NeuroBo Pharmaceuticals Inc.

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hypercholesteremia
Intervention Drug: Gemcabene
Enrollment 8
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Gemcabene
Hide Arm/Group Description

Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 milligram (mg) of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.

Participants were followed until Day 112.

Period Title: Overall Study
Started 8
Completed 8
Not Completed 0
Arm/Group Title Gemcabene
Hide Arm/Group Description Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Baseline Participants 8
Hide Baseline Analysis Population Description
Full Analysis Set (FAS): All enrolled participants who received at least 1 dose of study drug and had a valid post-baseline efficacy assessment, for a given dose level.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 8 participants
52.8  (14.48)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
Female
3
  37.5%
Male
5
  62.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
Hispanic or Latino 1
Not Hispanic or Latino 7
Unknown or Not Reported 0
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
American Indian or Alaska Native 0
Asian 0
Native Hawaiian or Other Pacific Islander 0
Black or African American 0
White 8
More than one race 0
Unknown or Not Reported 0
Low-density lipoprotein cholesterol (LDL-C)   [1] 
Mean (Standard Deviation)
Unit of measure:  Milligram per deciliter (mg/dL)
Number Analyzed 8 participants
351.25  (181.849)
[1]
Measure Description: Baseline value was obtained from Friedewald calculation.
Non-high-density lipoprotein cholesterol (non-HDL-C)  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 8 participants
379.94  (177.258)
Total Cholesterol (TC)  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 8 participants
425.38  (167.058)
Triglycerides (TG)  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 8 participants
143.56  (62.672)
High-density lipoprotein cholesterol (HDL-C)  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 8 participants
45.44  (18.661)
Very low-density lipoprotein cholesterol (VLDL-C)  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 8 participants
28.69  (12.530)
High-sensitivity C-reactive protein (hsCRP)  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 8 participants
5.95  (5.627)
Fibrinogen  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 8 participants
436.9  (89.23)
Lipoprotein(a)  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 8 participants
113.5  (92.97)
Apolipoprotein B (ApoB)  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 8 participants
221.3  (97.25)
Apolipoprotein A-I (ApoA-I)  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 8 participants
127.9  (46.98)
Apolipoprotein A-II (ApoA-II)  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 8 participants
30.3  (9.75)
Apolipoprotein C-II (ApoC-II)  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 8 participants
4.1  (1.21)
Apolipoprotein C-III (ApoC-III)  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 8 participants
10.6  (4.14)
Apolipoprotein E (ApoE)  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 8 participants
6.68  (1.738)
LDL-C by Receptor Mutation Status   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  mg/dL
Negative LDLr Number Analyzed 3 participants
551.33  (105.652)
Defective LDLr Number Analyzed 5 participants
231.20  (65.191)
Met EAS Clinical Diagnosis of HoFH Number Analyzed 6 participants
374.33  (208.717)
Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
282.00  (29.698)
[1]
Measure Description: Receptor mutation status was categorized as Low density lipoprotein receptor (LDLr) status and European Atherosclerosis Society (EAS) clinical diagnosis of HoFH.
[2]
Measure Analysis Population Description: FAS Population. Here, number analyzed signifies participants with available data .
Non-HDL-C by Receptor Mutation Status   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  mg/dL
Negative LDLr Number Analyzed 3 participants
572.83  (105.419)
Defective LDLr Number Analyzed 5 participants
264.20  (69.127)
Met EAS Clinical Diagnosis of HoFH Number Analyzed 6 participants
399.92  (205.100)
Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
320.00  (5.657)
[1]
Measure Description: Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH.
[2]
Measure Analysis Population Description: FAS Population. Here, number analyzed signifies participants with available data .
TC by Receptor Mutation Status   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  mg/dL
Negative LDLr Number Analyzed 3 participants
601.00  (100.714)
Defective LDLr Number Analyzed 5 participants
320.00  (82.189)
Met EAS Clinical Diagnosis of HoFH Number Analyzed 6 participants
439.75  (195.112)
Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
382.25  (7.425)
[1]
Measure Description: Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH.
[2]
Measure Analysis Population Description: FAS Population. Here, number analyzed signifies participants with available data .
TG by Receptor Mutation Status   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  mg/dL
Negative LDLr Number Analyzed 3 participants
107.67  (9.278)
Defective LDLr Number Analyzed 5 participants
165.10  (72.694)
Met EAS Clinical Diagnosis of HoFH Number Analyzed 6 participants
128.17  (38.577)
Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
189.75  (119.855)
[1]
Measure Description: Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH.
[2]
Measure Analysis Population Description: FAS Population. Here, number analyzed signifies participants with available data .
HDL-C by Receptor Mutation Status   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  mg/dL
Negative LDLr Number Analyzed 3 participants
28.17  (4.726)
Defective LDLr Number Analyzed 5 participants
55.80  (15.502)
Met EAS Clinical Diagnosis of HoFH Number Analyzed 6 participants
39.83  (17.394)
Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
62.25  (13.081)
[1]
Measure Description: Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH.
[2]
Measure Analysis Population Description: FAS Population. Here, number analyzed signifies participants with available data .
VLDL-C by Receptor Mutation Status   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  mg/dL
Negative LDLr Number Analyzed 3 participants
21.50  (1.803)
Defective LDLr Number Analyzed 5 participants
33.00  (14.530)
Met EAS Clinical Diagnosis of HoFH Number Analyzed 6 participants
25.58  (7.612)
Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
38.00  (24.042)
[1]
Measure Description: Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH.
[2]
Measure Analysis Population Description: FAS Population. Here, number analyzed signifies participants with available data .
1.Primary Outcome
Title Percent Change From Baseline in LDL-C at Day 28
Hide Description [Not Specified]
Time Frame Baseline, day 28
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): All enrolled participants who received at least 1 dose of study drug and had a valid post-baseline efficacy assessment, for a given dose level.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: Percent Change
-25.54  (8.301)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0041
Comments Mixed-effects model for repeated measures analysis with percent change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
2.Primary Outcome
Title Percent Change From Baseline in LDL-C at Day 56
Hide Description [Not Specified]
Time Frame Baseline, day 56
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: Percent Change
-29.94  (8.301)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0010
Comments Mixed-effects model for repeated measures analysis with percent change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
3.Primary Outcome
Title Percent Change From Baseline in LDL-C at Day 84
Hide Description [Not Specified]
Time Frame Baseline, day 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: Percent Change
-29.41  (8.301)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0012
Comments Mixed-effects model for repeated measures analysis with percent change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
4.Secondary Outcome
Title Change From Baseline in Fasting LDL-C
Hide Description [Not Specified]
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: mg/dL
Day 28 -82.15  (41.520)
Day 56 -99.77  (41.520)
Day 84 -94.90  (41.520)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0560
Comments Mixed-effects model for repeated measures analysis with Change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0219
Comments Mixed-effects model for repeated measures analysis with Change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0286
Comments Mixed-effects model for repeated measures analysis with Change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
5.Secondary Outcome
Title Percent Change From Baseline in Fasting Non-HDL-C
Hide Description [Not Specified]
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: Percent Change
Day 28 -23.87  (8.105)
Day 56 -27.33  (8.105)
Day 84 -26.59  (8.105)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0058
Comments Mixed-effects model for repeated measures analysis with percent change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0019
Comments Mixed-effects model for repeated measures analysis with percent change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0024
Comments Mixed-effects model for repeated measures analysis with percent change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
6.Secondary Outcome
Title Change From Baseline in Fasting Non-HDL-C
Hide Description [Not Specified]
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: mg/dL
Day 28 -86.64  (44.380)
Day 56 -102.89  (44.380)
Day 84 -98.26  (44.380)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0592
Comments Mixed-effects model for repeated measures analysis with change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0266
Comments Mixed-effects model for repeated measures analysis with change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0336
Comments Mixed-effects model for repeated measures analysis with change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
7.Secondary Outcome
Title Percent Change From Baseline in Fasting Total Cholesterol (TC)
Hide Description [Not Specified]
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: Percent Change
Day 28 -21.53  (7.287)
Day 56 -24.85  (7.287)
Day 84 -24.79  (7.287)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0057
Comments Mixed-effects model for repeated measures analysis with percent change in TC as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0017
Comments Mixed-effects model for repeated measures analysis with percent change in TC as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0017
Comments Mixed-effects model for repeated measures analysis with percent change in TC as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
8.Secondary Outcome
Title Change From Baseline in Fasting Total Cholesterol (TC)
Hide Description [Not Specified]
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: mg/dL
Day 28 -91.96  (46.421)
Day 56 -108.46  (46.421)
Day 84 -104.34  (46.421)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0057
Comments Mixed-effects model for repeated measures analysis with change in TC as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0255
Comments Mixed-effects model for repeated measures analysis with change in TC as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 86
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0312
Comments Mixed-effects model for repeated measures analysis with change in TC as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
9.Secondary Outcome
Title Percent Change From Baseline in Fasting Triglycerides (TG)
Hide Description [Not Specified]
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: Percent Change
Day 28 -11.99  (15.658)
Day 56 -8.37  (15.658)
Day 84 -6.55  (15.658)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4489
Comments Mixed-effects model for repeated measures analysis with percent change in TG as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 84
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5964
Comments Mixed-effects model for repeated measures analysis with percent change in TG as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6785
Comments Mixed-effects model for repeated measures analysis with percent change in TG as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
10.Secondary Outcome
Title Change From Baseline in Fasting Triglycerides (TG)
Hide Description [Not Specified]
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: mg/dL
Day 28 -27.15  (21.615)
Day 56 -21.77  (21.615)
Day 84 -22.27  (21.615)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300mg: Day 28
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2177
Comments Mixed-effects model for repeated measures analysis with change in TG as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600mg: Day 56
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3209
Comments Mixed-effects model for repeated measures analysis with change in TG as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900mg: Day 84
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3101
Comments Mixed-effects model for repeated measures analysis with change in TG as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
11.Secondary Outcome
Title Percent Change From Baseline in Fasting HDL-C
Hide Description [Not Specified]
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: Percent Change
Day 28 -11.54  (3.906)
Day 56 -12.95  (3.906)
Day 84 -12.54  (3.906)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0057
Comments Mixed-effects model for repeated measures analysis with percent change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0022
Comments Mixed-effects model for repeated measures analysis with percent change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0029
Comments Mixed-effects model for repeated measures analysis with percent change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
12.Secondary Outcome
Title Change From Baseline in Fasting HDL-C
Hide Description [Not Specified]
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: mg/dL
Day 28 -4.80  (1.306)
Day 56 -5.05  (1.306)
Day 84 -5.55  (1.306)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300mg: Day 28
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0008
Comments Mixed-effects model for repeated measures analysis with change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600mg: Day 56
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0005
Comments Mixed-effects model for repeated measures analysis with change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900mg: Day 84
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0002
Comments Mixed-effects model for repeated measures analysis with change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
13.Secondary Outcome
Title Percent Change From Baseline in Fasting VLDL-C
Hide Description [Not Specified]
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: Percent Change
Day 28 -12.84  (15.407)
Day 56 -7.79  (15.407)
Day 84 -6.55  (15.407)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4103
Comments Mixed-effects model for repeated measures analysis with percent change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6164
Comments Mixed-effects model for repeated measures analysis with percent change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6732
Comments Mixed-effects model for repeated measures analysis with percent change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
14.Secondary Outcome
Title Change From Baseline in Fasting VLDL-C
Hide Description [Not Specified]
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: mg/dL
Day 28 -5.57  (4.261)
Day 56 -4.19  (4.261)
Day 84 -4.44  (4.261)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300mg: Day 28
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2003
Comments Mixed-effects model for repeated measures analysis with change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600mg: Day 56
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3323
Comments Mixed-effects model for repeated measures analysis with change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900mg: Day 84
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3047
Comments Mixed-effects model for repeated measures analysis with change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
15.Secondary Outcome
Title Percent Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status
Hide Description Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: Percent Change
Day 28: Negative LDLr Number Analyzed 3 participants
-9.78  (10.196)
Day 56: Negative LDLr Number Analyzed 3 participants
-14.27  (10.196)
Day 84: Negative LDLr Number Analyzed 3 participants
-11.36  (10.196)
Day 28: Defective LDLr Number Analyzed 5 participants
-33.97  (6.164)
Day 56: Defective LDLr Number Analyzed 5 participants
-38.33  (6.164)
Day 84: Defective LDLr Number Analyzed 5 participants
-39.22  (6.164)
Day 28: Met EAS Clinical Diagnosis of HoFH Number Analyzed 6 participants
-20.11  (9.502)
Day 56: Met EAS Clinical Diagnosis of HoFH Number Analyzed 6 participants
-24.57  (9.502)
Day 84: Met EAS Clinical Diagnosis of HoFH Number Analyzed 6 participants
-22.92  (9.502)
Day 28: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-44.40  (4.244)
Day 56: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-48.65  (4.244)
Day 84: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-51.48  (4.244)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3601
Comments Mixed-effects model for repeated measures analysis with percent change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1920
Comments Mixed-effects model for repeated measures analysis with percent change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2912
Comments Mixed-effects model for repeated measures analysis with percent change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments Mixed-effects model for repeated measures analysis with percent change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments Mixed-effects model for repeated measures analysis with percent change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments Mixed-effects model for repeated measures analysis with percent change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0444
Comments Mixed-effects model for repeated measures analysis with percent change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0159
Comments Mixed-effects model for repeated measures analysis with percent change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0235
Comments Mixed-effects model for repeated measures analysis with percent change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0005
Comments Mixed-effects model for repeated measures analysis with percent change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0003
Comments Mixed-effects model for repeated measures analysis with percent change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0003
Comments Mixed-effects model for repeated measures analysis with percent change in LDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
16.Secondary Outcome
Title Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status
Hide Description Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: mg/dL
Day 28: Negative LDLr Number Analyzed 3 participants
-64.16  (67.549)
Day 56: Negative LDLr Number Analyzed 3 participants
-92.83  (67.549)
Day 84: Negative LDLr Number Analyzed 3 participants
-76.16  (67.549)
Day 28: Defective LDLr Number Analyzed 5 participants
-74.43  (40.665)
Day 56: Defective LDLr Number Analyzed 5 participants
-85.43  (40.655)
Day 84: Defective LDLr Number Analyzed 5 participants
-87.63  (40.655)
Day 28: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-106.54  (312.492)
Day 56: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-125.38  (312.492)
Day 84: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-116.21  (312.492)
Day 28: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-119.38  (15.061)
Day 56: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-133.38  (15.061)
Day 84: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-141.38  (15.061)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3646
Comments Mixed-effects model for repeated measures analysis: Change in LDL-C as dependent variable, visit as fixed effect, patient as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1994
Comments Mixed-effects model for repeated measures analysis: Change in LDL-C as dependent variable, visit as fixed effect, patient as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2858
Comments Mixed-effects model for repeated measures analysis: Change in LDL-C as dependent variable, visit as fixed effect, patient as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0829
Comments Mixed-effects model for repeated measures analysis: Change in LDL-C as dependent variable, visit as fixed effect, patient as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0492
Comments Mixed-effects model for repeated measures analysis: Change in LDL-C as dependent variable, visit as fixed effect, patient as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0442
Comments Mixed-effects model for repeated measures analysis: Change in LDL-C as dependent variable, visit as fixed effect, patient as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7360
Comments Mixed-effects model for repeated measures analysis: Change in LDL-C as dependent variable, visit as fixed effect, patient as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6917
Comments Mixed-effects model for repeated measures analysis: Change in LDL-C as dependent variable, visit as fixed effect, patient as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7131
Comments Mixed-effects model for repeated measures analysis: Change in LDL-C as dependent variable, visit as fixed effect, patient as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0014
Comments Mixed-effects model for repeated measures analysis: Change in LDL-C as dependent variable, visit as fixed effect, patient as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0009
Comments Mixed-effects model for repeated measures analysis: Change in LDL-C as dependent variable, visit as fixed effect, patient as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0007
Comments Mixed-effects model for repeated measures analysis: Change in LDL-C as dependent variable, visit as fixed effect, patient as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
17.Secondary Outcome
Title Percent Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status
Hide Description Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: Percent Change
Day 28: Negative LDLr Number Analyzed 3 participants
-9.47  (10.497)
Day 56: Negative LDLr Number Analyzed 3 participants
-14.21  (10.497)
Day 84: Negative LDLr Number Analyzed 3 participants
-11.75  (10.497)
Day 28: Defective LDLr Number Analyzed 5 participants
-31.14  (9.861)
Day 56: Defective LDLr Number Analyzed 5 participants
-33.83  (9.861)
Day 84: Defective LDLr Number Analyzed 5 participants
-34.12  (9.861)
Day 28: Met EAS Clinical Diagnosis of HoFH Number Analyzed 6 participants
-18.37  (8.887)
Day 56: Met EAS Clinical Diagnosis of HoFH Number Analyzed 6 participants
-21.14  (8.887)
Day 84: Met EAS Clinical Diagnosis of HoFH Number Analyzed 6 participants
-19.32  (8.887)
Day 28: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-41.29  (3.577)
Day 56: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-46.81  (3.577)
Day 84: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-49.32  (3.577)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3880
Comments Mixed-effects model for repeated measures analysis with percent change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2057
Comments Mixed-effects model for repeated measures analysis with percent change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2892
Comments Mixed-effects model for repeated measures analysis with percent change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0052
Comments Mixed-effects model for repeated measures analysis with percent change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0028
Comments Mixed-effects model for repeated measures analysis with percent change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0026
Comments Mixed-effects model for repeated measures analysis with percent change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0493
Comments Mixed-effects model for repeated measures analysis with percent change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0253
Comments Mixed-effects model for repeated measures analysis with percent change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0394
Comments Mixed-effects model for repeated measures analysis with percent change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0003
Comments Mixed-effects model for repeated measures analysis with percent change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0002
Comments Mixed-effects model for repeated measures analysis with percent change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0002
Comments Mixed-effects model for repeated measures analysis with percent change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
18.Secondary Outcome
Title Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status
Hide Description Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: mg/dL
Day 28: Negative LDLr Number Analyzed 3 participants
-66.01  (72.386)
Day 56: Negative LDLr Number Analyzed 3 participants
-95.34  (72.386)
Day 84: Negative LDLr Number Analyzed 3 participants
-80.34  (72.386)
Day 28: Defective LDLr Number Analyzed 5 participants
-82.10  (50.684)
Day 56: Defective LDLr Number Analyzed 5 participants
-90.50  (50.684)
Day 84: Defective LDLr Number Analyzed 5 participants
-92.10  (50.684)
Day 28: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-61.42  (33.020)
Day 56: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-77.08  (33.020)
Day 84: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-68.25  (33.020)
Day 28: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-134.18  (11.456)
Day 56: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-152.18  (11.456)
Day 84: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-160.18  (11.456)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3833
Comments Mixed-effects model for repeated measures analysis with change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2172
Comments Mixed-effects model for repeated measures analysis with change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2930
Comments Mixed-effects model for repeated measures analysis with change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1217
Comments Mixed-effects model for repeated measures analysis with change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0901
Comments Mixed-effects model for repeated measures analysis with change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0850
Comments Mixed-effects model for repeated measures analysis with change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0747
Comments Mixed-effects model for repeated measures analysis with change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0279
Comments Mixed-effects model for repeated measures analysis with change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0492
Comments Mixed-effects model for repeated measures analysis with change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0003
Comments Mixed-effects model for repeated measures analysis with change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0002
Comments Mixed-effects model for repeated measures analysis with change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0002
Comments Mixed-effects model for repeated measures analysis with change in Non-HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
19.Secondary Outcome
Title Percent Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status
Hide Description Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: Percent Change
Day 28: Met the Genetic Criteria of LDLr Number Analyzed 3 participants
-2.29  (17.315)
Day 56: Met the Genetic Criteria of LDLr Number Analyzed 3 participants
-4.81  (17.315)
Day 84: Met the Genetic Criteria of LDLr Number Analyzed 3 participants
-12.19  (17.315)
Day 28: Not Met the Genetic Criteria of LDLr Number Analyzed 5 participants
-18.82  (30.652)
Day 56: Not Met the Genetic Criteria of LDLr Number Analyzed 5 participants
-9.22  (30.652)
Day 84: Not Met the Genetic Criteria of LDLr Number Analyzed 5 participants
-2.82  (30.652)
Day 28: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-8.09  (14.502)
Day 56: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
2.06  (14.502)
Day 84: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
2.72  (14.502)
Day 28: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-15.53  (3.488)
Day 56: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-25.75  (3.488)
Day 84: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-22.80  (3.488)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8972
Comments Mixed-effects model for repeated measures analysis with percent change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7867
Comments Mixed-effects model for repeated measures analysis with percent change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4974
Comments Mixed-effects model for repeated measures analysis with percent change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5464
Comments Mixed-effects model for repeated measures analysis with percent change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7668
Comments Mixed-effects model for repeated measures analysis with percent change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.9276
Comments Mixed-effects model for repeated measures analysis with percent change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5821
Comments Mixed-effects model for repeated measures analysis with percent change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8881
Comments Mixed-effects model for repeated measures analysis with percent change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8525
Comments Mixed-effects model for repeated measures analysis with percent change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0112
Comments Mixed-effects model for repeated measures analysis with percent change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0018
Comments Mixed-effects model for repeated measures analysis with percent change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH).
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0028
Comments Mixed-effects model for repeated measures analysis with percent change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
20.Secondary Outcome
Title Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status
Hide Description Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: mg/dL
Day 28: Met the Genetic Criteria of LDLr Number Analyzed 3 participants
-0.50  (3.821)
Day 56: Met the Genetic Criteria of LDLr Number Analyzed 3 participants
-1.17  (3.821)
Day 84: Met the Genetic Criteria of LDLr Number Analyzed 3 participants
-2.83  (3.821)
Day 28: Not Met the Genetic Criteria of LDLr Number Analyzed 5 participants
-8.55  (6.869)
Day 56: Not Met the Genetic Criteria of LDLr Number Analyzed 5 participants
-5.95  (6.869)
Day 84: Not Met the Genetic Criteria of LDLr Number Analyzed 5 participants
-5.35  (6.869)
Day 28: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-2.18  (29.378)
Day 56: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
0.98  (29.378)
Day 84: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
0.65  (29.378)
Day 28: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-1.55  (1.816)
Day 56: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-5.55  (1.816)
Day 84: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-5.55  (1.816)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8985
Comments Mixed-effects model for repeated measures analysis with change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7664
Comments Mixed-effects model for repeated measures analysis with change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4755
Comments Mixed-effects model for repeated measures analysis with change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2286
Comments Mixed-effects model for repeated measures analysis with change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3975
Comments Mixed-effects model for repeated measures analysis with change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4460
Comments Mixed-effects model for repeated measures analysis with change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.9414
Comments Mixed-effects model for repeated measures analysis with change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.9735
Comments Mixed-effects model for repeated measures analysis with change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.9825
Comments Mixed-effects model for repeated measures analysis with change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4420
Comments Mixed-effects model for repeated measures analysis with change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0378
Comments Mixed-effects model for repeated measures analysis with change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0378
Comments Mixed-effects model for repeated measures analysis with change in VLDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
21.Secondary Outcome
Title Percent Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status
Hide Description Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: Percent Change
Day 28: Negative LDLr Number Analyzed 3 participants
-18.44  (14.443)
Day 56: Negative LDLr Number Analyzed 3 participants
-20.63  (14.443)
Day 84: Negative LDLr Number Analyzed 3 participants
-6.75  (14.443)
Day 28: Defective LDLr Number Analyzed 5 participants
-9.51  (3.507)
Day 56: Defective LDLr Number Analyzed 5 participants
-10.45  (3.507)
Day 84: Defective LDLr Number Analyzed 5 participants
-18.12  (3.507)
Day 28: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-11.92  (5.805)
Day 56: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-15.77  (5.805)
Day 84: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-15.69  (5.805)
Day 28: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-16.98  (3.417)
Day 56: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-11.07  (3.417)
Day 84: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-9.67  (3.417)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2305
Comments Mixed-effects model for repeated measures analysis with percent change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1836
Comments Mixed-effects model for repeated measures analysis with percent change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6501
Comments Mixed-effects model for repeated measures analysis with percent change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0139
Comments Mixed-effects model for repeated measures analysis with percent change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0077
Comments Mixed-effects model for repeated measures analysis with percent change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments Mixed-effects model for repeated measures analysis with percent change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0507
Comments Mixed-effects model for repeated measures analysis with percent change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0118
Comments Mixed-effects model for repeated measures analysis with percent change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0122
Comments Mixed-effects model for repeated measures analysis with percent change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0077
Comments Mixed-effects model for repeated measures analysis with percent change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0317
Comments Mixed-effects model for repeated measures analysis with percent change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH).
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0473
Comments Mixed-effects model for repeated measures analysis with percent change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
22.Secondary Outcome
Title Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status
Hide Description Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: mg/dL
Day 28: NegativeLDLr Number Analyzed 3 participants
-4.96  (3.738)
Day 56: NegativeLDLr Number Analyzed 3 participants
-5.29  (3.738)
Day 84: NegativeLDLr Number Analyzed 3 participants
-1.62  (3.738)
Day 28: Defective LDLr Number Analyzed 5 participants
-5.19  (1.508)
Day 56: Defective LDLr Number Analyzed 5 participants
-5.39  (1.508)
Day 84: Defective LDLr Number Analyzed 5 participants
-8.39  (1.508)
Day 28: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-4.16  (1.755)
Day 56: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-5.50  (1.755)
Day 84: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-6.50  (1.755)
Day 28: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-9.07  (1.867)
Day 56: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-6.07  (1.867)
Day 84: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-5.07  (1.867)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2143
Comments Mixed-effects model for repeated measures analysis with change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1874
Comments Mixed-effects model for repeated measures analysis with change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6731
Comments Mixed-effects model for repeated measures analysis with change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0027
Comments Mixed-effects model for repeated measures analysis with change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0020
Comments Mixed-effects model for repeated measures analysis with change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments Mixed-effects model for repeated measures analysis with change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0258
Comments Mixed-effects model for repeated measures analysis with change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0044
Comments Mixed-effects model for repeated measures analysis with change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0011
Comments Mixed-effects model for repeated measures analysis with change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0083
Comments Mixed-effects model for repeated measures analysis with change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0314
Comments Mixed-effects model for repeated measures analysis with change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0533
Comments Mixed-effects model for repeated measures analysis with change in HDL-C as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
23.Secondary Outcome
Title Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status
Hide Description Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Time Frame Baseline, days 28, 56 and 84
Hide Outcome Measure Data
Hide Analysis Population Description
FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Arm/Group Title Gemcabene
Hide Arm/Group Description:
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28 followed by 600 mg of Gemcabene, orally once daily from day 29 to 56 followed by 900 mg of Gemcabene, orally once daily from day 57 to 84.
Overall Number of Participants Analyzed 8
Least Squares Mean (Standard Error)
Unit of Measure: Percent Change
Day 28: Negative LDLr Number Analyzed 3 participants
-10.02  (10.577)
Day 56: Negative LDLr Number Analyzed 3 participants
-14.57  (10.577)
Day 84: Negative LDLr Number Analyzed 3 participants
-11.64  (10.577)
Day 28: Defective LDLr Number Analyzed 5 participants
-26.43  (9.116)
Day 56: Defective LDLr Number Analyzed 5 participants
-29.01  (9.116)
Day 84: Defective LDLr Number Analyzed 5 participants
-30.67  (9.116)
Day 28: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-17.30  (8.845)
Day 56: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-20.39  (8.845)
Day 84: Met EAS Clinical Diagnosis of HoFH Number Analyzed 8 participants
-19.69  (8.845)
Day 28: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-38.47  (2.496)
Day 56: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-42.48  (2.496)
Day 84: Not Met EAS Clinical Diagnosis of HoFH Number Analyzed 2 participants
-44.31  (2.496)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3657
Comments Mixed-effects model for repeated measures analysis with percent change in TC as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1983
Comments Mixed-effects model for repeated measures analysis with percent change in TC as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Negative LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2970
Comments Mixed-effects model for repeated measures analysis with percent change in TC as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 300 mg: Day 28 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0092
Comments Mixed-effects model for repeated measures analysis with percent change in TC as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 600 mg: Day 56 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0049
Comments Mixed-effects model for repeated measures analysis with percent change in TC as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Gemcabene
Comments Gemcabene 900 mg: Day 84 (Defective LDLr)
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0033
Comments Mixed-effects model for repeated measures analysis with percent change in TC as dependent variable, visit as fixed effect, participant as a random effect. Auto-regressive variance-covariance structure was used.
Method Mixed Models Analysis
Comments [Not Specified]