Hypercholesteremia
Conditions
Keywords
LDL-C, Lipid Regulator
Brief summary
The purpose of this study was to assess the efficacy, safety, and tolerability of multiple doses of Gemcabene in patients with HoFH on stable, lipid-lowering therapy.
Interventions
DRUGGemcabene
300 mg tablet orally once daily for four weeks followed by 600 mg tablet orally once daily for four weeks followed by 900 mg tablet orally once daily for four weeks.
Sponsors
NeuroBo Pharmaceuticals Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
17 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Provision of written and signed informed consent (by patient or legal guardian) prior to any study-specific procedure; * Male or female ≥17 years of age at time of consent; * Diagnosis of HoFH by genetic confirmation (including compound heterozygosity) or a clinical diagnosis based on either (1) a history of an untreated LDL-C concentration \>500 mg/dL (12.92 mmol/L) together with either appearance of xanthoma before 10 years of age, or evidence of heterozygous familial hypercholesterolemia in both parents or, if history is unavailable, (2) LDL-C \>300 mg/dL (7.76 mmol/L) on maximally tolerated lipid-lowering drug therapy; * Currently on a stable, low-fat, low-cholesterol diet in combination with a pre-existing, regulatory-approved, not excluded lipid-lowering therapy (i.e., statins, monoclonal antibodies to PCSK9, cholesterol absorption inhibitors, bile acid sequestrants, or nicotinic acid, or any combination thereof) at a stable dose for at least 4 weeks prior to the Screening Visit; * Fasting LDL-C value \>130 mg/dL (3.36 mmol/L) at the Screening Visit; * Physical examination, including vital signs, that is within normal limits or clinically acceptable to the Investigator; * Weight ≥50 kg; * Female patients must not be pregnant or lactating. Women of child-bearing potential must have a negative serum pregnancy test at the Screening Visit and negative urine dipstick on Day 1 prior to dosing in order to qualify for the study. Women who are surgically sterile or are clinically confirmed to be post-menopausal (i.e., documented amenorrhea for ≥1 year in the absence of other biological or physiological causes) are not considered to be of child-bearing potential; and * Women of child-bearing potential must agree to use acceptable methods of contraception throughout the duration of the study and for 30 days after the last dose of study drug. For this study, double-barrier contraception is required.
Exclusion criteria
* Other forms of primary hyperlipoproteinemia and secondary causes of hypercholesterolemia (e.g., nephrotic syndrome or hypothyroidism); * Abnormal liver function test at the Screening Visit (aspartate aminotransferase or alanine aminotransferase \>2 × the upper limit of normal \[ULN\]; total bilirubin \>1.5 × ULN; or alkaline phosphatase \>2 × ULN based on appropriate age and gender normal values). Patients with bilirubin \>1.5 × ULN and history of Gilbert's syndrome may be included; reflexive direct bilirubin testing will be used to confirm Gilbert's syndrome; * Moderate (Grade B) or severe (Grade C) chronic hepatic impairment according to the Child Pugh classification; * Active liver disease (e.g., cirrhosis, alcoholic liver disease, hepatitis B virus \[HBV\], hepatitis C virus \[HCV\], autoimmune hepatitis, liver failure, liver cancer), history of liver transplant, or known diagnosis of human immunodeficiency virus (HIV); * Triglycerides value \>400 mg/dL (4.52 mmol/L) at the Screening Visit; * Moderate to severe renal insufficiency defined as an estimated GFR \<30 mL/min/1.73m2 (calculated using The Chronic Kidney Disease Epidemiology Collaboration equation) at the Screening Visit; * Abnormal urinalysis (proteinuria greater than trace or any male or non-menstruating female with greater than trace hematuria), confirmed by reflexive urine protein:creatinine ratio testing; * Uncontrolled thyroid disease: hyperthyroidism or hypothyroidism as defined by thyroid stimulating hormone (TSH) below the lower limit of normal or \>1.5 × ULN, respectively, at the Screening Visit. If controlled, treatment should be stable for at least 3 months prior to the Screening Visit; * Type 1 diabetes mellitus or uncontrolled type 2 diabetes mellitus (hemoglobin A1c \[HbA1c\] value \>8%), or any diabetic patient taking insulin and/or thiazolidinediones; * New York Heart Association Class III or IV heart failure; * Myocardial infarction, severe or unstable angina pectoris, coronary angioplasty, coronary artery bypass graft, or other major cardiovascular events resulting in hospitalization within 3 months of the Screening Visit. Patients with adequately treated stable angina, per Investigator assessment, may be included; * Uncontrolled cardiac arrhythmia or prolonged QT on the Screening Visit or Day 1 prior to dosing ECG (QTcF \>450 msec for men and \>470 msec for women) or known family history of prolonged QT or unexplained sudden cardiac death; * Uncontrolled hypertension, defined as sitting systolic blood pressure \>180 mmHg or diastolic blood pressure \>110 mmHg, and confirmed by repeat measurement; * Currently receiving cancer treatments or, in the Investigator's opinion, at risk of relapse for recent cancer; * Use of fibrate lipid-lowering agent 6 weeks prior to the Screening Visit; * Hypersensitivity to or a history of significant adverse reactions to any fibrate lipid lowering agent; * Use of apheresis (LDL or plasma) 8 weeks prior to the Screening Visit; * Use of lomitapide 2 months prior to the Screening Visit; * Use of mipomersen 5 months prior to the Screening Visit; * Use of any excluded medications or supplements (e.g., potent cytochrome P450 \[CYP\] 3A4 inhibitors); * History of drug or alcohol abuse within the past year or inability to comply with protocol requirements, including subject restrictions; * Previously treated with gemcabene; * Participation in another clinical study of an investigational agent or device concurrently or within 1 month prior to the Screening Visit, or use of an investigational agent within 1 month or 5 half-lives (if known), whichever is longer, prior to the Screening Visit; or * Any other finding which, in the opinion of the Investigator, would compromise the patient's safety or participation in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percent Change From Baseline in LDL-C at Day 28 | Baseline, day 28 |
| Percent Change From Baseline in LDL-C at Day 56 | Baseline, day 56 |
| Percent Change From Baseline in LDL-C at Day 84 | Baseline, day 84 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change From Baseline in Fasting HDL-C | Baseline, days 28, 56 and 84 | — |
| Change From Baseline in Fasting HDL-C | Baseline, days 28, 56 and 84 | — |
| Percent Change From Baseline in Fasting VLDL-C | Baseline, days 28, 56 and 84 | — |
| Change From Baseline in Fasting VLDL-C | Baseline, days 28, 56 and 84 | — |
| Change From Baseline in Fasting Apolipoprotein C-II | Baseline, days 28, 56 and 84 | — |
| Percent Change From Baseline in Fasting Apolipoprotein C-III | Baseline, days 28, 56 and 84 | — |
| Change From Baseline in Fasting Apolipoprotein C-III | Baseline, days 28, 56 and 84 | — |
| Percent Change From Baseline in Fasting Apolipoprotein E | Baseline, days 28, 56 and 84 | — |
| Change From Baseline in Fasting Apolipoprotein E | Baseline, days 28, 56 and 84 | — |
| Change From Baseline in Fasting LDL-C | Baseline, days 28, 56 and 84 | — |
| Percent Change From Baseline in Fasting Non-HDL-C | Baseline, days 28, 56 and 84 | — |
| Change From Baseline in Fasting Non-HDL-C | Baseline, days 28, 56 and 84 | — |
| Percent Change From Baseline in Fasting Total Cholesterol (TC) | Baseline, days 28, 56 and 84 | — |
| Percent Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Baseline, days 28, 56 and 84 | Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure. |
| Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Baseline, days 28, 56 and 84 | Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure. |
| Percent Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Baseline, days 28, 56 and 84 | Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure. |
| Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Baseline, days 28, 56 and 84 | Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure. |
| Percent Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Baseline, days 28, 56 and 84 | Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure. |
| Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Baseline, days 28, 56 and 84 | Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure. |
| Percent Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Baseline, days 28, 56 and 84 | Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure. |
| Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Baseline, days 28, 56 and 84 | Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure. |
| Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status | Baseline, days 28, 56 and 84 | Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure. |
| Change From Baseline in Fasting TC as Per Receptor Mutation Status | Baseline, days 28, 56 and 84 | Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure. |
| Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status | Baseline, days 28, 56 and 84 | Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure. |
| Change From Baseline in Fasting TG as Per Receptor Mutation Status | Baseline, days 28, 56 and 84 | Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure. |
| Number of Participants Achieving LDL-C Reduction of ≥15% | Days 28, 56 and 84 | — |
| Number of Participants Achieving LDL-C Reduction of ≥20% | Days 28, 56 and 84 | — |
| Number of Participants Achieving LDL-C Reduction of ≥25% | Days 28, 56 and 84 | — |
| Number of Participants Achieving LDL-C Reduction of ≥30% | Days 28, 56 and 84 | — |
| Number of Participants Achieving an LDL-C Value <100 mg/dL (2.59 mmol/L) | Days 28, 56 and 84 | — |
| Percent Change From Baseline in High-sensitivity C-reactive Protein (hsCRP) | Baseline, days 28, 56 and 84 | — |
| Change From Baseline in High-sensitivity C-reactive Protein (hsCRP) | Baseline, days 28, 56 and 84 | — |
| Percent Change From Baseline in Fibrinogen | Baseline, days 28, 56 and 84 | — |
| Change From Baseline in Fibrinogen | Baseline, days 28, 56 and 84 | — |
| Percent Change From Baseline in Fasting Lipoprotein(a) | Baseline, days 28, 56 and 84 | — |
| Change From Baseline in Fasting Lipoprotein(a) | Baseline, days 28, 56 and 84 | — |
| Percent Change From Baseline in Fasting Apolipoprotein B | Baseline, days 28, 56 and 84 | — |
| Change From Baseline in Fasting Apolipoprotein B | Baseline, days 28, 56 and 84 | — |
| Percent Change From Baseline in Fasting Apolipoprotein A-I | Baseline, days 28, 56 and 84 | — |
| Change From Baseline in Fasting Total Cholesterol (TC) | Baseline, days 28, 56 and 84 | — |
| Percent Change From Baseline in Fasting Apolipoprotein A-II | Baseline, days 28, 56 and 84 | — |
| Change From Baseline in Fasting Apolipoprotein A-II | Baseline, days 28, 56 and 84 | — |
| Percent Change From Baseline in Fasting Apolipoprotein C-II | Baseline, days 28, 56 and 84 | — |
| Change From Baseline in Fasting Apolipoprotein A-I | Baseline, days 28, 56 and 84 | — |
| Percent Change From Baseline in Fasting Triglycerides (TG) | Baseline, days 28, 56 and 84 | — |
| Change From Baseline in Fasting Triglycerides (TG) | Baseline, days 28, 56 and 84 | — |
Countries
Canada, Israel, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Gemcabene 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. | 8 |
| Total | 8 |
Baseline characteristics
| Characteristic | Gemcabene |
|---|---|
| Age, Continuous | 52.8 years STANDARD_DEVIATION 14.48 |
| Apolipoprotein A-I (ApoA-I) | 127.9 mg/dL STANDARD_DEVIATION 46.98 |
| Apolipoprotein A-II (ApoA-II) | 30.3 mg/dL STANDARD_DEVIATION 9.75 |
| Apolipoprotein B (ApoB) | 221.3 mg/dL STANDARD_DEVIATION 97.25 |
| Apolipoprotein C-II (ApoC-II) | 4.1 mg/dL STANDARD_DEVIATION 1.21 |
| Apolipoprotein C-III (ApoC-III) | 10.6 mg/dL STANDARD_DEVIATION 4.14 |
| Apolipoprotein E (ApoE) | 6.68 mg/dL STANDARD_DEVIATION 1.738 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 7 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Fibrinogen | 436.9 mg/dL STANDARD_DEVIATION 89.23 |
| HDL-C by Receptor Mutation Status Defective LDLr | 55.80 mg/dL STANDARD_DEVIATION 15.502 |
| HDL-C by Receptor Mutation Status Met EAS Clinical Diagnosis of HoFH | 39.83 mg/dL STANDARD_DEVIATION 17.394 |
| HDL-C by Receptor Mutation Status Negative LDLr | 28.17 mg/dL STANDARD_DEVIATION 4.726 |
| HDL-C by Receptor Mutation Status Not Met EAS Clinical Diagnosis of HoFH | 62.25 mg/dL STANDARD_DEVIATION 13.081 |
| High-density lipoprotein cholesterol (HDL-C) | 45.44 mg/dL STANDARD_DEVIATION 18.661 |
| High-sensitivity C-reactive protein (hsCRP) | 5.95 mg/dL STANDARD_DEVIATION 5.627 |
| LDL-C by Receptor Mutation Status Defective LDLr | 231.20 mg/dL STANDARD_DEVIATION 65.191 |
| LDL-C by Receptor Mutation Status Met EAS Clinical Diagnosis of HoFH | 374.33 mg/dL STANDARD_DEVIATION 208.717 |
| LDL-C by Receptor Mutation Status Negative LDLr | 551.33 mg/dL STANDARD_DEVIATION 105.652 |
| LDL-C by Receptor Mutation Status Not Met EAS Clinical Diagnosis of HoFH | 282.00 mg/dL STANDARD_DEVIATION 29.698 |
| Lipoprotein(a) | 113.5 mg/dL STANDARD_DEVIATION 92.97 |
| Low-density lipoprotein cholesterol (LDL-C) | 351.25 Milligram per deciliter (mg/dL) STANDARD_DEVIATION 181.849 |
| Non-HDL-C by Receptor Mutation Status Defective LDLr | 264.20 mg/dL STANDARD_DEVIATION 69.127 |
| Non-HDL-C by Receptor Mutation Status Met EAS Clinical Diagnosis of HoFH | 399.92 mg/dL STANDARD_DEVIATION 205.1 |
| Non-HDL-C by Receptor Mutation Status Negative LDLr | 572.83 mg/dL STANDARD_DEVIATION 105.419 |
| Non-HDL-C by Receptor Mutation Status Not Met EAS Clinical Diagnosis of HoFH | 320.00 mg/dL STANDARD_DEVIATION 5.657 |
| Non-high-density lipoprotein cholesterol (non-HDL-C) | 379.94 mg/dL STANDARD_DEVIATION 177.258 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 8 Participants |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 5 Participants |
| TC by Receptor Mutation Status Defective LDLr | 320.00 mg/dL STANDARD_DEVIATION 82.189 |
| TC by Receptor Mutation Status Met EAS Clinical Diagnosis of HoFH | 439.75 mg/dL STANDARD_DEVIATION 195.112 |
| TC by Receptor Mutation Status Negative LDLr | 601.00 mg/dL STANDARD_DEVIATION 100.714 |
| TC by Receptor Mutation Status Not Met EAS Clinical Diagnosis of HoFH | 382.25 mg/dL STANDARD_DEVIATION 7.425 |
| TG by Receptor Mutation Status Defective LDLr | 165.10 mg/dL STANDARD_DEVIATION 72.694 |
| TG by Receptor Mutation Status Met EAS Clinical Diagnosis of HoFH | 128.17 mg/dL STANDARD_DEVIATION 38.577 |
| TG by Receptor Mutation Status Negative LDLr | 107.67 mg/dL STANDARD_DEVIATION 9.278 |
| TG by Receptor Mutation Status Not Met EAS Clinical Diagnosis of HoFH | 189.75 mg/dL STANDARD_DEVIATION 119.855 |
| Total Cholesterol (TC) | 425.38 mg/dL STANDARD_DEVIATION 167.058 |
| Triglycerides (TG) | 143.56 mg/dL STANDARD_DEVIATION 62.672 |
| Very low-density lipoprotein cholesterol (VLDL-C) | 28.69 mg/dL STANDARD_DEVIATION 12.53 |
| VLDL-C by Receptor Mutation Status Defective LDLr | 33.00 mg/dL STANDARD_DEVIATION 14.53 |
| VLDL-C by Receptor Mutation Status Met EAS Clinical Diagnosis of HoFH | 25.58 mg/dL STANDARD_DEVIATION 7.612 |
| VLDL-C by Receptor Mutation Status Negative LDLr | 21.50 mg/dL STANDARD_DEVIATION 1.803 |
| VLDL-C by Receptor Mutation Status Not Met EAS Clinical Diagnosis of HoFH | 38.00 mg/dL STANDARD_DEVIATION 24.042 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 8 | 0 / 8 | 0 / 8 |
| other Total, other adverse events | 7 / 8 | 4 / 8 | 6 / 8 |
| serious Total, serious adverse events | 0 / 8 | 0 / 8 | 0 / 8 |
Outcome results
Primary
Percent Change From Baseline in LDL-C at Day 28
Time frame: Baseline, day 28
Population: 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 | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Gemcabene | Percent Change From Baseline in LDL-C at Day 28 | -25.54 Percent Change | Standard Error 8.301 |
p-value: 0.0041Mixed Models Analysis
Primary
Percent Change From Baseline in LDL-C at Day 56
Time frame: Baseline, day 56
Population: FAS Population.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Gemcabene | Percent Change From Baseline in LDL-C at Day 56 | -29.94 Percent Change | Standard Error 8.301 |
p-value: 0.001Mixed Models Analysis
Primary
Percent Change From Baseline in LDL-C at Day 84
Time frame: Baseline, day 84
Population: FAS Population.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Gemcabene | Percent Change From Baseline in LDL-C at Day 84 | -29.41 Percent Change | Standard Error 8.301 |
p-value: 0.0012Mixed Models Analysis
Secondary
Change From Baseline in Fasting Apolipoprotein A-I
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting Apolipoprotein A-I | Day 28 | -7.26 mg/dL | Standard Error 4.967 |
| Gemcabene | Change From Baseline in Fasting Apolipoprotein A-I | Day 56 | -4.88 mg/dL | Standard Error 4.967 |
| Gemcabene | Change From Baseline in Fasting Apolipoprotein A-I | Day 84 | -11.88 mg/dL | Standard Error 4.967 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.166Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.3423Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.0313Mixed Models Analysis
Secondary
Change From Baseline in Fasting Apolipoprotein A-II
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting Apolipoprotein A-II | Day 28 | 0.38 mg/dL | Standard Error 1.423 |
| Gemcabene | Change From Baseline in Fasting Apolipoprotein A-II | Day 56 | 1.00 mg/dL | Standard Error 1.423 |
| Gemcabene | Change From Baseline in Fasting Apolipoprotein A-II | Day 84 | 0.25 mg/dL | Standard Error 1.423 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.7952Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.4931Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.8622Mixed Models Analysis
Secondary
Change From Baseline in Fasting Apolipoprotein B
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting Apolipoprotein B | Day 28 | -39.24 mg/dL | Standard Error 19.882 |
| Gemcabene | Change From Baseline in Fasting Apolipoprotein B | Day 56 | -57.87 mg/dL | Standard Error 19.882 |
| Gemcabene | Change From Baseline in Fasting Apolipoprotein B | Day 84 | -49.37 mg/dL | Standard Error 19.882 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.0685Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.0114Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.0263Mixed Models Analysis
Secondary
Change From Baseline in Fasting Apolipoprotein C-II
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population. Here, overall number of participants analyzed (N) signifies those participants who were evaluable for this outcome measure only.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting Apolipoprotein C-II | Day 28 | 0.71 mg/dL | Standard Error 0.615 |
| Gemcabene | Change From Baseline in Fasting Apolipoprotein C-II | Day 56 | 1.14 mg/dL | Standard Error 0.615 |
| Gemcabene | Change From Baseline in Fasting Apolipoprotein C-II | Day 84 | 1.71 mg/dL | Standard Error 0.615 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.2683Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.088Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.0165Mixed Models Analysis
Secondary
Change From Baseline in Fasting Apolipoprotein C-III
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting Apolipoprotein C-III | Day 28 | -1.37 mg/dL | Standard Error 1.26 |
| Gemcabene | Change From Baseline in Fasting Apolipoprotein C-III | Day 56 | -1.62 mg/dL | Standard Error 1.26 |
| Gemcabene | Change From Baseline in Fasting Apolipoprotein C-III | Day 84 | -1.37 mg/dL | Standard Error 1.26 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.2937Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.2182Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.2937Mixed Models Analysis
Secondary
Change From Baseline in Fasting Apolipoprotein E
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting Apolipoprotein E | Day 28 | -1.40 mg/dL | Standard Error 0.641 |
| Gemcabene | Change From Baseline in Fasting Apolipoprotein E | Day 56 | -1.65 mg/dL | Standard Error 0.641 |
| Gemcabene | Change From Baseline in Fasting Apolipoprotein E | Day 84 | -1.45 mg/dL | Standard Error 0.641 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.0465Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.0221Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.0402Mixed Models Analysis
Secondary
Change From Baseline in Fasting HDL-C
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting HDL-C | Day 28 | -4.80 mg/dL | Standard Error 1.306 |
| Gemcabene | Change From Baseline in Fasting HDL-C | Day 56 | -5.05 mg/dL | Standard Error 1.306 |
| Gemcabene | Change From Baseline in Fasting HDL-C | Day 84 | -5.55 mg/dL | Standard Error 1.306 |
Comparison: Gemcabene 300mg: Day 28p-value: 0.0008Mixed Models Analysis
Comparison: Gemcabene 600mg: Day 56p-value: 0.0005Mixed Models Analysis
Comparison: Gemcabene 900mg: Day 84p-value: 0.0002Mixed Models Analysis
Secondary
Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status
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
Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 28: NegativeLDLr | -4.96 mg/dL | Standard Error 3.738 |
| Gemcabene | Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 56: NegativeLDLr | -5.29 mg/dL | Standard Error 3.738 |
| Gemcabene | Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 84: NegativeLDLr | -1.62 mg/dL | Standard Error 3.738 |
| Gemcabene | Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 28: Defective LDLr | -5.19 mg/dL | Standard Error 1.508 |
| Gemcabene | Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 56: Defective LDLr | -5.39 mg/dL | Standard Error 1.508 |
| Gemcabene | Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 84: Defective LDLr | -8.39 mg/dL | Standard Error 1.508 |
| Gemcabene | Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 28: Met EAS Clinical Diagnosis of HoFH | -4.16 mg/dL | Standard Error 1.755 |
| Gemcabene | Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 56: Met EAS Clinical Diagnosis of HoFH | -5.50 mg/dL | Standard Error 1.755 |
| Gemcabene | Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 84: Met EAS Clinical Diagnosis of HoFH | -6.50 mg/dL | Standard Error 1.755 |
| Gemcabene | Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 28: Not Met EAS Clinical Diagnosis of HoFH | -9.07 mg/dL | Standard Error 1.867 |
| Gemcabene | Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 56: Not Met EAS Clinical Diagnosis of HoFH | -6.07 mg/dL | Standard Error 1.867 |
| Gemcabene | Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 84: Not Met EAS Clinical Diagnosis of HoFH | -5.07 mg/dL | Standard Error 1.867 |
Comparison: Gemcabene 300 mg: Day 28 (Negative LDLr)p-value: 0.2143Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Negative LDLr)p-value: 0.1874Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Negative LDLr)p-value: 0.6731Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Defective LDLr)p-value: 0.0027Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Defective LDLr)p-value: 0.002Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Defective LDLr)p-value: <0.0001Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0258Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0044Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0011Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0083Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0314Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0533Mixed Models Analysis
Secondary
Change From Baseline in Fasting LDL-C
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting LDL-C | Day 28 | -82.15 mg/dL | Standard Error 41.52 |
| Gemcabene | Change From Baseline in Fasting LDL-C | Day 56 | -99.77 mg/dL | Standard Error 41.52 |
| Gemcabene | Change From Baseline in Fasting LDL-C | Day 84 | -94.90 mg/dL | Standard Error 41.52 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.056Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.0219Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.0286Mixed Models Analysis
Secondary
Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status
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
Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 28: Negative LDLr | -64.16 mg/dL | Standard Error 67.549 |
| Gemcabene | Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 56: Negative LDLr | -92.83 mg/dL | Standard Error 67.549 |
| Gemcabene | Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 84: Negative LDLr | -76.16 mg/dL | Standard Error 67.549 |
| Gemcabene | Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 28: Defective LDLr | -74.43 mg/dL | Standard Error 40.665 |
| Gemcabene | Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 56: Defective LDLr | -85.43 mg/dL | Standard Error 40.655 |
| Gemcabene | Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 84: Defective LDLr | -87.63 mg/dL | Standard Error 40.655 |
| Gemcabene | Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 28: Met EAS Clinical Diagnosis of HoFH | -106.54 mg/dL | Standard Error 312.492 |
| Gemcabene | Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 56: Met EAS Clinical Diagnosis of HoFH | -125.38 mg/dL | Standard Error 312.492 |
| Gemcabene | Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 84: Met EAS Clinical Diagnosis of HoFH | -116.21 mg/dL | Standard Error 312.492 |
| Gemcabene | Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 28: Not Met EAS Clinical Diagnosis of HoFH | -119.38 mg/dL | Standard Error 15.061 |
| Gemcabene | Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 56: Not Met EAS Clinical Diagnosis of HoFH | -133.38 mg/dL | Standard Error 15.061 |
| Gemcabene | Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 84: Not Met EAS Clinical Diagnosis of HoFH | -141.38 mg/dL | Standard Error 15.061 |
Comparison: Gemcabene 300 mg: Day 28 (Negative LDLr)p-value: 0.3646Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Negative LDLr)p-value: 0.1994Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Negative LDLr)p-value: 0.2858Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Defective LDLr)p-value: 0.0829Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Defective LDLr)p-value: 0.0492Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Defective LDLr)p-value: 0.0442Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.736Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.6917Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.7131Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0014Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0009Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0007Mixed Models Analysis
Secondary
Change From Baseline in Fasting Lipoprotein(a)
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting Lipoprotein(a) | Day 28 | 19.39 nmol/L | Standard Error 26.804 |
| Gemcabene | Change From Baseline in Fasting Lipoprotein(a) | Day 56 | 18.51 nmol/L | Standard Error 26.804 |
| Gemcabene | Change From Baseline in Fasting Lipoprotein(a) | Day 84 | 21.51 nmol/L | Standard Error 26.804 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.4814Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.5011Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.4356Mixed Models Analysis
Secondary
Change From Baseline in Fasting Non-HDL-C
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting Non-HDL-C | Day 28 | -86.64 mg/dL | Standard Error 44.38 |
| Gemcabene | Change From Baseline in Fasting Non-HDL-C | Day 56 | -102.89 mg/dL | Standard Error 44.38 |
| Gemcabene | Change From Baseline in Fasting Non-HDL-C | Day 84 | -98.26 mg/dL | Standard Error 44.38 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.0592Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.0266Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.0336Mixed Models Analysis
Secondary
Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status
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
Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 28: Negative LDLr | -66.01 mg/dL | Standard Error 72.386 |
| Gemcabene | Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 56: Negative LDLr | -95.34 mg/dL | Standard Error 72.386 |
| Gemcabene | Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 84: Negative LDLr | -80.34 mg/dL | Standard Error 72.386 |
| Gemcabene | Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 28: Defective LDLr | -82.10 mg/dL | Standard Error 50.684 |
| Gemcabene | Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 56: Defective LDLr | -90.50 mg/dL | Standard Error 50.684 |
| Gemcabene | Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 84: Defective LDLr | -92.10 mg/dL | Standard Error 50.684 |
| Gemcabene | Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 28: Met EAS Clinical Diagnosis of HoFH | -61.42 mg/dL | Standard Error 33.02 |
| Gemcabene | Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 56: Met EAS Clinical Diagnosis of HoFH | -77.08 mg/dL | Standard Error 33.02 |
| Gemcabene | Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 84: Met EAS Clinical Diagnosis of HoFH | -68.25 mg/dL | Standard Error 33.02 |
| Gemcabene | Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 28: Not Met EAS Clinical Diagnosis of HoFH | -134.18 mg/dL | Standard Error 11.456 |
| Gemcabene | Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 56: Not Met EAS Clinical Diagnosis of HoFH | -152.18 mg/dL | Standard Error 11.456 |
| Gemcabene | Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 84: Not Met EAS Clinical Diagnosis of HoFH | -160.18 mg/dL | Standard Error 11.456 |
Comparison: Gemcabene 300 mg: Day 28 (Negative LDLr)p-value: 0.3833Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Negative LDLr)p-value: 0.2172Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Negative LDLr)p-value: 0.293Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Defective LDLr)p-value: 0.1217Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Defective LDLr)p-value: 0.0901Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Defective LDLr)p-value: 0.085Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0747Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0279Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0492Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0003Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0002Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0002Mixed Models Analysis
Secondary
Change From Baseline in Fasting TC as Per Receptor Mutation Status
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
Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 28: Negative LDLr | -70.92 mg/dL | Standard Error 75.095 |
| Gemcabene | Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 56: Negative LDLr | -100.58 mg/dL | Standard Error 75.095 |
| Gemcabene | Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 84: Negative LDLr | -81.92 mg/dL | Standard Error 75.095 |
| Gemcabene | Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 28: Defective LDLr | -84.61 mg/dL | Standard Error 57.378 |
| Gemcabene | Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 56: Defective LDLr | -93.21 mg/dL | Standard Error 57.378 |
| Gemcabene | Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 84: Defective LDLr | -97.81 mg/dL | Standard Error 57.378 |
| Gemcabene | Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 28: Met EAS Clinical Diagnosis of HoFH | -65.58 mg/dL | Standard Error 33.715 |
| Gemcabene | Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 56: Met EAS Clinical Diagnosis of HoFH | -82.58 mg/dL | Standard Error 33.715 |
| Gemcabene | Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 84: Met EAS Clinical Diagnosis of HoFH | -74.75 mg/dL | Standard Error 33.715 |
| Gemcabene | Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 28: Not Met EAS Clinical Diagnosis of HoFH | -143.67 mg/dL | Standard Error 9.885 |
| Gemcabene | Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 56: Not Met EAS Clinical Diagnosis of HoFH | -158.67 mg/dL | Standard Error 9.885 |
| Gemcabene | Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 84: Not Met EAS Clinical Diagnosis of HoFH | -165.67 mg/dL | Standard Error 9.885 |
Comparison: Gemcabene 300 mg: Day 28 (Negative LDLrp-value: 0.3672Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Negative LDLr)p-value: 0.2101Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Negative LDLr)p-value: 0.3009Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Defective LDLr)p-value: 0.1567Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Defective LDLr)p-value: 0.1207Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Defective LDLr)p-value: 0.1045Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0631Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0217Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0359Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0001Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)p-value: <0.0001Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH)p-value: <0.0001Mixed Models Analysis
Secondary
Change From Baseline in Fasting TG as Per Receptor Mutation Status
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
Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 28: Negative LDLr | -2.00 mg/dL | Standard Error 19.308 |
| Gemcabene | Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 56: Negative LDLr | -6.33 mg/dL | Standard Error 19.308 |
| Gemcabene | Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 84: Negative LDLr | -13.33 mg/dL | Standard Error 19.308 |
| Gemcabene | Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 28: Defective LDLr | -41.94 mg/dL | Standard Error 35.093 |
| Gemcabene | Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 56: Defective LDLr | -30.74 mg/dL | Standard Error 35.093 |
| Gemcabene | Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 84: Defective LDLr | -27.34 mg/dL | Standard Error 35.093 |
| Gemcabene | Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 28: Met EAS Clinical Diagnosis of HoFH | -10.33 mg/dL | Standard Error 102.229 |
| Gemcabene | Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 56: Met EAS Clinical Diagnosis of HoFH | 3.83 mg/dL | Standard Error 102.229 |
| Gemcabene | Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 84: Met EAS Clinical Diagnosis of HoFH | 3.17 mg/dL | Standard Error 102.229 |
| Gemcabene | Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 28: Not Met EAS Clinical Diagnosis of HoFH | -7.82 mg/dL | Standard Error 8.738 |
| Gemcabene | Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 56: Not Met EAS Clinical Diagnosis of HoFH | -28.82 mg/dL | Standard Error 8.738 |
| Gemcabene | Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 84: Not Met EAS Clinical Diagnosis of HoFH | -28.82 mg/dL | Standard Error 8.738 |
Comparison: Gemcabene 300 mg: Day 28 (Negative LDLr)p-value: 0.9195Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Negative LDLr)p-value: 0.7497Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Negative LDLr)p-value: 0.5056Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Defective LDLr)p-value: 0.2468Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Defective LDLr)p-value: 0.392Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Defective LDLr)p-value: 0.4456Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.9203Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.9704Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.9755Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.4213Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.03Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.03Mixed Models Analysis
Secondary
Change From Baseline in Fasting Total Cholesterol (TC)
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting Total Cholesterol (TC) | Day 28 | -91.96 mg/dL | Standard Error 46.421 |
| Gemcabene | Change From Baseline in Fasting Total Cholesterol (TC) | Day 56 | -108.46 mg/dL | Standard Error 46.421 |
| Gemcabene | Change From Baseline in Fasting Total Cholesterol (TC) | Day 84 | -104.34 mg/dL | Standard Error 46.421 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.0057Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.0255Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 86p-value: 0.0312Mixed Models Analysis
Secondary
Change From Baseline in Fasting Triglycerides (TG)
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting Triglycerides (TG) | Day 28 | -27.15 mg/dL | Standard Error 21.615 |
| Gemcabene | Change From Baseline in Fasting Triglycerides (TG) | Day 56 | -21.77 mg/dL | Standard Error 21.615 |
| Gemcabene | Change From Baseline in Fasting Triglycerides (TG) | Day 84 | -22.27 mg/dL | Standard Error 21.615 |
Comparison: Gemcabene 300mg: Day 28p-value: 0.2177Mixed Models Analysis
Comparison: Gemcabene 600mg: Day 56p-value: 0.3209Mixed Models Analysis
Comparison: Gemcabene 900mg: Day 84p-value: 0.3101Mixed Models Analysis
Secondary
Change From Baseline in Fasting VLDL-C
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting VLDL-C | Day 28 | -5.57 mg/dL | Standard Error 4.261 |
| Gemcabene | Change From Baseline in Fasting VLDL-C | Day 56 | -4.19 mg/dL | Standard Error 4.261 |
| Gemcabene | Change From Baseline in Fasting VLDL-C | Day 84 | -4.44 mg/dL | Standard Error 4.261 |
Comparison: Gemcabene 300mg: Day 28p-value: 0.2003Mixed Models Analysis
Comparison: Gemcabene 600mg: Day 56p-value: 0.3323Mixed Models Analysis
Comparison: Gemcabene 900mg: Day 84p-value: 0.3047Mixed Models Analysis
Secondary
Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status
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
Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 28: Met the Genetic Criteria of LDLr | -0.50 mg/dL | Standard Error 3.821 |
| Gemcabene | Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 56: Met the Genetic Criteria of LDLr | -1.17 mg/dL | Standard Error 3.821 |
| Gemcabene | Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 84: Met the Genetic Criteria of LDLr | -2.83 mg/dL | Standard Error 3.821 |
| Gemcabene | Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 28: Not Met the Genetic Criteria of LDLr | -8.55 mg/dL | Standard Error 6.869 |
| Gemcabene | Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 56: Not Met the Genetic Criteria of LDLr | -5.95 mg/dL | Standard Error 6.869 |
| Gemcabene | Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 84: Not Met the Genetic Criteria of LDLr | -5.35 mg/dL | Standard Error 6.869 |
| Gemcabene | Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 28: Met EAS Clinical Diagnosis of HoFH | -2.18 mg/dL | Standard Error 29.378 |
| Gemcabene | Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 56: Met EAS Clinical Diagnosis of HoFH | 0.98 mg/dL | Standard Error 29.378 |
| Gemcabene | Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 84: Met EAS Clinical Diagnosis of HoFH | 0.65 mg/dL | Standard Error 29.378 |
| Gemcabene | Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 28: Not Met EAS Clinical Diagnosis of HoFH | -1.55 mg/dL | Standard Error 1.816 |
| Gemcabene | Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 56: Not Met EAS Clinical Diagnosis of HoFH | -5.55 mg/dL | Standard Error 1.816 |
| Gemcabene | Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 84: Not Met EAS Clinical Diagnosis of HoFH | -5.55 mg/dL | Standard Error 1.816 |
Comparison: Gemcabene 900 mg: Day 84 (Defective LDLr)p-value: 0.446Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Negative LDLr)p-value: 0.8985Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Negative LDLr)p-value: 0.7664Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Negative LDLr)p-value: 0.4755Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Defective LDLr)p-value: 0.2286Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Defective LDLr)p-value: 0.3975Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.9414Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.9735Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.9825Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.442Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0378Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0378Mixed Models Analysis
Secondary
Change From Baseline in Fibrinogen
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in Fibrinogen | Day 28 | 34.17 mg/dL | Standard Error 19.141 |
| Gemcabene | Change From Baseline in Fibrinogen | Day 56 | -34.58 mg/dL | Standard Error 19.141 |
| Gemcabene | Change From Baseline in Fibrinogen | Day 84 | -28.46 mg/dL | Standard Error 19.141 |
Comparison: Gemcabene 900 mg: Day 84p-value: 0.1592Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28p-value: 0.0959Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.0923Mixed Models Analysis
Secondary
Change From Baseline in High-sensitivity C-reactive Protein (hsCRP)
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Change From Baseline in High-sensitivity C-reactive Protein (hsCRP) | Day 28 | 5.30 mg/L | Standard Error 3.105 |
| Gemcabene | Change From Baseline in High-sensitivity C-reactive Protein (hsCRP) | Day 56 | -1.91 mg/L | Standard Error 3.105 |
| Gemcabene | Change From Baseline in High-sensitivity C-reactive Protein (hsCRP) | Day 84 | 0.46 mg/L | Standard Error 3.105 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.1099Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.5478Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.8837Mixed Models Analysis
Secondary
Number of Participants Achieving an LDL-C Value <100 mg/dL (2.59 mmol/L)
Time frame: Days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Gemcabene | Number of Participants Achieving an LDL-C Value <100 mg/dL (2.59 mmol/L) | Day 28 | 1 Participants |
| Gemcabene | Number of Participants Achieving an LDL-C Value <100 mg/dL (2.59 mmol/L) | Day 56 | 1 Participants |
| Gemcabene | Number of Participants Achieving an LDL-C Value <100 mg/dL (2.59 mmol/L) | Day 84 | 1 Participants |
Secondary
Number of Participants Achieving LDL-C Reduction of ≥15%
Time frame: Days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Gemcabene | Number of Participants Achieving LDL-C Reduction of ≥15% | Day 28 | 6 Participants |
| Gemcabene | Number of Participants Achieving LDL-C Reduction of ≥15% | Day 56 | 6 Participants |
| Gemcabene | Number of Participants Achieving LDL-C Reduction of ≥15% | Day 84 | 7 Participants |
Secondary
Number of Participants Achieving LDL-C Reduction of ≥20%
Time frame: Days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Gemcabene | Number of Participants Achieving LDL-C Reduction of ≥20% | Day 28 | 5 Participants |
| Gemcabene | Number of Participants Achieving LDL-C Reduction of ≥20% | Day 56 | 6 Participants |
| Gemcabene | Number of Participants Achieving LDL-C Reduction of ≥20% | Day 84 | 6 Participants |
Secondary
Number of Participants Achieving LDL-C Reduction of ≥25%
Time frame: Days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Gemcabene | Number of Participants Achieving LDL-C Reduction of ≥25% | Day 28 | 5 Participants |
| Gemcabene | Number of Participants Achieving LDL-C Reduction of ≥25% | Day 56 | 5 Participants |
| Gemcabene | Number of Participants Achieving LDL-C Reduction of ≥25% | Day 84 | 5 Participants |
Secondary
Number of Participants Achieving LDL-C Reduction of ≥30%
Time frame: Days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Gemcabene | Number of Participants Achieving LDL-C Reduction of ≥30% | Day 28 | 4 Participants |
| Gemcabene | Number of Participants Achieving LDL-C Reduction of ≥30% | Day 56 | 5 Participants |
| Gemcabene | Number of Participants Achieving LDL-C Reduction of ≥30% | Day 84 | 4 Participants |
Secondary
Percent Change From Baseline in Fasting Apolipoprotein A-I
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein A-I | Day 28 | -6.27 Percent Change | Standard Error 4.082 |
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein A-I | Day 56 | -3.86 Percent Change | Standard Error 4.082 |
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein A-I | Day 84 | -8.06 Percent Change | Standard Error 4.082 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.1466Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.3598Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.0685Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting Apolipoprotein A-II
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein A-II | Day 28 | 2.00 Percent Change | Standard Error 5.473 |
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein A-II | Day 56 | 4.41 Percent Change | Standard Error 5.473 |
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein A-II | Day 84 | 1.97 Percent Change | Standard Error 5.473 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.7196Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.4343Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.7241Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting Apolipoprotein B
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein B | Day 28 | -17.95 Percent Change | Standard Error 9.034 |
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein B | Day 56 | -23.98 Percent Change | Standard Error 9.034 |
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein B | Day 84 | -21.58 Percent Change | Standard Error 9.034 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.0669Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.0189Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.0316Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting Apolipoprotein C-II
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population. Here, overall number of participants analyzed (N) signifies those participants who were evaluable for this outcome measure only.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein C-II | Day 28 | -1.69 Percent Change | Standard Error 74.431 |
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein C-II | Day 56 | 8.31 Percent Change | Standard Error 74.431 |
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein C-II | Day 84 | 21.64 Percent Change | Standard Error 74.431 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.9823Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.9129Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 84p-value: 0.7762Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting Apolipoprotein C-III
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein C-III | Day 28 | -8.86 Percent Change | Standard Error 11.616 |
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein C-III | Day 56 | -10.71 Percent Change | Standard Error 11.616 |
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein C-III | Day 84 | -7.47 Percent Change | Standard Error 11.616 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.4585Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.3721Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.5305Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting Apolipoprotein E
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein E | Day 28 | -19.57 Percent Change | Standard Error 8.699 |
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein E | Day 56 | -23.01 Percent Change | Standard Error 8.699 |
| Gemcabene | Percent Change From Baseline in Fasting Apolipoprotein E | Day 84 | -19.20 Percent Change | Standard Error 8.699 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.0411Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.0192Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.0445Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting HDL-C
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting HDL-C | Day 28 | -11.54 Percent Change | Standard Error 3.906 |
| Gemcabene | Percent Change From Baseline in Fasting HDL-C | Day 56 | -12.95 Percent Change | Standard Error 3.906 |
| Gemcabene | Percent Change From Baseline in Fasting HDL-C | Day 84 | -12.54 Percent Change | Standard Error 3.906 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.0057Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.0022Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.0029Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status
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
Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 28: Negative LDLr | -18.44 Percent Change | Standard Error 14.443 |
| Gemcabene | Percent Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 56: Negative LDLr | -20.63 Percent Change | Standard Error 14.443 |
| Gemcabene | Percent Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 84: Negative LDLr | -6.75 Percent Change | Standard Error 14.443 |
| Gemcabene | Percent Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 28: Defective LDLr | -9.51 Percent Change | Standard Error 3.507 |
| Gemcabene | Percent Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 56: Defective LDLr | -10.45 Percent Change | Standard Error 3.507 |
| Gemcabene | Percent Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 84: Defective LDLr | -18.12 Percent Change | Standard Error 3.507 |
| Gemcabene | Percent Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 28: Met EAS Clinical Diagnosis of HoFH | -11.92 Percent Change | Standard Error 5.805 |
| Gemcabene | Percent Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 56: Met EAS Clinical Diagnosis of HoFH | -15.77 Percent Change | Standard Error 5.805 |
| Gemcabene | Percent Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 84: Met EAS Clinical Diagnosis of HoFH | -15.69 Percent Change | Standard Error 5.805 |
| Gemcabene | Percent Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 28: Not Met EAS Clinical Diagnosis of HoFH | -16.98 Percent Change | Standard Error 3.417 |
| Gemcabene | Percent Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 56: Not Met EAS Clinical Diagnosis of HoFH | -11.07 Percent Change | Standard Error 3.417 |
| Gemcabene | Percent Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status | Day 84: Not Met EAS Clinical Diagnosis of HoFH | -9.67 Percent Change | Standard Error 3.417 |
Comparison: Gemcabene 300 mg: Day 28 (Negative LDLr)p-value: 0.2305Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Negative LDLr)p-value: 0.1836Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Negative LDLr)p-value: 0.6501Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Defective LDLr)p-value: 0.0139Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Defective LDLr)p-value: 0.0077Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Defective LDLr)p-value: <0.0001Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0507Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0118Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0122Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0077Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0317Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH).p-value: 0.0473Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status
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
Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 28: Negative LDLr | -9.78 Percent Change | Standard Error 10.196 |
| Gemcabene | Percent Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 56: Negative LDLr | -14.27 Percent Change | Standard Error 10.196 |
| Gemcabene | Percent Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 84: Negative LDLr | -11.36 Percent Change | Standard Error 10.196 |
| Gemcabene | Percent Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 28: Defective LDLr | -33.97 Percent Change | Standard Error 6.164 |
| Gemcabene | Percent Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 56: Defective LDLr | -38.33 Percent Change | Standard Error 6.164 |
| Gemcabene | Percent Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 84: Defective LDLr | -39.22 Percent Change | Standard Error 6.164 |
| Gemcabene | Percent Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 28: Met EAS Clinical Diagnosis of HoFH | -20.11 Percent Change | Standard Error 9.502 |
| Gemcabene | Percent Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 56: Met EAS Clinical Diagnosis of HoFH | -24.57 Percent Change | Standard Error 9.502 |
| Gemcabene | Percent Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 84: Met EAS Clinical Diagnosis of HoFH | -22.92 Percent Change | Standard Error 9.502 |
| Gemcabene | Percent Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 28: Not Met EAS Clinical Diagnosis of HoFH | -44.40 Percent Change | Standard Error 4.244 |
| Gemcabene | Percent Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 56: Not Met EAS Clinical Diagnosis of HoFH | -48.65 Percent Change | Standard Error 4.244 |
| Gemcabene | Percent Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status | Day 84: Not Met EAS Clinical Diagnosis of HoFH | -51.48 Percent Change | Standard Error 4.244 |
Comparison: Gemcabene 300 mg: Day 28 (Negative LDLr)p-value: 0.3601Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Negative LDLr)p-value: 0.192Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Negative LDLr)p-value: 0.2912Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Defective LDLr)p-value: <0.0001Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Defective LDLr)p-value: <0.0001Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Defective LDLr)p-value: <0.0001Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0444Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0159Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0235Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0005Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0003Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0003Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting Lipoprotein(a)
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting Lipoprotein(a) | Day 28 | 6.90 Percent Change | Standard Error 12.362 |
| Gemcabene | Percent Change From Baseline in Fasting Lipoprotein(a) | Day 56 | 5.84 Percent Change | Standard Error 12.362 |
| Gemcabene | Percent Change From Baseline in Fasting Lipoprotein(a) | Day 84 | 15.62 Percent Change | Standard Error 12.362 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.5856Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.644Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.2269Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting Non-HDL-C
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting Non-HDL-C | Day 28 | -23.87 Percent Change | Standard Error 8.105 |
| Gemcabene | Percent Change From Baseline in Fasting Non-HDL-C | Day 56 | -27.33 Percent Change | Standard Error 8.105 |
| Gemcabene | Percent Change From Baseline in Fasting Non-HDL-C | Day 84 | -26.59 Percent Change | Standard Error 8.105 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.0058Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.0019Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.0024Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status
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
Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 28: Negative LDLr | -9.47 Percent Change | Standard Error 10.497 |
| Gemcabene | Percent Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 56: Negative LDLr | -14.21 Percent Change | Standard Error 10.497 |
| Gemcabene | Percent Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 84: Negative LDLr | -11.75 Percent Change | Standard Error 10.497 |
| Gemcabene | Percent Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 28: Defective LDLr | -31.14 Percent Change | Standard Error 9.861 |
| Gemcabene | Percent Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 56: Defective LDLr | -33.83 Percent Change | Standard Error 9.861 |
| Gemcabene | Percent Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 84: Defective LDLr | -34.12 Percent Change | Standard Error 9.861 |
| Gemcabene | Percent Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 28: Met EAS Clinical Diagnosis of HoFH | -18.37 Percent Change | Standard Error 8.887 |
| Gemcabene | Percent Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 56: Met EAS Clinical Diagnosis of HoFH | -21.14 Percent Change | Standard Error 8.887 |
| Gemcabene | Percent Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 84: Met EAS Clinical Diagnosis of HoFH | -19.32 Percent Change | Standard Error 8.887 |
| Gemcabene | Percent Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 28: Not Met EAS Clinical Diagnosis of HoFH | -41.29 Percent Change | Standard Error 3.577 |
| Gemcabene | Percent Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 56: Not Met EAS Clinical Diagnosis of HoFH | -46.81 Percent Change | Standard Error 3.577 |
| Gemcabene | Percent Change From Baseline in Fasting Non-HDL-C as Per Receptor Mutation Status | Day 84: Not Met EAS Clinical Diagnosis of HoFH | -49.32 Percent Change | Standard Error 3.577 |
Comparison: Gemcabene 300 mg: Day 28 (Negative LDLr)p-value: 0.388Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Negative LDLr)p-value: 0.2057Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Negative LDLr)p-value: 0.2892Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Defective LDLr)p-value: 0.0052Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Negative LDLr)p-value: 0.0028Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Negative LDLr)p-value: 0.0026Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0493Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0253Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0394Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0003Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0002Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0002Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status
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
Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 28: Negative LDLr | -10.02 Percent Change | Standard Error 10.577 |
| Gemcabene | Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 56: Negative LDLr | -14.57 Percent Change | Standard Error 10.577 |
| Gemcabene | Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 84: Negative LDLr | -11.64 Percent Change | Standard Error 10.577 |
| Gemcabene | Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 28: Defective LDLr | -26.43 Percent Change | Standard Error 9.116 |
| Gemcabene | Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 56: Defective LDLr | -29.01 Percent Change | Standard Error 9.116 |
| Gemcabene | Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 84: Defective LDLr | -30.67 Percent Change | Standard Error 9.116 |
| Gemcabene | Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 28: Met EAS Clinical Diagnosis of HoFH | -17.30 Percent Change | Standard Error 8.845 |
| Gemcabene | Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 56: Met EAS Clinical Diagnosis of HoFH | -20.39 Percent Change | Standard Error 8.845 |
| Gemcabene | Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 84: Met EAS Clinical Diagnosis of HoFH | -19.69 Percent Change | Standard Error 8.845 |
| Gemcabene | Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 28: Not Met EAS Clinical Diagnosis of HoFH | -38.47 Percent Change | Standard Error 2.496 |
| Gemcabene | Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 56: Not Met EAS Clinical Diagnosis of HoFH | -42.48 Percent Change | Standard Error 2.496 |
| Gemcabene | Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status | Day 84: Not Met EAS Clinical Diagnosis of HoFH | -44.31 Percent Change | Standard Error 2.496 |
Comparison: Gemcabene 300 mg: Day 28 (Negative LDLr)p-value: 0.3657Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Negative LDLr)p-value: 0.1983Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Negative LDLr)p-value: 0.297Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Defective LDLr)p-value: 0.0092Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Defective LDLr)p-value: 0.0049Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Defective LDLr)p-value: 0.0033Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0618Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0298Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.0352Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0001Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)p-value: <0.0001Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH)p-value: <0.0001Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status
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
Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 28: Negative LDLr | -1.30 Percent Change | Standard Error 17.39 |
| Gemcabene | Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 56: Negative LDLr | -5.17 Percent Change | Standard Error 17.39 |
| Gemcabene | Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 84: Negative LDLr | -11.36 Percent Change | Standard Error 17.39 |
| Gemcabene | Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 28: Defective LDLr | -17.89 Percent Change | Standard Error 32.682 |
| Gemcabene | Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 56: Defective LDLr | -9.77 Percent Change | Standard Error 32.682 |
| Gemcabene | Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 84: Defective LDLr | -3.14 Percent Change | Standard Error 32.682 |
| Gemcabene | Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 28: Met EAS Clinical Diagnosis of HoFH | -7.02 Percent Change | Standard Error 14.51 |
| Gemcabene | Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 56: Met EAS Clinical Diagnosis of HoFH | 1.43 Percent Change | Standard Error 14.51 |
| Gemcabene | Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 84: Met EAS Clinical Diagnosis of HoFH | 2.98 Percent Change | Standard Error 14.51 |
| Gemcabene | Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 28: Not Met EAS Clinical Diagnosis of HoFH | -15.72 Percent Change | Standard Error 3.302 |
| Gemcabene | Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 56: Not Met EAS Clinical Diagnosis of HoFH | -26.60 Percent Change | Standard Error 3.302 |
| Gemcabene | Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status | Day 84: Not Met EAS Clinical Diagnosis of HoFH | -23.96 Percent Change | Standard Error 3.302 |
Comparison: Gemcabene 300 mg: Day 28 (Negative LDLr)p-value: 0.9417Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Negative LDLr)p-value: 0.7722Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Negative LDLr)p-value: 0.5283Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Defective LDLr)p-value: 0.5905Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Defective LDLr)p-value: 0.7682Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Defective LDLr)p-value: 0.9245Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.6325Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.9221Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.8387Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0089Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0013Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0019Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting Total Cholesterol (TC)
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting Total Cholesterol (TC) | Day 28 | -21.53 Percent Change | Standard Error 7.287 |
| Gemcabene | Percent Change From Baseline in Fasting Total Cholesterol (TC) | Day 56 | -24.85 Percent Change | Standard Error 7.287 |
| Gemcabene | Percent Change From Baseline in Fasting Total Cholesterol (TC) | Day 84 | -24.79 Percent Change | Standard Error 7.287 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.0057Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.0017Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.0017Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting Triglycerides (TG)
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting Triglycerides (TG) | Day 28 | -11.99 Percent Change | Standard Error 15.658 |
| Gemcabene | Percent Change From Baseline in Fasting Triglycerides (TG) | Day 56 | -8.37 Percent Change | Standard Error 15.658 |
| Gemcabene | Percent Change From Baseline in Fasting Triglycerides (TG) | Day 84 | -6.55 Percent Change | Standard Error 15.658 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.4489Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 84p-value: 0.5964Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.6785Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting VLDL-C
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting VLDL-C | Day 28 | -12.84 Percent Change | Standard Error 15.407 |
| Gemcabene | Percent Change From Baseline in Fasting VLDL-C | Day 56 | -7.79 Percent Change | Standard Error 15.407 |
| Gemcabene | Percent Change From Baseline in Fasting VLDL-C | Day 84 | -6.55 Percent Change | Standard Error 15.407 |
Comparison: Gemcabene 900 mg: Day 84p-value: 0.6732Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28p-value: 0.4103Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.6164Mixed Models Analysis
Secondary
Percent Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status
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
Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 28: Met the Genetic Criteria of LDLr | -2.29 Percent Change | Standard Error 17.315 |
| Gemcabene | Percent Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 56: Met the Genetic Criteria of LDLr | -4.81 Percent Change | Standard Error 17.315 |
| Gemcabene | Percent Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 84: Met the Genetic Criteria of LDLr | -12.19 Percent Change | Standard Error 17.315 |
| Gemcabene | Percent Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 28: Not Met the Genetic Criteria of LDLr | -18.82 Percent Change | Standard Error 30.652 |
| Gemcabene | Percent Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 56: Not Met the Genetic Criteria of LDLr | -9.22 Percent Change | Standard Error 30.652 |
| Gemcabene | Percent Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 84: Not Met the Genetic Criteria of LDLr | -2.82 Percent Change | Standard Error 30.652 |
| Gemcabene | Percent Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 28: Met EAS Clinical Diagnosis of HoFH | -8.09 Percent Change | Standard Error 14.502 |
| Gemcabene | Percent Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 56: Met EAS Clinical Diagnosis of HoFH | 2.06 Percent Change | Standard Error 14.502 |
| Gemcabene | Percent Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 84: Met EAS Clinical Diagnosis of HoFH | 2.72 Percent Change | Standard Error 14.502 |
| Gemcabene | Percent Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 28: Not Met EAS Clinical Diagnosis of HoFH | -15.53 Percent Change | Standard Error 3.488 |
| Gemcabene | Percent Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 56: Not Met EAS Clinical Diagnosis of HoFH | -25.75 Percent Change | Standard Error 3.488 |
| Gemcabene | Percent Change From Baseline in Fasting VLDL-C as Per Receptor Mutation Status | Day 84: Not Met EAS Clinical Diagnosis of HoFH | -22.80 Percent Change | Standard Error 3.488 |
Comparison: Gemcabene 300 mg: Day 28 (Negative LDLr)p-value: 0.8972Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Negative LDLr)p-value: 0.7867Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Negative LDLr)p-value: 0.4974Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Defective LDLr)p-value: 0.5464Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Defective LDLr)p-value: 0.7668Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Defective LDLr)p-value: 0.9276Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.5821Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.8881Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Met EAS Clinical Diagnosis of HoFH)p-value: 0.8525Mixed Models Analysis
Comparison: Gemcabene 300 mg: Day 28 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0112Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56 (Not Met EAS Clinical Diagnosis of HoFH)p-value: 0.0018Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84 (Not Met EAS Clinical Diagnosis of HoFH).p-value: 0.0028Mixed Models Analysis
Secondary
Percent Change From Baseline in Fibrinogen
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in Fibrinogen | Day 28 | 8.20 Percent Change | Standard Error 3.985 |
| Gemcabene | Percent Change From Baseline in Fibrinogen | Day 56 | -6.08 Percent Change | Standard Error 3.985 |
| Gemcabene | Percent Change From Baseline in Fibrinogen | Day 84 | -5.09 Percent Change | Standard Error 3.985 |
Comparison: Gemcabene 300 mg: Day 28p-value: 0.0587Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.1491Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.2224Mixed Models Analysis
Secondary
Percent Change From Baseline in High-sensitivity C-reactive Protein (hsCRP)
Time frame: Baseline, days 28, 56 and 84
Population: FAS Population.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Gemcabene | Percent Change From Baseline in High-sensitivity C-reactive Protein (hsCRP) | Day 28 | 63.11 Percent Change | Standard Error 26.022 |
| Gemcabene | Percent Change From Baseline in High-sensitivity C-reactive Protein (hsCRP) | Day 56 | -26.67 Percent Change | Standard Error 26.022 |
| Gemcabene | Percent Change From Baseline in High-sensitivity C-reactive Protein (hsCRP) | Day 84 | -16.89 Percent Change | Standard Error 26.022 |
Comparison: Gemcabene 300mg: Day 28p-value: 0.0294Mixed Models Analysis
Comparison: Gemcabene 600 mg: Day 56p-value: 0.3228Mixed Models Analysis
Comparison: Gemcabene 900 mg: Day 84p-value: 0.5268Mixed Models Analysis