Trial Outcomes & Findings for Efficacy and Safety of Gemcabene in Patients With Homozygous Familial Hypercholesterolemia on Stable, Lipid-Lowering Therapy (COBALT-1) (NCT NCT02722408)
NCT ID: NCT02722408
Last Updated: 2020-06-25
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
8 participants
Primary outcome timeframe
Baseline, day 28
Results posted on
2020-06-25
Participant Flow
Participant milestones
| Measure |
Gemcabene
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.
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|---|---|
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Overall Study
STARTED
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8
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Overall Study
COMPLETED
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8
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
FAS Population. Here, number analyzed signifies participants with available data .
Baseline characteristics by cohort
| Measure |
Gemcabene
n=8 Participants
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.
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|---|---|
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Age, Continuous
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52.8 years
STANDARD_DEVIATION 14.48 • n=8 Participants
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Sex: Female, Male
Female
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3 Participants
n=8 Participants
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Sex: Female, Male
Male
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5 Participants
n=8 Participants
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|
Ethnicity (NIH/OMB)
Hispanic or Latino
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1 Participants
n=8 Participants
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
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7 Participants
n=8 Participants
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Ethnicity (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=8 Participants
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Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=8 Participants
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Race (NIH/OMB)
Asian
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0 Participants
n=8 Participants
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Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=8 Participants
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Race (NIH/OMB)
Black or African American
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0 Participants
n=8 Participants
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Race (NIH/OMB)
White
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8 Participants
n=8 Participants
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Race (NIH/OMB)
More than one race
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0 Participants
n=8 Participants
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Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=8 Participants
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Low-density lipoprotein cholesterol (LDL-C)
|
351.25 Milligram per deciliter (mg/dL)
STANDARD_DEVIATION 181.849 • n=8 Participants
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Non-high-density lipoprotein cholesterol (non-HDL-C)
|
379.94 mg/dL
STANDARD_DEVIATION 177.258 • n=8 Participants
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Total Cholesterol (TC)
|
425.38 mg/dL
STANDARD_DEVIATION 167.058 • n=8 Participants
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Triglycerides (TG)
|
143.56 mg/dL
STANDARD_DEVIATION 62.672 • n=8 Participants
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High-density lipoprotein cholesterol (HDL-C)
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45.44 mg/dL
STANDARD_DEVIATION 18.661 • n=8 Participants
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Very low-density lipoprotein cholesterol (VLDL-C)
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28.69 mg/dL
STANDARD_DEVIATION 12.530 • n=8 Participants
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High-sensitivity C-reactive protein (hsCRP)
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5.95 mg/dL
STANDARD_DEVIATION 5.627 • n=8 Participants
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Fibrinogen
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436.9 mg/dL
STANDARD_DEVIATION 89.23 • n=8 Participants
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Lipoprotein(a)
|
113.5 mg/dL
STANDARD_DEVIATION 92.97 • n=8 Participants
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Apolipoprotein B (ApoB)
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221.3 mg/dL
STANDARD_DEVIATION 97.25 • n=8 Participants
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Apolipoprotein A-I (ApoA-I)
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127.9 mg/dL
STANDARD_DEVIATION 46.98 • n=8 Participants
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Apolipoprotein A-II (ApoA-II)
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30.3 mg/dL
STANDARD_DEVIATION 9.75 • n=8 Participants
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Apolipoprotein C-II (ApoC-II)
|
4.1 mg/dL
STANDARD_DEVIATION 1.21 • n=8 Participants
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Apolipoprotein C-III (ApoC-III)
|
10.6 mg/dL
STANDARD_DEVIATION 4.14 • n=8 Participants
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Apolipoprotein E (ApoE)
|
6.68 mg/dL
STANDARD_DEVIATION 1.738 • n=8 Participants
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LDL-C by Receptor Mutation Status
Negative LDLr
|
551.33 mg/dL
STANDARD_DEVIATION 105.652 • n=3 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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LDL-C by Receptor Mutation Status
Defective LDLr
|
231.20 mg/dL
STANDARD_DEVIATION 65.191 • n=5 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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LDL-C by Receptor Mutation Status
Met EAS Clinical Diagnosis of HoFH
|
374.33 mg/dL
STANDARD_DEVIATION 208.717 • n=6 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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LDL-C by Receptor Mutation Status
Not Met EAS Clinical Diagnosis of HoFH
|
282.00 mg/dL
STANDARD_DEVIATION 29.698 • n=2 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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Non-HDL-C by Receptor Mutation Status
Negative LDLr
|
572.83 mg/dL
STANDARD_DEVIATION 105.419 • n=3 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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Non-HDL-C by Receptor Mutation Status
Defective LDLr
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264.20 mg/dL
STANDARD_DEVIATION 69.127 • n=5 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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Non-HDL-C by Receptor Mutation Status
Met EAS Clinical Diagnosis of HoFH
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399.92 mg/dL
STANDARD_DEVIATION 205.100 • n=6 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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Non-HDL-C by Receptor Mutation Status
Not Met EAS Clinical Diagnosis of HoFH
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320.00 mg/dL
STANDARD_DEVIATION 5.657 • n=2 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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TC by Receptor Mutation Status
Negative LDLr
|
601.00 mg/dL
STANDARD_DEVIATION 100.714 • n=3 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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TC by Receptor Mutation Status
Defective LDLr
|
320.00 mg/dL
STANDARD_DEVIATION 82.189 • n=5 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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TC by Receptor Mutation Status
Met EAS Clinical Diagnosis of HoFH
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439.75 mg/dL
STANDARD_DEVIATION 195.112 • n=6 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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TC by Receptor Mutation Status
Not Met EAS Clinical Diagnosis of HoFH
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382.25 mg/dL
STANDARD_DEVIATION 7.425 • n=2 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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TG by Receptor Mutation Status
Negative LDLr
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107.67 mg/dL
STANDARD_DEVIATION 9.278 • n=3 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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TG by Receptor Mutation Status
Defective LDLr
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165.10 mg/dL
STANDARD_DEVIATION 72.694 • n=5 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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TG by Receptor Mutation Status
Met EAS Clinical Diagnosis of HoFH
|
128.17 mg/dL
STANDARD_DEVIATION 38.577 • n=6 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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TG by Receptor Mutation Status
Not Met EAS Clinical Diagnosis of HoFH
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189.75 mg/dL
STANDARD_DEVIATION 119.855 • n=2 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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HDL-C by Receptor Mutation Status
Negative LDLr
|
28.17 mg/dL
STANDARD_DEVIATION 4.726 • n=3 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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HDL-C by Receptor Mutation Status
Defective LDLr
|
55.80 mg/dL
STANDARD_DEVIATION 15.502 • n=5 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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HDL-C by Receptor Mutation Status
Met EAS Clinical Diagnosis of HoFH
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39.83 mg/dL
STANDARD_DEVIATION 17.394 • n=6 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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HDL-C by Receptor Mutation Status
Not Met EAS Clinical Diagnosis of HoFH
|
62.25 mg/dL
STANDARD_DEVIATION 13.081 • n=2 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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VLDL-C by Receptor Mutation Status
Negative LDLr
|
21.50 mg/dL
STANDARD_DEVIATION 1.803 • n=3 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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VLDL-C by Receptor Mutation Status
Defective LDLr
|
33.00 mg/dL
STANDARD_DEVIATION 14.530 • n=5 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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VLDL-C by Receptor Mutation Status
Met EAS Clinical Diagnosis of HoFH
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25.58 mg/dL
STANDARD_DEVIATION 7.612 • n=6 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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VLDL-C by Receptor Mutation Status
Not Met EAS Clinical Diagnosis of HoFH
|
38.00 mg/dL
STANDARD_DEVIATION 24.042 • n=2 Participants • FAS Population. Here, number analyzed signifies participants with available data .
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PRIMARY outcome
Timeframe: Baseline, day 28Population: 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.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
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|---|---|
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Percent Change From Baseline in LDL-C at Day 28
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-25.54 Percent Change
Standard Error 8.301
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PRIMARY outcome
Timeframe: Baseline, day 56Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
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|---|---|
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Percent Change From Baseline in LDL-C at Day 56
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-29.94 Percent Change
Standard Error 8.301
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PRIMARY outcome
Timeframe: Baseline, day 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
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|---|---|
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Percent Change From Baseline in LDL-C at Day 84
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-29.41 Percent Change
Standard Error 8.301
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SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
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|---|---|
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Change From Baseline in Fasting LDL-C
Day 28
|
-82.15 mg/dL
Standard Error 41.520
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Change From Baseline in Fasting LDL-C
Day 56
|
-99.77 mg/dL
Standard Error 41.520
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Change From Baseline in Fasting LDL-C
Day 84
|
-94.90 mg/dL
Standard Error 41.520
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SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
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|---|---|
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Percent Change From Baseline in Fasting Non-HDL-C
Day 28
|
-23.87 Percent Change
Standard Error 8.105
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Percent Change From Baseline in Fasting Non-HDL-C
Day 56
|
-27.33 Percent Change
Standard Error 8.105
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Percent Change From Baseline in Fasting Non-HDL-C
Day 84
|
-26.59 Percent Change
Standard Error 8.105
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SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
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|---|---|
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Change From Baseline in Fasting Non-HDL-C
Day 28
|
-86.64 mg/dL
Standard Error 44.380
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Change From Baseline in Fasting Non-HDL-C
Day 56
|
-102.89 mg/dL
Standard Error 44.380
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Change From Baseline in Fasting Non-HDL-C
Day 84
|
-98.26 mg/dL
Standard Error 44.380
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
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|---|---|
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Percent Change From Baseline in Fasting Total Cholesterol (TC)
Day 28
|
-21.53 Percent Change
Standard Error 7.287
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Percent Change From Baseline in Fasting Total Cholesterol (TC)
Day 56
|
-24.85 Percent Change
Standard Error 7.287
|
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Percent Change From Baseline in Fasting Total Cholesterol (TC)
Day 84
|
-24.79 Percent Change
Standard Error 7.287
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
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|---|---|
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Change From Baseline in Fasting Total Cholesterol (TC)
Day 28
|
-91.96 mg/dL
Standard Error 46.421
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Change From Baseline in Fasting Total Cholesterol (TC)
Day 56
|
-108.46 mg/dL
Standard Error 46.421
|
|
Change From Baseline in Fasting Total Cholesterol (TC)
Day 84
|
-104.34 mg/dL
Standard Error 46.421
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
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|---|---|
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Percent Change From Baseline in Fasting Triglycerides (TG)
Day 28
|
-11.99 Percent Change
Standard Error 15.658
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Percent Change From Baseline in Fasting Triglycerides (TG)
Day 56
|
-8.37 Percent Change
Standard Error 15.658
|
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Percent Change From Baseline in Fasting Triglycerides (TG)
Day 84
|
-6.55 Percent Change
Standard Error 15.658
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
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|---|---|
|
Change From Baseline in Fasting Triglycerides (TG)
Day 28
|
-27.15 mg/dL
Standard Error 21.615
|
|
Change From Baseline in Fasting Triglycerides (TG)
Day 56
|
-21.77 mg/dL
Standard Error 21.615
|
|
Change From Baseline in Fasting Triglycerides (TG)
Day 84
|
-22.27 mg/dL
Standard Error 21.615
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
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|---|---|
|
Percent Change From Baseline in Fasting HDL-C
Day 28
|
-11.54 Percent Change
Standard Error 3.906
|
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Percent Change From Baseline in Fasting HDL-C
Day 56
|
-12.95 Percent Change
Standard Error 3.906
|
|
Percent Change From Baseline in Fasting HDL-C
Day 84
|
-12.54 Percent Change
Standard Error 3.906
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
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|---|---|
|
Change From Baseline in Fasting HDL-C
Day 56
|
-5.05 mg/dL
Standard Error 1.306
|
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Change From Baseline in Fasting HDL-C
Day 28
|
-4.80 mg/dL
Standard Error 1.306
|
|
Change From Baseline in Fasting HDL-C
Day 84
|
-5.55 mg/dL
Standard Error 1.306
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Percent Change From Baseline in Fasting VLDL-C
Day 28
|
-12.84 Percent Change
Standard Error 15.407
|
|
Percent Change From Baseline in Fasting VLDL-C
Day 56
|
-7.79 Percent Change
Standard Error 15.407
|
|
Percent Change From Baseline in Fasting VLDL-C
Day 84
|
-6.55 Percent Change
Standard Error 15.407
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Change From Baseline in Fasting VLDL-C
Day 28
|
-5.57 mg/dL
Standard Error 4.261
|
|
Change From Baseline in Fasting VLDL-C
Day 56
|
-4.19 mg/dL
Standard Error 4.261
|
|
Change From Baseline in Fasting VLDL-C
Day 84
|
-4.44 mg/dL
Standard Error 4.261
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status
Day 28: Negative LDLr
|
-64.16 mg/dL
Standard Error 67.549
|
|
Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status
Day 56: Negative LDLr
|
-92.83 mg/dL
Standard Error 67.549
|
|
Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status
Day 84: Negative LDLr
|
-76.16 mg/dL
Standard Error 67.549
|
|
Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status
Day 28: Defective LDLr
|
-74.43 mg/dL
Standard Error 40.665
|
|
Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status
Day 56: Defective LDLr
|
-85.43 mg/dL
Standard Error 40.655
|
|
Change From Baseline in Fasting LDL-C as Per Receptor Mutation Status
Day 84: Defective LDLr
|
-87.63 mg/dL
Standard Error 40.655
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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 33.020
|
|
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 33.020
|
|
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 33.020
|
|
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
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status
Day 84: NegativeLDLr
|
-1.62 mg/dL
Standard Error 3.738
|
|
Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status
Day 28: NegativeLDLr
|
-4.96 mg/dL
Standard Error 3.738
|
|
Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status
Day 56: NegativeLDLr
|
-5.29 mg/dL
Standard Error 3.738
|
|
Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status
Day 28: Defective LDLr
|
-5.19 mg/dL
Standard Error 1.508
|
|
Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status
Day 56: Defective LDLr
|
-5.39 mg/dL
Standard Error 1.508
|
|
Change From Baseline in Fasting HDL-C as Per Receptor Mutation Status
Day 84: Defective LDLr
|
-8.39 mg/dL
Standard Error 1.508
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status
Day 28: Defective LDLr
|
-26.43 Percent Change
Standard Error 9.116
|
|
Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status
Day 56: Defective LDLr
|
-29.01 Percent Change
Standard Error 9.116
|
|
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
|
|
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
|
|
Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status
Day 28: Negative LDLr
|
-10.02 Percent Change
Standard Error 10.577
|
|
Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status
Day 56: Negative LDLr
|
-14.57 Percent Change
Standard Error 10.577
|
|
Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status
Day 84: Negative LDLr
|
-11.64 Percent Change
Standard Error 10.577
|
|
Percent Change From Baseline in Fasting TC as Per Receptor Mutation Status
Day 84: Defective LDLr
|
-30.67 Percent Change
Standard Error 9.116
|
|
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
|
|
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
|
|
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
|
|
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
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Change From Baseline in Fasting TC as Per Receptor Mutation Status
Day 28: Negative LDLr
|
-70.92 mg/dL
Standard Error 75.095
|
|
Change From Baseline in Fasting TC as Per Receptor Mutation Status
Day 56: Negative LDLr
|
-100.58 mg/dL
Standard Error 75.095
|
|
Change From Baseline in Fasting TC as Per Receptor Mutation Status
Day 84: Negative LDLr
|
-81.92 mg/dL
Standard Error 75.095
|
|
Change From Baseline in Fasting TC as Per Receptor Mutation Status
Day 28: Defective LDLr
|
-84.61 mg/dL
Standard Error 57.378
|
|
Change From Baseline in Fasting TC as Per Receptor Mutation Status
Day 56: Defective LDLr
|
-93.21 mg/dL
Standard Error 57.378
|
|
Change From Baseline in Fasting TC as Per Receptor Mutation Status
Day 84: Defective LDLr
|
-97.81 mg/dL
Standard Error 57.378
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status
Day 28: Negative LDLr
|
-1.30 Percent Change
Standard Error 17.390
|
|
Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status
Day 56: Negative LDLr
|
-5.17 Percent Change
Standard Error 17.390
|
|
Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status
Day 84: Negative LDLr
|
-11.36 Percent Change
Standard Error 17.390
|
|
Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status
Day 28: Defective LDLr
|
-17.89 Percent Change
Standard Error 32.682
|
|
Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status
Day 56: Defective LDLr
|
-9.77 Percent Change
Standard Error 32.682
|
|
Percent Change From Baseline in Fasting TG as Per Receptor Mutation Status
Day 84: Defective LDLr
|
-3.14 Percent Change
Standard Error 32.682
|
|
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 14.510
|
|
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 14.510
|
|
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 14.510
|
|
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
|
|
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
|
|
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
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population. Here, number analyzed signifies those participants who were evaluable at each specified time point.
Receptor mutation status was categorized as LDLr status and EAS clinical diagnosis of HoFH which was reported in this outcome measure.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
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
|
|
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
|
|
Change From Baseline in Fasting TG as Per Receptor Mutation Status
Day 28: Negative LDLr
|
-2.00 mg/dL
Standard Error 19.308
|
|
Change From Baseline in Fasting TG as Per Receptor Mutation Status
Day 56: Negative LDLr
|
-6.33 mg/dL
Standard Error 19.308
|
|
Change From Baseline in Fasting TG as Per Receptor Mutation Status
Day 84: Negative LDLr
|
-13.33 mg/dL
Standard Error 19.308
|
|
Change From Baseline in Fasting TG as Per Receptor Mutation Status
Day 28: Defective LDLr
|
-41.94 mg/dL
Standard Error 35.093
|
|
Change From Baseline in Fasting TG as Per Receptor Mutation Status
Day 56: Defective LDLr
|
-30.74 mg/dL
Standard Error 35.093
|
|
Change From Baseline in Fasting TG as Per Receptor Mutation Status
Day 84: Defective LDLr
|
-27.34 mg/dL
Standard Error 35.093
|
|
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
|
|
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
|
|
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
|
|
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
|
SECONDARY outcome
Timeframe: Days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Number of Participants Achieving LDL-C Reduction of ≥15%
Day 28
|
6 Participants
|
|
Number of Participants Achieving LDL-C Reduction of ≥15%
Day 56
|
6 Participants
|
|
Number of Participants Achieving LDL-C Reduction of ≥15%
Day 84
|
7 Participants
|
SECONDARY outcome
Timeframe: Days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Number of Participants Achieving LDL-C Reduction of ≥20%
Day 28
|
5 Participants
|
|
Number of Participants Achieving LDL-C Reduction of ≥20%
Day 56
|
6 Participants
|
|
Number of Participants Achieving LDL-C Reduction of ≥20%
Day 84
|
6 Participants
|
SECONDARY outcome
Timeframe: Days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Number of Participants Achieving LDL-C Reduction of ≥25%
Day 28
|
5 Participants
|
|
Number of Participants Achieving LDL-C Reduction of ≥25%
Day 56
|
5 Participants
|
|
Number of Participants Achieving LDL-C Reduction of ≥25%
Day 84
|
5 Participants
|
SECONDARY outcome
Timeframe: Days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Number of Participants Achieving LDL-C Reduction of ≥30%
Day 28
|
4 Participants
|
|
Number of Participants Achieving LDL-C Reduction of ≥30%
Day 56
|
5 Participants
|
|
Number of Participants Achieving LDL-C Reduction of ≥30%
Day 84
|
4 Participants
|
SECONDARY outcome
Timeframe: Days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Number of Participants Achieving an LDL-C Value <100 mg/dL (2.59 mmol/L)
Day 28
|
1 Participants
|
|
Number of Participants Achieving an LDL-C Value <100 mg/dL (2.59 mmol/L)
Day 56
|
1 Participants
|
|
Number of Participants Achieving an LDL-C Value <100 mg/dL (2.59 mmol/L)
Day 84
|
1 Participants
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Percent Change From Baseline in High-sensitivity C-reactive Protein (hsCRP)
Day 28
|
63.11 Percent Change
Standard Error 26.022
|
|
Percent Change From Baseline in High-sensitivity C-reactive Protein (hsCRP)
Day 56
|
-26.67 Percent Change
Standard Error 26.022
|
|
Percent Change From Baseline in High-sensitivity C-reactive Protein (hsCRP)
Day 84
|
-16.89 Percent Change
Standard Error 26.022
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Change From Baseline in High-sensitivity C-reactive Protein (hsCRP)
Day 28
|
5.30 mg/L
Standard Error 3.105
|
|
Change From Baseline in High-sensitivity C-reactive Protein (hsCRP)
Day 56
|
-1.91 mg/L
Standard Error 3.105
|
|
Change From Baseline in High-sensitivity C-reactive Protein (hsCRP)
Day 84
|
0.46 mg/L
Standard Error 3.105
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Percent Change From Baseline in Fibrinogen
Day 28
|
8.20 Percent Change
Standard Error 3.985
|
|
Percent Change From Baseline in Fibrinogen
Day 56
|
-6.08 Percent Change
Standard Error 3.985
|
|
Percent Change From Baseline in Fibrinogen
Day 84
|
-5.09 Percent Change
Standard Error 3.985
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Change From Baseline in Fibrinogen
Day 28
|
34.17 mg/dL
Standard Error 19.141
|
|
Change From Baseline in Fibrinogen
Day 56
|
-34.58 mg/dL
Standard Error 19.141
|
|
Change From Baseline in Fibrinogen
Day 84
|
-28.46 mg/dL
Standard Error 19.141
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Percent Change From Baseline in Fasting Lipoprotein(a)
Day 84
|
15.62 Percent Change
Standard Error 12.362
|
|
Percent Change From Baseline in Fasting Lipoprotein(a)
Day 28
|
6.90 Percent Change
Standard Error 12.362
|
|
Percent Change From Baseline in Fasting Lipoprotein(a)
Day 56
|
5.84 Percent Change
Standard Error 12.362
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Change From Baseline in Fasting Lipoprotein(a)
Day 28
|
19.39 nmol/L
Standard Error 26.804
|
|
Change From Baseline in Fasting Lipoprotein(a)
Day 56
|
18.51 nmol/L
Standard Error 26.804
|
|
Change From Baseline in Fasting Lipoprotein(a)
Day 84
|
21.51 nmol/L
Standard Error 26.804
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Percent Change From Baseline in Fasting Apolipoprotein B
Day 28
|
-17.95 Percent Change
Standard Error 9.034
|
|
Percent Change From Baseline in Fasting Apolipoprotein B
Day 56
|
-23.98 Percent Change
Standard Error 9.034
|
|
Percent Change From Baseline in Fasting Apolipoprotein B
Day 84
|
-21.58 Percent Change
Standard Error 9.034
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Change From Baseline in Fasting Apolipoprotein B
Day 28
|
-39.24 mg/dL
Standard Error 19.882
|
|
Change From Baseline in Fasting Apolipoprotein B
Day 56
|
-57.87 mg/dL
Standard Error 19.882
|
|
Change From Baseline in Fasting Apolipoprotein B
Day 84
|
-49.37 mg/dL
Standard Error 19.882
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Percent Change From Baseline in Fasting Apolipoprotein A-I
Day 28
|
-6.27 Percent Change
Standard Error 4.082
|
|
Percent Change From Baseline in Fasting Apolipoprotein A-I
Day 56
|
-3.86 Percent Change
Standard Error 4.082
|
|
Percent Change From Baseline in Fasting Apolipoprotein A-I
Day 84
|
-8.06 Percent Change
Standard Error 4.082
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Change From Baseline in Fasting Apolipoprotein A-I
Day 28
|
-7.26 mg/dL
Standard Error 4.967
|
|
Change From Baseline in Fasting Apolipoprotein A-I
Day 56
|
-4.88 mg/dL
Standard Error 4.967
|
|
Change From Baseline in Fasting Apolipoprotein A-I
Day 84
|
-11.88 mg/dL
Standard Error 4.967
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Percent Change From Baseline in Fasting Apolipoprotein A-II
Day 28
|
2.00 Percent Change
Standard Error 5.473
|
|
Percent Change From Baseline in Fasting Apolipoprotein A-II
Day 56
|
4.41 Percent Change
Standard Error 5.473
|
|
Percent Change From Baseline in Fasting Apolipoprotein A-II
Day 84
|
1.97 Percent Change
Standard Error 5.473
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Change From Baseline in Fasting Apolipoprotein A-II
Day 28
|
0.38 mg/dL
Standard Error 1.423
|
|
Change From Baseline in Fasting Apolipoprotein A-II
Day 56
|
1.00 mg/dL
Standard Error 1.423
|
|
Change From Baseline in Fasting Apolipoprotein A-II
Day 84
|
0.25 mg/dL
Standard Error 1.423
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population. Here, overall number of participants analyzed (N) signifies those participants who were evaluable for this outcome measure only.
Outcome measures
| Measure |
Gemcabene
n=7 Participants
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.
|
|---|---|
|
Percent Change From Baseline in Fasting Apolipoprotein C-II
Day 28
|
-1.69 Percent Change
Standard Error 74.431
|
|
Percent Change From Baseline in Fasting Apolipoprotein C-II
Day 56
|
8.31 Percent Change
Standard Error 74.431
|
|
Percent Change From Baseline in Fasting Apolipoprotein C-II
Day 84
|
21.64 Percent Change
Standard Error 74.431
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population. Here, overall number of participants analyzed (N) signifies those participants who were evaluable for this outcome measure only.
Outcome measures
| Measure |
Gemcabene
n=7 Participants
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.
|
|---|---|
|
Change From Baseline in Fasting Apolipoprotein C-II
Day 28
|
0.71 mg/dL
Standard Error 0.615
|
|
Change From Baseline in Fasting Apolipoprotein C-II
Day 56
|
1.14 mg/dL
Standard Error 0.615
|
|
Change From Baseline in Fasting Apolipoprotein C-II
Day 84
|
1.71 mg/dL
Standard Error 0.615
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Percent Change From Baseline in Fasting Apolipoprotein C-III
Day 28
|
-8.86 Percent Change
Standard Error 11.616
|
|
Percent Change From Baseline in Fasting Apolipoprotein C-III
Day 56
|
-10.71 Percent Change
Standard Error 11.616
|
|
Percent Change From Baseline in Fasting Apolipoprotein C-III
Day 84
|
-7.47 Percent Change
Standard Error 11.616
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Change From Baseline in Fasting Apolipoprotein C-III
Day 28
|
-1.37 mg/dL
Standard Error 1.260
|
|
Change From Baseline in Fasting Apolipoprotein C-III
Day 56
|
-1.62 mg/dL
Standard Error 1.260
|
|
Change From Baseline in Fasting Apolipoprotein C-III
Day 84
|
-1.37 mg/dL
Standard Error 1.260
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Percent Change From Baseline in Fasting Apolipoprotein E
Day 56
|
-23.01 Percent Change
Standard Error 8.699
|
|
Percent Change From Baseline in Fasting Apolipoprotein E
Day 28
|
-19.57 Percent Change
Standard Error 8.699
|
|
Percent Change From Baseline in Fasting Apolipoprotein E
Day 84
|
-19.20 Percent Change
Standard Error 8.699
|
SECONDARY outcome
Timeframe: Baseline, days 28, 56 and 84Population: FAS Population.
Outcome measures
| Measure |
Gemcabene
n=8 Participants
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.
|
|---|---|
|
Change From Baseline in Fasting Apolipoprotein E
Day 28
|
-1.40 mg/dL
Standard Error 0.641
|
|
Change From Baseline in Fasting Apolipoprotein E
Day 56
|
-1.65 mg/dL
Standard Error 0.641
|
|
Change From Baseline in Fasting Apolipoprotein E
Day 84
|
-1.45 mg/dL
Standard Error 0.641
|
Adverse Events
Gemcabene 300 mg
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Gemcabene 600 mg
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Gemcabene 900 mg
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Gemcabene 300 mg
n=8 participants at risk
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 300 mg of Gemcabene, orally once daily from day 1 to 28.
|
Gemcabene 600 mg
n=8 participants at risk
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 600 mg of Gemcabene, orally once daily from day 29 to 56.
|
Gemcabene 900 mg
n=8 participants at risk
Participants with homozygous familial hypercholesterolemia (HoFH) on stable lipid lowering therapy received 900 mg of Gemcabene, orally once daily from day 57 to 84.
|
|---|---|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
25.0%
2/8 • Baseline to end of study (Up to 112 days)
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
|
Gastrointestinal disorders
Dry mouth
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
|
Gastrointestinal disorders
Dyspepsia
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
|
Gastrointestinal disorders
Nausea
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
|
Gastrointestinal disorders
Rectal haemorrhage
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
|
Infections and infestations
Cystitis
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
|
Infections and infestations
Influenza
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
|
Blood and lymphatic system disorders
Anaemia
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
|
General disorders
Energy increased
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
|
General disorders
Influenza like illness
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
|
Nervous system disorders
Headache
|
25.0%
2/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
|
Renal and urinary disorders
Myoglobinuria
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
|
Renal and urinary disorders
Proteinuria
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
|
Injury, poisoning and procedural complications
Thermal burn
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
|
Investigations
Haemoglobin decreased
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
|
Vascular disorders
Hypertension
|
12.5%
1/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
0.00%
0/8 • Baseline to end of study (Up to 112 days)
|
Additional Information
Vice President Clinical Operations
NeuroBo Pharmaceuticals
Phone: +1 (857) 702-9600
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place