Trial Outcomes & Findings for Study of MGL-3196 in Patients With Heterozygous Familial Hypercholesterolemia (HeFH) (NCT NCT03038022)
NCT ID: NCT03038022
Last Updated: 2025-12-23
Results Overview
LDL-C was determined by ultracentrifugation or direct measure. Least-squares (LS) mean was provided for the comparison of MGL-3196 versus placebo and it used a linear model with percent change from baseline as the dependent variable and treatment as a factor.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
116 participants
Primary outcome timeframe
Baseline up to Week 12
Results posted on
2025-12-23
Participant Flow
Participant milestones
| Measure |
MGL-3196
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured pharmacokinetic (PK) assessments of MGL-3196 exposure.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
78
|
38
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
78
|
38
|
|
Overall Study
COMPLETED
|
72
|
36
|
|
Overall Study
NOT COMPLETED
|
6
|
2
|
Reasons for withdrawal
| Measure |
MGL-3196
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured pharmacokinetic (PK) assessments of MGL-3196 exposure.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
2
|
0
|
|
Overall Study
Adverse Event
|
4
|
2
|
Baseline Characteristics
Study of MGL-3196 in Patients With Heterozygous Familial Hypercholesterolemia (HeFH)
Baseline characteristics by cohort
| Measure |
MGL-3196
n=78 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=38 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Total
n=116 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Sex: Female, Male
Male
|
39 Participants
n=9 Participants
|
22 Participants
n=6 Participants
|
61 Participants
n=9 Participants
|
|
Sex: Female, Male
Female
|
39 Participants
n=9 Participants
|
16 Participants
n=6 Participants
|
55 Participants
n=9 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=9 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=9 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
56 Participants
n=9 Participants
|
25 Participants
n=6 Participants
|
81 Participants
n=9 Participants
|
|
Age, Categorical
>=65 years
|
22 Participants
n=9 Participants
|
13 Participants
n=6 Participants
|
35 Participants
n=9 Participants
|
|
Age, Continuous
|
56.4 years
STANDARD_DEVIATION 12.38 • n=9 Participants
|
59.1 years
STANDARD_DEVIATION 11.33 • n=6 Participants
|
57.3 years
STANDARD_DEVIATION 12.06 • n=9 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=9 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
78 Participants
n=9 Participants
|
38 Participants
n=6 Participants
|
116 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=9 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=9 Participants
|
1 Participants
n=6 Participants
|
1 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=9 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=9 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
White
|
78 Participants
n=9 Participants
|
37 Participants
n=6 Participants
|
115 Participants
n=9 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=9 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=9 Participants
|
|
Baseline Low-density Lipoprotein Cholesterol (LDL-C) (Direct measure or ultracentrifugation)
|
131.4 mg/dL
STANDARD_DEVIATION 47.24 • n=9 Participants
|
137.0 mg/dL
STANDARD_DEVIATION 57.53 • n=6 Participants
|
133.3 mg/dL
STANDARD_DEVIATION 50.65 • n=9 Participants
|
|
Statin Use
Atorvastatin
|
40 Participants
n=9 Participants
|
16 Participants
n=6 Participants
|
56 Participants
n=9 Participants
|
|
Statin Use
Rosuvastatin
|
34 Participants
n=9 Participants
|
17 Participants
n=6 Participants
|
51 Participants
n=9 Participants
|
|
Statin Use
Pravastatin
|
1 Participants
n=9 Participants
|
1 Participants
n=6 Participants
|
2 Participants
n=9 Participants
|
|
Statin Use
None
|
3 Participants
n=9 Participants
|
4 Participants
n=6 Participants
|
7 Participants
n=9 Participants
|
PRIMARY outcome
Timeframe: Baseline up to Week 12Population: Modified Intent-to-Treat (mITT) population: all participants who were randomized in the study, received at least 2 weeks of study drug, and had a valid lipid measurement at Week 2 or later.
LDL-C was determined by ultracentrifugation or direct measure. Least-squares (LS) mean was provided for the comparison of MGL-3196 versus placebo and it used a linear model with percent change from baseline as the dependent variable and treatment as a factor.
Outcome measures
| Measure |
MGL-3196
n=76 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=37 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Percent Change in LDL-C From Baseline To Week 12
|
-10.6 percent change
Standard Error 2.59
|
8.2 percent change
Standard Error 3.71
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12Population: Safety population: all participants who were randomized in the study and received at least 1 dose of study drug.
T4 was assessed at each study visit.
Outcome measures
| Measure |
MGL-3196
n=78 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=38 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Change From Baseline to Week 12 of Free Thyroxine (T4)
|
-0.268 ng/dL
Standard Error 0.0130
|
0.009 ng/dL
Standard Error 0.0185
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12Population: Safety population: all participants who were randomized in the study and received at least 1 dose of study drug.
Free T3 was assessed at each study visit.
Outcome measures
| Measure |
MGL-3196
n=78 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=38 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Change From Baseline to Week 12 of Free Triiodothyronine (T3)
|
-0.04 ng/L
Standard Error 0.038
|
0.10 ng/L
Standard Error 0.054
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12Population: Safety population: all participants who were randomized in the study and received at least 1 dose of study drug.
TSH was assessed at each study visit.
Outcome measures
| Measure |
MGL-3196
n=78 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=38 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Change From Baseline to Week 12 of Thyrotropin (TSH)
|
-0.027 mIU/L
Standard Error 0.0690
|
0.114 mIU/L
Standard Error 0.0987
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12Population: Safety population: all participants who were randomized in the study and received at least 1 dose of study drug.
TBG was assessed at all study visits except the Screening Visit.
Outcome measures
| Measure |
MGL-3196
n=78 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=38 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Change From Baseline to Week 12 of Thyroxine Binding Globulin (TBG)
|
-2.31 mg/L
Standard Error 0.256
|
-0.44 mg/L
Standard Error 0.367
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12Population: Safety population: all participants who were randomized in the study and received at least 1 dose of study drug.
Reverse T3 was assessed at each study visit.
Outcome measures
| Measure |
MGL-3196
n=78 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=38 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Change From Baseline to Week 12 of Reverse Triiodothyronine (T3)
|
-4.36 ng/dL
Standard Error 0.392
|
-0.58 ng/dL
Standard Error 0.554
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12Non-HDL-C were determined by ultracentrifugation. LS mean was provided for the comparison of MGL-3196 versus placebo and it used a linear model with percent change from baseline as the dependent variable and treatment as a factor
Outcome measures
| Measure |
MGL-3196
n=76 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=37 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Percent Change in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 12
|
-12.0 percent change
Standard Error 2.41
|
7.5 percent change
Standard Error 3.46
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12Population: mITT population: all participants who were randomized in the study, received at least 2 weeks of study drug, and had a valid lipid measurement at Week 2 or later.
Triglycerides were determined by ultracentrifugation. LS mean was provided for the comparison of MGL-3196 versus placebo and it used a linear model with percent change from baseline as the dependent variable and treatment as a factor.
Outcome measures
| Measure |
MGL-3196
n=76 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=37 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Percent Change In Triglycerides From Baseline To Week 12
|
-18.3 percent change
Standard Error 3.24
|
7.2 percent change
Standard Error 4.64
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12Population: mITT population: all participants who were randomized in the study, received at least 2 weeks of study drug, and had a valid lipid measurement at Week 2 or later.
Lp(a) was determined by ultracentrifugation. LS mean was provided for the comparison of MGL-3196 versus placebo and it used a linear model with percent change from baseline as the dependent variable and treatment as a factor.
Outcome measures
| Measure |
MGL-3196
n=76 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=37 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Percent Change In Lipoprotein(a) (Lp[a]) From Baseline To Week 12
|
-21.8 percent change
Standard Error 2.87
|
4.4 percent change
Standard Error 4.11
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12Apolipoprotein CIII was determined by ultracentrifugation. LS mean was provided for the comparison of MGL-3196 versus placebo and it used a linear model with percent change from baseline as the dependent variable and treatment as a factor.
Outcome measures
| Measure |
MGL-3196
n=76 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=37 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Percent Change in Apolipoprotein CIII From Baseline to Week 12
|
-19.3 percent change
Standard Error 2.9
|
3.4 percent change
Standard Error 4.1
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12Population: mITT population: all participants who were randomized in the study, received at least 2 weeks of study drug, and had a valid lipid measurement at Week 2 or later.
ApoB was determined by ultracentrifugation. LS mean was provided for the comparison of MGL-3196 versus placebo and it used a linear model with percent change from baseline as the dependent variable and treatment as a factor.
Outcome measures
| Measure |
MGL-3196
n=76 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=37 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Percent Change In Apolipoprotein B (ApoB) From Baseline To Week 12
|
-14.2 percent change
Standard Error 1.66
|
3.8 percent change
Standard Error 2.37
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12The ApoB/ApoA1 ratio was determined by ultracentrifugation. LS mean was provided for the comparison of MGL-3196 versus placebo and it used a linear model with percent change from baseline as the dependent variable and treatment as a factor.
Outcome measures
| Measure |
MGL-3196
n=76 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=37 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Percent Change in Apolipoprotein B/Apolipoprotein A1 (ApoB/ApoA1) Ratio From Baseline to Week 12
|
-5.0 percent change
Standard Error 1.8
|
4.2 percent change
Standard Error 2.5
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12The Cholesterol/HDL-C Ratio was determined by ultracentrifugation. LS mean was provided for the comparison of MGL-3196 versus placebo and it used a linear model with percent change from baseline as the dependent variable and treatment as a factor.
Outcome measures
| Measure |
MGL-3196
n=76 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=37 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Percent Change In Cholesterol/HDL-C Ratio From Baseline to Week 12
|
-3.5 percent change
Standard Error 2.3
|
6.3 percent change
Standard Error 3.3
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12LDL-C was determined by ultracentrifugation or direct measure . Least-squares (LS) mean was provided for the comparison of MGL-3196 versus placebo and it used a linear model with percent change from baseline as the dependent variable and treatment as a factor.
Outcome measures
| Measure |
MGL-3196
n=76 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=37 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Absolute Change in LDL-C From Baseline to Week 12
|
-17.0 mg/dL
Standard Error 3.31
|
9.9 mg/dL
Standard Error 4.74
|
—
|
SECONDARY outcome
Timeframe: Baseline to Week 2 and Week 4LDL-C was determined by ultracentrifugation or direct measure. Least-squares (LS) mean was provided for the comparison of MGL-3196 versus placebo and it used a linear model with percent change from baseline as the dependent variable and treatment as a factor.
Outcome measures
| Measure |
MGL-3196
n=76 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=37 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Percent Change From Baseline in LDL-C at Week 2 and Week 4 in Patients in the 100 mg and the 60 mg Groups
Week 2
|
-16.6 percent change
Interval -22.6 to -10.6
|
1.6 percent change
Interval -3.2 to 6.5
|
—
|
|
Percent Change From Baseline in LDL-C at Week 2 and Week 4 in Patients in the 100 mg and the 60 mg Groups
Week 4
|
-9.8 percent change
Interval -16.3 to -3.4
|
0.1 percent change
Interval -5.2 to 5.4
|
—
|
SECONDARY outcome
Timeframe: Baseline to Week 12Population: mITT Population
The effect on percent change in LDL-C from baseline to Week 12 was determined by patient exposure category (higher/lower). Patients taking MGL-3196 were categorized into lower and higher exposure groups. Patients in the higher exposure group must either have had estimated Week 2 AUC ≥5,000 mg∙h/L, or if Week 2 AUC \<5,000 mg∙h/L but Week 4 pre-dose concentration \>1.5 ng/mL (if on 60 mg MGL-3196). All other patients were in the lower exposure group.
Outcome measures
| Measure |
MGL-3196
n=37 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=39 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
n=37 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Percent Change in LDL-C From Baseline to Week 12 by Systemic Exposure Category
|
-8.1 percent change
Standard Error 3.71
|
-13.0 percent change
Standard Error 3.62
|
8.2 percent change
Standard Error 3.71
|
SECONDARY outcome
Timeframe: Baseline up to Week 12The effect of once-daily oral dosing of MGL-3196 versus placebo for 12 weeks in terms of change from baseline was assessed for LDL Particles.
Outcome measures
| Measure |
MGL-3196
n=74 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=35 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Change in Lipid Particle Concentration From Baseline To Week 12: Total LDL Particles
|
1223 nmol/L
Standard Deviation 334
|
1428 nmol/L
Standard Deviation 475
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12The effect of once-daily oral dosing of MGL-3196 versus placebo for 12 weeks in terms of change from baseline was assessed for Small LDL Particles.
Outcome measures
| Measure |
MGL-3196
n=74 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=35 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Change in Lipid Particle Concentration From Baseline To Week 12: Small LDL Particles
|
655 nmol/L
Standard Deviation 214
|
760 nmol/L
Standard Deviation 265
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12The effect of once-daily oral dosing of MGL-3196 versus placebo for 12 weeks in terms of change from baseline was assessed for Intermediate LDL Particles.
Outcome measures
| Measure |
MGL-3196
n=74 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=35 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Change in Lipid Particle Concentration From Baseline To Week 12: Intermediate LDL Particles
|
157 nmol/L
Standard Deviation 96
|
168 nmol/L
Standard Deviation 184
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12The effect of once-daily oral dosing of MGL-3196 versus placebo for 12 weeks in terms of change from baseline was assessed for Large LDL Particles.
Outcome measures
| Measure |
MGL-3196
n=74 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=35 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Change in Lipid Particle Concentration From Baseline To Week 12: Large LDL Particles
|
411 nmol/L
Standard Deviation 247
|
501 nmol/L
Standard Deviation 302
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12The effect of once-daily oral dosing of MGL-3196 versus placebo for 12 weeks in terms of change from baseline was assessed for VLDL and Chylomicron Particles.
Outcome measures
| Measure |
MGL-3196
n=74 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=35 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Change in Lipid Particle Concentration From Baseline To Week 12:Total Very Low-Density Lipoprotein (VLDL) and Chylomicron Particles
|
24.8 nmol/L
Standard Deviation 18.5
|
37.3 nmol/L
Standard Deviation 24.7
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12The effect of once-daily oral dosing of MGL-3196 versus placebo for 12 weeks in terms of change from baseline was assessed for Total HDL Particles.
Outcome measures
| Measure |
MGL-3196
n=74 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=35 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Change in Lipid Particle Concentration From Baseline To Week 12: Total HDL Particles
|
29.79 micromol/L
Standard Deviation 4.699
|
30.92 micromol/L
Standard Deviation 5.940
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12The effect of once-daily oral dosing of MGL-3196 versus placebo for 12 weeks in terms of change from baseline was assessed for LDL Particle size.
Outcome measures
| Measure |
MGL-3196
n=74 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=35 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Change in Lipid Particle Concentration From Baseline To Week 12: LDL Particle Size
|
20.94 nm
Standard Deviation 0.546
|
20.89 nm
Standard Deviation 0.549
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12The effect of once-daily oral dosing of MGL-3196 versus placebo for 12 weeks in terms of change from baseline was assessed for VLDL Particle Size.
Outcome measures
| Measure |
MGL-3196
n=67 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=34 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Change in Lipid Particle Concentration From Baseline To Week 12: VLDL Particle Size
|
52.27 nm
Standard Deviation 7.063
|
51.33 nm
Standard Deviation 8.801
|
—
|
SECONDARY outcome
Timeframe: Baseline up to Week 12The effect of once-daily oral dosing of MGL-3196 versus placebo for 12 weeks in terms of change from baseline was assessed for HDL Particle Size.
Outcome measures
| Measure |
MGL-3196
n=74 Participants
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=35 Participants
Participants administered matching oral placebo once daily for 12 weeks.
|
Placebo
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|---|
|
Mean Change in Lipid Particle Concentration From Baseline To Week 12: HDL Particle Size
|
9.30 nm
Standard Deviation 0.589
|
9.19 nm
Standard Deviation 0.586
|
—
|
Adverse Events
MGL-3196
Serious events: 0 serious events
Other events: 65 other events
Deaths: 0 deaths
Placebo
Serious events: 1 serious events
Other events: 28 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
MGL-3196
n=78 participants at risk
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=38 participants at risk
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|
|
General disorders
Pyrexia
|
0.00%
0/78 • Baseline (Day 1) up to Week 12
|
2.6%
1/38 • Baseline (Day 1) up to Week 12
|
Other adverse events
| Measure |
MGL-3196
n=78 participants at risk
Participants randomized to MGL-3196 received 100 mg daily during the first 2 weeks, 60 mg daily during Weeks 2 to 4, and then either 60 or 100 mg daily to Week 12 based on Week 2 measured PK assessments of MGL-3196 exposure.
|
Placebo
n=38 participants at risk
Participants administered matching oral placebo once daily for 12 weeks.
|
|---|---|---|
|
Investigations
Hepatic enzyme increased
|
9.0%
7/78 • Baseline (Day 1) up to Week 12
|
0.00%
0/38 • Baseline (Day 1) up to Week 12
|
|
Infections and infestations
Nasopharyngitis
|
12.8%
10/78 • Baseline (Day 1) up to Week 12
|
15.8%
6/38 • Baseline (Day 1) up to Week 12
|
|
Nervous system disorders
Headache
|
12.8%
10/78 • Baseline (Day 1) up to Week 12
|
15.8%
6/38 • Baseline (Day 1) up to Week 12
|
|
Nervous system disorders
Dizziness
|
5.1%
4/78 • Baseline (Day 1) up to Week 12
|
10.5%
4/38 • Baseline (Day 1) up to Week 12
|
|
General disorders
Fatigue
|
9.0%
7/78 • Baseline (Day 1) up to Week 12
|
5.3%
2/38 • Baseline (Day 1) up to Week 12
|
|
Skin and subcutaneous tissue disorders
Night Sweats
|
0.00%
0/78 • Baseline (Day 1) up to Week 12
|
5.3%
2/38 • Baseline (Day 1) up to Week 12
|
|
Injury, poisoning and procedural complications
Fall
|
2.6%
2/78 • Baseline (Day 1) up to Week 12
|
5.3%
2/38 • Baseline (Day 1) up to Week 12
|
|
Psychiatric disorders
Insomnia
|
1.3%
1/78 • Baseline (Day 1) up to Week 12
|
5.3%
2/38 • Baseline (Day 1) up to Week 12
|
|
Investigations
Aspartate aminotransferase increased
|
10.3%
8/78 • Baseline (Day 1) up to Week 12
|
0.00%
0/38 • Baseline (Day 1) up to Week 12
|
|
Gastrointestinal disorders
Diarrhea
|
19.2%
15/78 • Baseline (Day 1) up to Week 12
|
10.5%
4/38 • Baseline (Day 1) up to Week 12
|
|
Gastrointestinal disorders
Nausea
|
20.5%
16/78 • Baseline (Day 1) up to Week 12
|
5.3%
2/38 • Baseline (Day 1) up to Week 12
|
|
Gastrointestinal disorders
Abdominal Pain
|
6.4%
5/78 • Baseline (Day 1) up to Week 12
|
5.3%
2/38 • Baseline (Day 1) up to Week 12
|
|
Gastrointestinal disorders
Flatulence
|
6.4%
5/78 • Baseline (Day 1) up to Week 12
|
0.00%
0/38 • Baseline (Day 1) up to Week 12
|
|
Gastrointestinal disorders
Vomiting
|
5.1%
4/78 • Baseline (Day 1) up to Week 12
|
2.6%
1/38 • Baseline (Day 1) up to Week 12
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
5.1%
4/78 • Baseline (Day 1) up to Week 12
|
0.00%
0/38 • Baseline (Day 1) up to Week 12
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
6.4%
5/78 • Baseline (Day 1) up to Week 12
|
13.2%
5/38 • Baseline (Day 1) up to Week 12
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
2.6%
2/78 • Baseline (Day 1) up to Week 12
|
7.9%
3/38 • Baseline (Day 1) up to Week 12
|
|
Musculoskeletal and connective tissue disorders
Muscle Spasms
|
5.1%
4/78 • Baseline (Day 1) up to Week 12
|
0.00%
0/38 • Baseline (Day 1) up to Week 12
|
|
Investigations
Alanine aminotransferase increased
|
10.3%
8/78 • Baseline (Day 1) up to Week 12
|
0.00%
0/38 • Baseline (Day 1) up to Week 12
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release up to 45 days prior to submission and can embargo communications regarding study results for a period that is more than 90 days from the date that the communication is submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot unilaterally extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER