Trial Outcomes & Findings for Pediatric Study to Evaluate the Efficacy and Safety of Ezetimibe Monotherapy in Children With Primary Hypercholesterolemia (P05522) (NCT NCT00867165)
NCT ID: NCT00867165
Last Updated: 2024-05-21
Results Overview
Serum LDL-C levels calculated at baseline and after 12 weeks of study drug administration. LDL-C were calculated by the method of Friedewald equation, LDL-C = Total Cholesterol (TC) - (High-density lipoprotein cholesterol \[HDL-C\] + triglyceride \[TG\]/5).
COMPLETED
PHASE3
138 participants
Baseline and Week 12
2024-05-21
Participant Flow
Participant milestones
| Measure |
Ezetimibe
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Overall Study
STARTED
|
93
|
45
|
|
Overall Study
COMPLETED
|
89
|
45
|
|
Overall Study
NOT COMPLETED
|
4
|
0
|
Reasons for withdrawal
| Measure |
Ezetimibe
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Overall Study
Adverse Event
|
3
|
0
|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
Baseline Characteristics
Pediatric Study to Evaluate the Efficacy and Safety of Ezetimibe Monotherapy in Children With Primary Hypercholesterolemia (P05522)
Baseline characteristics by cohort
| Measure |
Ezetimibe
n=93 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=45 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
Total
n=138 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
8.2 years
STANDARD_DEVIATION 1.7 • n=99 Participants
|
8.5 years
STANDARD_DEVIATION 1.6 • n=107 Participants
|
8.3 years
STANDARD_DEVIATION 1.6 • n=206 Participants
|
|
Sex: Female, Male
Female
|
52 Participants
n=99 Participants
|
27 Participants
n=107 Participants
|
79 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
41 Participants
n=99 Participants
|
18 Participants
n=107 Participants
|
59 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Baseline and Week 12Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum LDL-C levels calculated at baseline and after 12 weeks of study drug administration. LDL-C were calculated by the method of Friedewald equation, LDL-C = Total Cholesterol (TC) - (High-density lipoprotein cholesterol \[HDL-C\] + triglyceride \[TG\]/5).
Outcome measures
| Measure |
Ezetimibe
n=85 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=42 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Week 12
|
-27.70 Percent Change
Interval -30.8 to -24.59
|
-0.95 Percent Change
Interval -4.94 to 3.04
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum TC levels measured using enzymatic methods at baseline and after 12 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=85 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=42 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Total Cholesterol (TC) at Week 12
|
-20.74 Percentage Change
Interval -23.29 to -18.18
|
0.19 Percentage Change
Interval -3.07 to 3.44
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum Apo B measured at baseline and after 12 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=85 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=42 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Apolipoprotein B (Apo B) at Week 12
|
-21.66 Percent Change
Interval -24.94 to -18.38
|
-1.42 Percent Change
Interval -5.55 to 2.7
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum HDL-C levels measured by photometry after precipitation at baseline and after 12 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=85 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=42 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline High-density Lipoprotein Cholesterol (HDL-C) at Week 12
|
2.11 Percentage Change
Interval -2.27 to 6.48
|
1.41 Percentage Change
Interval -4.14 to 6.96
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum Non-HDL-C calculated at baseline and after 12 weeks of study drug administration. Non-HDL-C values were calculated as follows: Non-HDL-C (mg/dL) = TC - HDL-C.
Outcome measures
| Measure |
Ezetimibe
n=85 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=42 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Non-HDL-C at Week 12
|
-25.47 Percentage Change
Interval -28.45 to -22.49
|
0.28 Percentage Change
Interval -3.52 to 4.08
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum TG levels measured using enzymatic methods at baseline and after 12 weeks of study drug.
Outcome measures
| Measure |
Ezetimibe
n=85 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=42 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Triglycerides (TG) at Week 12
|
-6.23 Percentage change
Interval -12.73 to 0.75
|
8.44 Percentage change
Interval -2.03 to 20.03
|
SECONDARY outcome
Timeframe: Baseline and Week 2Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum LDL-C levels calculated at baseline and after 2 weeks of study drug administration. LDL-C were calculated by the method of Friedewald equation, LDL-C = Total Cholesterol (TC) - (High-density lipoprotein cholesterol \[HDL-C\] + triglyceride \[TG\]/5).
Outcome measures
| Measure |
Ezetimibe
n=91 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percent Change From Baseline in LDL-C at Week 2
|
-24.75 Percentage Change
Interval -27.8 to -21.69
|
0.23 Percentage Change
Interval -3.74 to 4.2
|
SECONDARY outcome
Timeframe: Baseline and Week 4Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum LDL-C levels calculated at baseline and after 4 weeks of study drug administration. LDL-C were calculated by the method of Friedewald equation, LDL-C = Total Cholesterol (TC) - (High-density lipoprotein cholesterol \[HDL-C\] + triglyceride \[TG\]/5).
Outcome measures
| Measure |
Ezetimibe
n=90 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percent Change From Baseline in LDL-C at Week 4
|
-26.93 Percentage Change
Interval -29.81 to -24.05
|
-2.99 Percentage Change
Interval -6.66 to 0.68
|
SECONDARY outcome
Timeframe: Baseline and Week 8Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum LDL-C levels calculated at baseline and after 8 weeks of study drug administration. LDL-C were calculated by the method of Friedewald equation, LDL-C = Total Cholesterol (TC) - (High-density lipoprotein cholesterol \[HDL-C\] + triglyceride \[TG\]/5).
Outcome measures
| Measure |
Ezetimibe
n=90 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=44 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percent Change From Baseline in LDL-C at Week 8
|
-27.22 Percentage Change
Interval -30.3 to -24.15
|
0.49 Percentage Change
Interval -3.46 to 4.43
|
SECONDARY outcome
Timeframe: Baseline and Week 2Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum TC levels measured using enzymatic methods at baseline and after 2 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=91 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in TC at Week 2
|
-18.69 Percentage Change
Interval -21.21 to -16.18
|
1.40 Percentage Change
Interval -1.83 to 4.63
|
SECONDARY outcome
Timeframe: Baseline and Week 4Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum TC levels measured using enzymatic methods at baseline and after 4 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=90 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in TC at Week 4
|
-20.57 Percentage Change
Interval -23.05 to -18.08
|
-0.29 Percentage Change
Interval -3.45 to 2.86
|
SECONDARY outcome
Timeframe: Baseline and Week 8Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum TC levels measured using enzymatic methods at baseline and after 8 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=90 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=44 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in TC at Week 8
|
-20.84 Percentage Change
Interval -23.4 to -18.27
|
0.54 Percentage Change
Interval -2.73 to 3.81
|
SECONDARY outcome
Timeframe: Baseline and Week 2Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum HDL-C levels measured by photometry after precipitation at baseline and after 2 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=91 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline HDL-C at Week 2
|
0.01 Percentage Change
Interval -4.28 to 4.3
|
0.96 Percentage Change
Interval -4.51 to 6.43
|
SECONDARY outcome
Timeframe: Baseline and Week 4Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum HDL-C levels measured by photometry after precipitation at baseline and after 4 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=90 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline HDL-C at Week 4
|
0.86 Percentage Change
Interval -3.69 to 5.42
|
0.33 Percentage Change
Interval -5.54 to 6.19
|
SECONDARY outcome
Timeframe: Baseline and Week 8Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum HDL-C levels measured by photometry after precipitation at baseline and after 8 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=90 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=44 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline HDL-C at Week 8
|
1.41 Percentage Change
Interval -2.95 to 5.77
|
-0.52 Percentage Change
Interval -6.04 to 5.0
|
SECONDARY outcome
Timeframe: Baseline and Week 2Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum Non-HDL-C calculated at baseline and after 2 weeks of study drug administration. Non-HDL-C values were calculated as follows: Non-HDL-C (mg/dL) = TC - HDL-C.
Outcome measures
| Measure |
Ezetimibe
n=91 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Non-HDL-C at Week 2
|
-22.54 Percentage Change
Interval -25.41 to -19.67
|
1.64 Percentage Change
Interval -2.03 to 5.3
|
SECONDARY outcome
Timeframe: Baseline and Week 4Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum Non-HDL-C calculated at baseline and after 4 weeks of study drug administration. Non-HDL-C values were calculated as follows: Non-HDL-C (mg/dL) = TC - HDL-C.
Outcome measures
| Measure |
Ezetimibe
n=90 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Non-HDL-C at Week 4
|
-24.94 Percentage Change
Interval -27.75 to -22.14
|
-0.33 Percentage Change
Interval -3.88 to 3.22
|
SECONDARY outcome
Timeframe: Baseline and Week 8Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum Non-HDL-C calculated at baseline and after 8 weeks of study drug administration. Non-HDL-C values were calculated as follows: Non-HDL-C (mg/dL) = TC - HDL-C.
Outcome measures
| Measure |
Ezetimibe
n=90 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=44 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Non-HDL-C at Week 8
|
-25.41 Percentage Change
Interval -28.37 to -22.45
|
1.14 Percentage Change
Interval -2.63 to 4.92
|
SECONDARY outcome
Timeframe: Baseline and Week 2Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum TG levels measured using enzymatic methods at baseline and after 2 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=91 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in TG at Week 2
|
-3.90 Percentage Change
Interval -10.66 to 3.37
|
5.83 Percentage Change
Interval -4.76 to 17.59
|
SECONDARY outcome
Timeframe: Baseline and Week 4Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum TG levels measured using enzymatic methods at baseline and after 4 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=90 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in TG at Week 4
|
-5.54 Percentage Change
Interval -11.05 to 0.31
|
9.65 Percentage Change
Interval 0.6 to 19.53
|
SECONDARY outcome
Timeframe: Baseline and Week 8Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum TG levels measured using enzymatic methods at baseline and after 8 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=90 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=44 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in TG at Week 8
|
-9.99 Percentage Change
Interval -16.28 to -3.21
|
-1.38 Percentage Change
Interval -11.02 to 9.31
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum Apo A-I levels measured at baseline and after 12 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=85 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=42 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Apolipoprotein A-I (Apo A-I) at Week 12
|
1.80 Percentage Change
Interval -1.86 to 5.46
|
3.71 Percentage Change
Interval -0.87 to 8.3
|
SECONDARY outcome
Timeframe: Baseline and Week 2Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum TC:HDL-C Ratio calculated at baseline and after 2 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=91 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in TC:HDL-C Ratio at Week 2
|
-17.21 Percentage Change
Interval -21.08 to -13.34
|
2.56 Percentage Change
Interval -2.37 to 7.48
|
SECONDARY outcome
Timeframe: Baseline and Week 4Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum TC:HDL-C Ratio calculated at baseline and after 4 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=90 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in TC:HDL-C Ratio at Week 4
|
-19.36 Percentage Change
Interval -23.16 to -15.56
|
1.48 Percentage Change
Interval -3.3 to 6.25
|
SECONDARY outcome
Timeframe: Baseline and Week 8Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum TC:HDL-C Ratio calculated at baseline and after 8 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=90 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=44 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in TC:HDL-C Ratio at Week 8
|
-20.07 Percentage Change
Interval -24.2 to -15.94
|
3.41 Percentage Change
Interval -1.87 to 8.7
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum TC:HDL-C Ratio calculated at baseline and after 12 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=85 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=42 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in TC:HDL-C Ratio at Week 12
|
-21.09 Percentage Change
Interval -24.98 to -17.19
|
1.20 Percentage Change
Interval -3.7 to 6.11
|
SECONDARY outcome
Timeframe: Baseline and Week 2Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum LDL-C:HDL-C Ratio calculated at baseline and after 2 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=91 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in LDL-C:HDL-C Ratio at Week 2
|
-23.35 Percentage Change
Interval -27.69 to -19.01
|
1.15 Percentage Change
Interval -4.35 to 6.65
|
SECONDARY outcome
Timeframe: Baseline and Week 4Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum LDL-C:HDL-C Ratio calculated at baseline and after 4 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=90 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=42 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in LDL-C:HDL-C Ratio at Week 4
|
-25.72 Percentage Change
Interval -30.12 to -21.33
|
-0.57 Percentage Change
Interval -6.14 to 5.0
|
SECONDARY outcome
Timeframe: Baseline and Week 8Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum LDL-C:HDL-C Ratio calculated at baseline and after 8 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=90 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in LDL-C:HDL-C Ratio at Week 8
|
-26.43 Percentage Change
Interval -31.21 to -21.66
|
2.87 Percentage Change
Interval -3.28 to 9.02
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum LDL-C:HDL-C Ratio calculated at baseline and after 12 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=85 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=42 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in LDL-C:HDL-C Ratio at Week 12
|
-28.01 Percentage Change
Interval -32.65 to -23.36
|
0.34 Percentage Change
Interval -5.57 to 6.25
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Serum Apo B:Apo A-I Ratio calculated at baseline and after 12 weeks of study drug administration
Outcome measures
| Measure |
Ezetimibe
n=85 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=42 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Apo B:Apo A-I Ratio at Week 12
|
-22.16 Percentage Change
Interval -26.13 to -18.19
|
-2.87 Percentage Change
Interval -7.84 to 2.09
|
SECONDARY outcome
Timeframe: Baseline and Week 4Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma hs-CRP measured at baseline and after 4 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=90 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in High-sensitivity C-reactive Protein (Hs-CRP) at Week 4
|
10.00 Percentage Change
Interval -15.65 to 43.45
|
56.11 Percentage Change
Interval 7.68 to 126.32
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma hs-CRP measured at baseline and after 12 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=85 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=42 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in High-sensitivity C-reactive Protein (Hs-CRP) at Week 12
|
9.35 Percentage Change
Interval -13.47 to 38.19
|
-7.92 Percentage Change
Interval -33.48 to 27.46
|
SECONDARY outcome
Timeframe: Baseline and Week 2Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma sitosterol measured at baseline and after 2 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=89 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percent Change From Baseline in Sitosterol at Week 2
|
-50.20 Percentage Change
Interval -54.65 to -45.76
|
-0.59 Percentage Change
Interval -6.26 to 5.08
|
SECONDARY outcome
Timeframe: Baseline and Week 4Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma sitosterol measured at baseline and after 4 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=87 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Sitosterol at Week 4
|
-58.82 Percentage Change
Interval -63.04 to -54.61
|
-0.30 Percentage Change
Interval -5.52 to 4.93
|
SECONDARY outcome
Timeframe: Baseline and Week 8Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma sitosterol measured at baseline and after 8 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=86 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=44 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Sitosterol at Week 8
|
-61.59 Percentage Change
Interval -66.39 to -56.78
|
-0.57 Percentage Change
Interval -6.69 to 5.56
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma sitosterol measured at baseline and after 12 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=82 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=41 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Sitosterol at Week 12
|
-60.65 Percentage Change
Interval -67.39 to -53.92
|
2.09 Percentage Change
Interval -7.01 to 11.18
|
SECONDARY outcome
Timeframe: Baseline and Week 2Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma campesterol measured at baseline and after 2 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=89 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Campesterol at Week 2
|
-53.87 Percentage Change
Interval -57.99 to -49.74
|
-4.38 Percentage Change
Interval -9.66 to 0.9
|
SECONDARY outcome
Timeframe: Baseline and Week 4Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma campesterol measured at baseline and after 4 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=87 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Campesterol at Week 4
|
-62.27 Percentage Change
Interval -66.21 to -58.32
|
-4.28 Percentage Change
Interval -9.2 to 0.65
|
SECONDARY outcome
Timeframe: Baseline and Week 8Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma campesterol measured at baseline and after 8 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=86 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=44 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Campesterol at Week 8
|
-66.79 Percentage Change
Interval -71.21 to -62.37
|
-4.19 Percentage Change
Interval -9.83 to 1.45
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma campesterol measured at baseline and after 12 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=82 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=41 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Campesterol at Week 12
|
-67.59 Percentage Change
Interval -73.68 to -61.5
|
-2.92 Percentage Change
Interval -11.15 to 5.31
|
SECONDARY outcome
Timeframe: Baseline and Week 2Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma cholestanol measured at baseline and after 2 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=89 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Cholestanol at Week 2
|
-25.44 Percentage Change
Interval -30.3 to -20.59
|
-7.09 Percentage Change
Interval -13.29 to -0.9
|
SECONDARY outcome
Timeframe: Baseline and Week 4Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma cholestanol measured at baseline and after 4 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=87 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Cholestanol at Week 4
|
-27.40 Percentage Change
Interval -31.95 to -22.85
|
-1.79 Percentage Change
Interval -7.42 to 3.85
|
SECONDARY outcome
Timeframe: Baseline and Week 8Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma cholestanol measured at baseline and after 8 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=86 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=44 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Cholestanol at Week 8
|
-29.19 Percentage Change
Interval -33.98 to -24.39
|
-0.44 Percentage Change
Interval -6.44 to 5.56
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma cholestanol measured at baseline and after 12 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=82 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=41 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Cholestanol at Week 12
|
-28.71 Percentage Change
Interval -34.44 to -22.99
|
3.42 Percentage Change
Interval -4.07 to 10.92
|
SECONDARY outcome
Timeframe: Baseline and Week 2Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma lathosterol measured at baseline and after 2 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=89 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Lathosterol at Week 2
|
34.07 Percentage Change
Interval 24.68 to 43.45
|
14.06 Percentage Change
Interval 2.22 to 25.9
|
SECONDARY outcome
Timeframe: Baseline and Week 4Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma lathosterol measured at baseline and after 4 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=87 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=43 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Lathosterol at Week 4
|
33.02 Percentage Change
Interval 23.44 to 42.6
|
6.73 Percentage Change
Interval -5.27 to 18.73
|
SECONDARY outcome
Timeframe: Baseline and Week 8Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma lathosterol measured at baseline and after 8 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=86 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=44 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Lathosterol at Week 8
|
35.87 Percentage Change
Interval 26.14 to 45.61
|
-0.82 Percentage Change
Interval -12.99 to 11.34
|
SECONDARY outcome
Timeframe: Baseline and Week 12Population: Full Analysis Set (FAS) population defined as all randomized participants who took at least one dose of study medication and had a baseline value and at least one valid post-baseline evaluation.
Plasma lathosterol measured at baseline and after 12 weeks of study drug administration.
Outcome measures
| Measure |
Ezetimibe
n=82 Participants
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=41 Participants
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Percentage Change From Baseline in Lathosterol at Week 12
|
36.62 Percentage Change
Interval 26.46 to 46.78
|
12.57 Percentage Change
Interval -0.36 to 25.5
|
Adverse Events
Ezetimibe
Placebo
Serious adverse events
| Measure |
Ezetimibe
n=92 participants at risk
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=45 participants at risk
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Congenital, familial and genetic disorders
Epilepsy congenital
|
1.1%
1/92 • Number of events 3
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
0.00%
0/45
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
|
Infections and infestations
Appendicitis
|
1.1%
1/92 • Number of events 1
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
0.00%
0/45
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
Other adverse events
| Measure |
Ezetimibe
n=92 participants at risk
Ezetimibe 10-mg tablet once daily for 12 weeks
|
Placebo
n=45 participants at risk
Placebo to match ezetimibe 10-mg tablet once daily for 12 weeks
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
4.3%
4/92 • Number of events 4
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
11.1%
5/45 • Number of events 6
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
|
Gastrointestinal disorders
Diarrhoea
|
1.1%
1/92 • Number of events 2
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
8.9%
4/45 • Number of events 5
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
|
Gastrointestinal disorders
Nausea
|
1.1%
1/92 • Number of events 1
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
6.7%
3/45 • Number of events 3
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
|
Gastrointestinal disorders
Vomiting
|
2.2%
2/92 • Number of events 2
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
6.7%
3/45 • Number of events 3
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
|
Infections and infestations
Influenza
|
5.4%
5/92 • Number of events 5
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
6.7%
3/45 • Number of events 3
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
|
Infections and infestations
Nasopharyngitis
|
10.9%
10/92 • Number of events 10
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
11.1%
5/45 • Number of events 5
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
|
Infections and infestations
Upper respiratory tract infection
|
7.6%
7/92 • Number of events 7
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
2.2%
1/45 • Number of events 1
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
|
Nervous system disorders
Headache
|
4.3%
4/92 • Number of events 9
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
13.3%
6/45 • Number of events 6
Safety analysis was performed on the All-Patients-as-Treated (APaT) Population defined as all randomized patients who received at least one dose of double-blind study therapy.
|
Additional Information
Senior Vice President, Global Clinical Development
Merck Sharp & Dohme.
Results disclosure agreements
- Principal investigator is a sponsor employee The investigator agreed not to publish or publicly present any interim results of the trial without the prior written consent of the sponsor. The investigator further agreed to provide to the sponsor 45 days prior to submission for publication or presentation, review copies of abstracts or manuscripts for publication that report any results of the trial.
- Publication restrictions are in place
Restriction type: OTHER