Trial Outcomes & Findings for A Confirmatory Trial of ETC-1002 in Patients With Hyper-LDL Cholesterolemia (NCT NCT05683340)

NCT ID: NCT05683340

Last Updated: 2025-03-13

Results Overview

Percent change from Baseline was calculated as the (\[post-Baseline value minus the Baseline value\] divided by the Baseline value) x 100. Baseline was defined as the mean of the values from Week -1 and Day 1.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 96 participants
• Primary outcome timeframe: Baseline, week12
• Results posted on: 2025-03-13

Participant Flow

Participant milestones

Measure	ETC-1002 180 mg ETC-1002 180 mg tablet once daily for 12 weeks.	Placebo Placebo tablet once daily for 12 weeks.
Overall Study STARTED	48	48
Overall Study COMPLETED	46	45
Overall Study NOT COMPLETED	2	3

Reasons for withdrawal

Measure	ETC-1002 180 mg ETC-1002 180 mg tablet once daily for 12 weeks.	Placebo Placebo tablet once daily for 12 weeks.
Overall Study Adverse Event	1	1
Overall Study Protocol Violation	1	0
Overall Study Withdrawal by Subject	0	2

Baseline Characteristics

A Confirmatory Trial of ETC-1002 in Patients With Hyper-LDL Cholesterolemia

Baseline characteristics by cohort

Measure	ETC-1002 180 mg n=48 Participants ETC-1002 180 mg tablet once daily for 12 weeks.	Placebo n=48 Participants Placebo tablet once daily for 12 weeks.	Total n=96 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	20 Participants n=5 Participants	25 Participants n=7 Participants	45 Participants n=5 Participants
Age, Categorical >=65 years	28 Participants n=5 Participants	23 Participants n=7 Participants	51 Participants n=5 Participants
Age, Continuous	64.6 years STANDARD_DEVIATION 13.02 • n=5 Participants	63.9 years STANDARD_DEVIATION 12.98 • n=7 Participants	64.2 years STANDARD_DEVIATION 12.93 • n=5 Participants
Sex: Female, Male Female	28 Participants n=5 Participants	23 Participants n=7 Participants	51 Participants n=5 Participants
Sex: Female, Male Male	20 Participants n=5 Participants	25 Participants n=7 Participants	45 Participants n=5 Participants
Race/Ethnicity, Customized Japanese	48 Participants n=5 Participants	48 Participants n=7 Participants	96 Participants n=5 Participants
Region of Enrollment Japan	48 participants n=5 Participants	48 participants n=7 Participants	96 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, week12

Population: The full analysis set (FAS) will include all subjects who receive at least one dose of IMP during the treatment period and for whom LDL-C values at baseline and at least one post-dose are observed.

Percent change from Baseline was calculated as the ([post-Baseline value minus the Baseline value] divided by the Baseline value) x 100. Baseline was defined as the mean of the values from Week -1 and Day 1.

Outcome measures

Measure	ETC-1002 180 mg n=46 Participants ETC-1002 180 mg tablet once daily for 12 weeks.	Placebo n=45 Participants Placebo tablet once daily for 12 weeks.
Percent Change in LDL-C From Baseline to Week 12	-25.25 Percent Change Standard Error 1.864	-3.46 Percent Change Standard Error 1.901

SECONDARY outcome

Timeframe: Baseline, week12

Percent change from Baseline was calculated as the ([post-Baseline value minus the Baseline value] divided by the Baseline value) x 100.

Outcome measures

Measure	ETC-1002 180 mg n=46 Participants ETC-1002 180 mg tablet once daily for 12 weeks.	Placebo n=45 Participants Placebo tablet once daily for 12 weeks.
Percent Change in Non-HDL Cholesterol From Baseline to Week 12	-20.33 Percent Change Standard Error 1.704	-2.76 Percent Change Standard Error 1.720

SECONDARY outcome

Timeframe: Baseline, week12

Percent change from Baseline was calculated as the ([post-Baseline value minus the Baseline value] divided by the Baseline value) x 100.

Outcome measures

Measure	ETC-1002 180 mg n=46 Participants ETC-1002 180 mg tablet once daily for 12 weeks.	Placebo n=45 Participants Placebo tablet once daily for 12 weeks.
Percent Change in Total Cholesterol From Baseline to Week 12	-16.36 Percent Change Standard Error 1.405	-2.23 Percent Change Standard Error 1.409

SECONDARY outcome

Timeframe: Baseline, week12

Percent change from Baseline was calculated as the ([post-Baseline value minus the Baseline value] divided by the Baseline value) x 100.

Outcome measures

Measure	ETC-1002 180 mg n=46 Participants ETC-1002 180 mg tablet once daily for 12 weeks.	Placebo n=45 Participants Placebo tablet once daily for 12 weeks.
Percent Change in Apolipoprotein B From Baseline to Week 12	-18.10 Percent Change Standard Error 1.762	-0.67 Percent Change Standard Error 1.799

SECONDARY outcome

Timeframe: Baseline, week12

Percent change from Baseline was calculated as the ([post-Baseline value minus the Baseline value] divided by the Baseline value) x 100.

Outcome measures

Measure	ETC-1002 180 mg n=46 Participants ETC-1002 180 mg tablet once daily for 12 weeks.	Placebo n=45 Participants Placebo tablet once daily for 12 weeks.
Percent Change in High Sensitivity C Reactive Protein From Baseline to Week 12	15.40 Percent Change Standard Error 65.246	119.60 Percent Change Standard Error 66.542

SECONDARY outcome

Timeframe: Baseline, week12

Percent change from Baseline was calculated as the ([post-Baseline value minus the Baseline value] divided by the Baseline value) x 100.

Outcome measures

Measure	ETC-1002 180 mg n=46 Participants ETC-1002 180 mg tablet once daily for 12 weeks.	Placebo n=45 Participants Placebo tablet once daily for 12 weeks.
Percent Change in Hemoglobin A1c From Baseline to Week 12	-0.87 Percent Change Standard Error 0.590	0.16 Percent Change Standard Error 0.594

SECONDARY outcome

Timeframe: Baseline, week12

The proportion of subjects whose LDL-C value achieves the lipid management goal at Week 12.

Outcome measures

Measure	ETC-1002 180 mg n=48 Participants ETC-1002 180 mg tablet once daily for 12 weeks.	Placebo n=48 Participants Placebo tablet once daily for 12 weeks.
Proportion of Subjects Whose LDL-C Value Achieved the Lipid Management Goals Based on Risk Assessment at Week 12	30 Participants	4 Participants

Adverse Events

ETC-1002 180 mg

Serious events: 0 serious events

Other events: 13 other events

Deaths: 0 deaths

Placebo

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	ETC-1002 180 mg n=48 participants at risk ETC-1002 180 mg tablet once daily for 12 weeks.	Placebo n=48 participants at risk Placebo tablet once daily for 12 weeks.
Infections and infestations Nasopharyngitis	14.6% 7/48 • Adverse events were monitored from signing of the informed consent form until follow-up for up to 28 (+7) days after the last dose of study medication, up to 21 weeks The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.	10.4% 5/48 • Adverse events were monitored from signing of the informed consent form until follow-up for up to 28 (+7) days after the last dose of study medication, up to 21 weeks The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.
Musculoskeletal and connective tissue disorders Arthralgia	6.2% 3/48 • Adverse events were monitored from signing of the informed consent form until follow-up for up to 28 (+7) days after the last dose of study medication, up to 21 weeks The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.	0.00% 0/48 • Adverse events were monitored from signing of the informed consent form until follow-up for up to 28 (+7) days after the last dose of study medication, up to 21 weeks The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.
Musculoskeletal and connective tissue disorders Back pain	6.2% 3/48 • Adverse events were monitored from signing of the informed consent form until follow-up for up to 28 (+7) days after the last dose of study medication, up to 21 weeks The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.	2.1% 1/48 • Adverse events were monitored from signing of the informed consent form until follow-up for up to 28 (+7) days after the last dose of study medication, up to 21 weeks The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.

Additional Information

Director of Clinical Trials

Otsuka Pharmaceutical Co., Ltd.

Phone: +81363617366

Email: CL_OPCJ_RDA_Team@otsuka.jp

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place