Trial Outcomes & Findings for A Long-term Trial of ETC-1002 in Patients With Hyper-LDL Cholesterolemia (NCT NCT05687071)
NCT ID: NCT05687071
Last Updated: 2026-02-20
Results Overview
COMPLETED
PHASE3
130 participants
From baseline to week 52
2026-02-20
Participant Flow
Participant milestones
| Measure |
ETC-1002 180 mg
ETC-1002 180 mg tablet once daily for 52 weeks.
|
|---|---|
|
Overall Study
STARTED
|
130
|
|
Overall Study
COMPLETED
|
122
|
|
Overall Study
NOT COMPLETED
|
8
|
Reasons for withdrawal
| Measure |
ETC-1002 180 mg
ETC-1002 180 mg tablet once daily for 52 weeks.
|
|---|---|
|
Overall Study
Adverse Event
|
6
|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Protocol Violation
|
1
|
Baseline Characteristics
A Long-term Trial of ETC-1002 in Patients With Hyper-LDL Cholesterolemia
Baseline characteristics by cohort
| Measure |
ETC-1002 180 mg
n=130 Participants
ETC-1002 180 mg tablet once daily for 52 weeks.
|
|---|---|
|
Age, Continuous
|
62.0 years
STANDARD_DEVIATION 11.72 • n=14 Participants
|
|
Age, Customized
<65 years
|
72 Participants
n=14 Participants
|
|
Age, Customized
>=65 and <75 years
|
41 Participants
n=14 Participants
|
|
Age, Customized
>=75 years
|
17 Participants
n=14 Participants
|
|
Sex: Female, Male
Female
|
56 Participants
n=14 Participants
|
|
Sex: Female, Male
Male
|
74 Participants
n=14 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=14 Participants
|
|
Race (NIH/OMB)
Asian
|
130 Participants
n=14 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=14 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=14 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=14 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=14 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=14 Participants
|
|
Region of Enrollment
Japan
|
130 Participants
n=14 Participants
|
PRIMARY outcome
Timeframe: From baseline to week 52Population: Safety Analysis Set consisted of subjects who received at least one dose of the IMP during the treatment period.
Outcome measures
| Measure |
ETC-1002 180 mg
n=130 Participants
ETC-1002 180 mg tablet once daily for 52 weeks.
|
|---|---|
|
Number of Subjects Experiencing Treatment-Emergent Adverse Events (TEAEs)
|
109 Participants
|
PRIMARY outcome
Timeframe: Baseline, week52Population: Efficacy Analysis Set consisted of subjects who received at least one dose of the IMP during the treatment period, and who have LDL-C measurements at baseline and at one or more time points after administration of the IMP during the treatment period. Efficacy sample consisted of all participants with both baseline and observation at each visit of the given parameter.
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=122 Participants
ETC-1002 180 mg tablet once daily for 52 weeks.
|
|---|---|
|
Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 52
|
-21.55 Percent Change
Standard Deviation 18.727
|
SECONDARY outcome
Timeframe: Baseline, week52Population: Efficacy Analysis Set consisted of subjects who received at least one dose of the IMP during the treatment period, and who have LDL-C measurements at baseline and at one or more time points after administration of the IMP during the treatment period. Efficacy sample consisted of all participants with both baseline and observation at each visit of the given parameter.
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=122 Participants
ETC-1002 180 mg tablet once daily for 52 weeks.
|
|---|---|
|
Percent Change in Non-HDL Cholesterol From Baseline to Week 52
|
-19.36 Percent change
Standard Deviation 15.661
|
SECONDARY outcome
Timeframe: Baseline, week52Population: Efficacy Analysis Set consisted of subjects who received at least one dose of the IMP during the treatment period, and who have LDL-C measurements at baseline and at one or more time points after administration of the IMP during the treatment period. Efficacy sample consisted of all participants with both baseline and observation at each visit of the given parameter.
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=122 Participants
ETC-1002 180 mg tablet once daily for 52 weeks.
|
|---|---|
|
Percent Change in Total Cholesterol From Baseline to Week 52
|
-16.10 Percent change
Standard Deviation 12.761
|
SECONDARY outcome
Timeframe: Baseline, week52Population: Efficacy Analysis Set consisted of subjects who received at least one dose of the IMP during the treatment period, and who have LDL-C measurements at baseline and at one or more time points after administration of the IMP during the treatment period. Efficacy sample consisted of all participants with both baseline and observation at each visit of the given parameter.
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=122 Participants
ETC-1002 180 mg tablet once daily for 52 weeks.
|
|---|---|
|
Percent Change in Apolipoprotein B From Baseline to Week 52
|
-17.00 Percent change
Standard Deviation 13.503
|
SECONDARY outcome
Timeframe: Baseline, week52Population: Efficacy Analysis Set consisted of subjects who received at least one dose of the IMP during the treatment period, and who have LDL-C measurements at baseline and at one or more time points after administration of the IMP during the treatment period. Efficacy sample consisted of all participants with both baseline and observation at each visit of the given parameter.
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=122 Participants
ETC-1002 180 mg tablet once daily for 52 weeks.
|
|---|---|
|
Percent Change in High Sensitivity C Reactive Protein From Baseline to Week 52
|
-14.39 Percent change
Standard Deviation 100.709
|
SECONDARY outcome
Timeframe: Baseline, week52Population: Efficacy Analysis Set consisted of subjects who received at least one dose of the IMP during the treatment period, and who have LDL-C measurements at baseline and at one or more time points after administration of the IMP during the treatment period. Efficacy sample consisted of all participants with both baseline and observation at each visit of the given parameter.
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=122 Participants
ETC-1002 180 mg tablet once daily for 52 weeks.
|
|---|---|
|
Percent Change in Hemoglobin A1c From Baseline to Week 52
|
0.24 Percent change
Standard Deviation 5.137
|
SECONDARY outcome
Timeframe: Baseline, week52Population: Efficacy Analysis Set consisted of subjects who received at least one dose of the IMP during the treatment period, and who have LDL-C measurements at baseline and at one or more time points after administration of the IMP during the treatment period. Efficacy sample consisted of all participants with both baseline and observation at each visit of the given parameter.
The proportion of subjects whose LDL-C value achieves the lipid management goal at Week 52.
Outcome measures
| Measure |
ETC-1002 180 mg
n=122 Participants
ETC-1002 180 mg tablet once daily for 52 weeks.
|
|---|---|
|
Proportion of Subjects Whose LDL-C Value Achieved the Lipid Management Goals Based on Risk Assessment at Week 52
|
80 Participants
|
Adverse Events
ETC-1002 180 mg
Serious adverse events
| Measure |
ETC-1002 180 mg
n=130 participants at risk
ETC-1002 180 mg tablet once daily for 52 weeks.
|
|---|---|
|
Ear and labyrinth disorders
Sudden hearing loss
|
0.77%
1/130 • 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.
The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.
|
|
Eye disorders
Cataract
|
0.77%
1/130 • 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.
The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.
|
|
Eye disorders
Eyelid ptosis
|
0.77%
1/130 • 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.
The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.
|
|
Hepatobiliary disorders
Cholangitis
|
0.77%
1/130 • 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.
The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.
|
|
Musculoskeletal and connective tissue disorders
Lumbar spinal stenosis
|
0.77%
1/130 • 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.
The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
0.77%
1/130 • 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.
The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Cartilage neoplasm
|
0.77%
1/130 • 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.
The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.
|
|
Reproductive system and breast disorders
Prostatitis
|
0.77%
1/130 • 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.
The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
0.77%
1/130 • 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.
The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.
|
Other adverse events
| Measure |
ETC-1002 180 mg
n=130 participants at risk
ETC-1002 180 mg tablet once daily for 52 weeks.
|
|---|---|
|
Infections and infestations
Nasopharyngitis
|
22.3%
29/130 • 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.
The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.
|
|
Infections and infestations
Pharyngitis
|
5.4%
7/130 • 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.
The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.
|
|
Infections and infestations
COVID-19
|
12.3%
16/130 • 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.
The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.
|
|
Investigations
Blood uric acid increased
|
5.4%
7/130 • 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.
The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.
|
|
Metabolism and nutrition disorders
Hyperuricaemia
|
6.9%
9/130 • 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.
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
|
6.9%
9/130 • 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.
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
|
8.5%
11/130 • 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.
The safety analysis set will include subjects who receive at least one dose of IMP during the treatment period.
|
|
Vascular disorders
Hypertension
|
5.4%
7/130 • 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.
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.
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place