| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03067441 | Assessment of the Long-Term Safety and Efficacy of Bempedoic Acid (CLEAR Harmony OLE) | PHASE3 | COMPLETED | 1,462 | — | — | Feb 3, 2017 | Nov 5, 2019 | Mar 1, 2021 | 3 | United States |
| NCT03001076 | Evaluation of the Efficacy and Safety of Bempedoic Acid (ETC-1002) as Add-on to Ezetimibe Therapy in Patients With Elevated LDL-C (CLEAR Tranquility) | PHASE3 | COMPLETED | 269 | — | — | Nov 29, 2016 | Feb 12, 2018 | May 11, 2020 | 1 | United States |
| NCT02991118 | Evaluation of Long-Term Efficacy of Bempedoic Acid (ETC-1002) in Patients With Hyperlipidemia at High Cardiovascular Risk | PHASE3 | COMPLETED | 779 | — | — | Nov 18, 2016 | Aug 22, 2018 | Apr 27, 2020 | 2 | United States |
| NCT02988115 | Evaluation of the Efficacy and Safety of Bempedoic Acid (ETC-1002) in Patients With Hyperlipidemia and Statin Intolerant | PHASE3 | COMPLETED | 345 | — | — | Nov 16, 2016 | Mar 16, 2018 | Apr 3, 2020 | 67 | United States, Canada |
| NCT05694260 | A Clinical Study in Children With Heterozygous Familial Hypercholesterolemia (HeFH) Aged 6 to 17 Treated Once Daily With Bempedoic Acid Oral Dosing (CLEAR Path 1) | PHASE2 | COMPLETED | 31 | — | — | Jan 12, 2023 | Jun 4, 2025 | Aug 8, 2025 | 24 | United States, Canada +4 |
| NCT03193047 | Evaluation of the Efficacy and Safety of Bempedoic Acid (ETC-1002) 180mg When Added to PCSK9 Inhibitor Therapy | PHASE2 | COMPLETED | 59 | — | — | Apr 7, 2017 | Feb 19, 2018 | Apr 3, 2020 | 1 | United States |
| NCT03051100 | Evaluation of the Efficacy and Safety of Bempedoic Acid (ETC-1002) 180mg, Ezetimibe 10mg, and Atorvastatin 20 mg Triplet Therapy in Patients With Elevated LDL-C | PHASE2 | COMPLETED | 63 | — | — | Jan 19, 2017 | Jul 5, 2017 | Apr 3, 2020 | 11 | United States |
TEAEs are defined as adverse events that began or worsened in severity after the first dose of investigational medicinal product (IMP) until 30 days after the last dose in the Open-Label Extension (OLE) Study.
Blood samples were drawn after a minimum 10-hour fast (water was allowed) at pre-specified intervals. Samples were collected and analyzed for LDL-C. Baseline was defined as the mean of the LDL-C values from the last two non-missing values on or prior to Day 1. Percent change from baseline was calculated as: (\[LDL-C value at Week 12 minus Baseline value\] divided by \[Baseline Value\]) multiplied by 100. Bempedoic Acid = BA. Percent change from Baseline in LDL-C was analyzed using an analysis of covariance (ANCOVA) model with percent change from Baseline as the dependent variable, treatment as a fixed effects and Baseline as a covariate. In the ANCOVA model, missing LDL-C data at Week 12 were imputed using multiple imputation method taking into account adherence to treatment.
PCFB was calculated as the (\[post-Baseline (BL) value minus the BL value\] divided by the BL value ) x 100. BL was defined as the mean of the last two non-missing values on or prior to Day 1. If only one value was available, that single value was used as BL. PCFB in LDL-C was analyzed using analysis of covariance (ANCOVA), with treatment group and stratification factor (primary prevention; secondary prevention) as fixed effects and BL as a covariate. For participants with missing lipid data at Week 12 who were no longer taking study treatment, missing values were imputed using multiple imputation via a regression-based model including stratification and BL data from placebo participants only. In this imputation model, treatment group was not included. For participants with missing lipid data at Week 12 who were still taking study treatment, missing values were imputed using multiple imputation via a regression-based model including treatment, stratification and BL value.
Percent change from Baseline is calculated as the (\[post-Baseline value minus the Baseline value\] divided by the Baseline value ) x 100. Baseline is defined as the average of the last two non-missing values within Month -1 (Screening Visit 4) and Day 1 (Treatment Visit 1) values (including unscheduled assessments). If only one value was available, then that single value was used at Baseline. Percent change from Baseline was analyzed using analysis of covariance (ANCOVA), with treatment group as a factor and Baseline as a covariate. Missing data were imputed using last observation carried forward (LOCF) (only post-Baseline values were carried forward).
Percent change from Baseline is calculated as the (\[post-Baseline value minus the Baseline value\] divided by the Baseline value ) x 100. Baseline is defined as the mean of the values from Week -1 (Screening Visit 2) and predose Day 1/Week 0 (Treatment Visit 1). Percent change from Baseline in LDL-C was analyzed using analysis of covariance (ANCOVA) with treatment group as a factor and Baseline value as a covariate. Missing LDL-C values were imputed using last observation carried forward (LOCF), with only post-Baseline values carried forward. If LDL-C was measured (i.e., if TG was \>400 mg/dL or LDL-C was \<50 mg/dL), the measured values were used in the analysis.
| Arm | Type | Description |
|---|---|---|
| Open-Label bempedoic acid | EXPERIMENTAL | bempedoic acid 180 mg tablet |
| bempedoic acid | EXPERIMENTAL | bempedoic acid 180 mg tablet taken orally, daily. Patients remain on ongoing ezetimibe therapy (study provided) |
| placebo | PLACEBO_COMPARATOR | Matching placebo tablet taken orally, daily. Patients remain on ongoing ezetimibe therapy (study provided) |
| Cohort 1 | EXPERIMENTAL | Participants at 16 to \<30 kilograms (kg) body weight at screening receiving once daily 60 milligrams (mg) bempedoic acid for 8 weeks followed by 90 mg bempedoic acid for 8 weeks. |
| Cohort 2 | EXPERIMENTAL | Participants at 30 to 60 kg body weight at screening receiving once daily120 mg bempedoic acid for 8 weeks followed by 150 mg bempedoic acid for 8 weeks. |
| Cohort 3 | EXPERIMENTAL | Participants at greater than 60 kg body weight at screening receiving once daily 180 mg bempedoic acid for 8 weeks. |
| Triplet Therapy | EXPERIMENTAL | Bempedoic acid 180 mg, ezetimibe 10 mg, and atorvastatin 20 mg taken orally, daily. |
| Name | Type | Description |
|---|---|---|
| bempedoic acid | DRUG | bempedoic acid 180 mg tablets taken orally, once per day. |
| Ezetimibe | DRUG | ezetimibe 10 mg tablet |
| Placebo | OTHER | matching placebo tablet |
| bempedoic acid 180mg | DRUG | Daily bempedoic acid 180mg tablet in addition to monthly PCSK9i (evolocumab) background therapy |
| evolocumab | DRUG | Monthly PCSK9i (evolocumab) background therapy |
| Ezetimibe 10mg | DRUG | ezetimibe 10 mg |
| Atorvastatin 20mg | DRUG | atorvastatin 20 mg |
Inclusion Criteria: * Successfully completed CLEAR Harmony (1002-040) parent study Exclusion Criteria: * Experienced a treatment-related SAE that led to study drug discontinuation in the CLEAR Harmony (1002-040) parent study. * Medical condition requires lipid measurement and/or adjustment of bac...