E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Treatment of high cardiovascular risk patients (heterozygous familial hypercholesterolemia [HeFH] and atherosclerotic cardiovascular diseases [ASCVD]) with hyperlipidemia |
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E.1.1.1 | Medical condition in easily understood language |
Treatment of hyperlipidemia; a condition where there is an excess of lipids (fats e.g. cholesterol ) in the blood, in patients with a high risk of developing heart problems. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nutritional and Metabolic Diseases [C18] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10020604 |
E.1.2 | Term | Hypercholesterolemia |
E.1.2 | System Organ Class | 100000004861 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10020667 |
E.1.2 | Term | Hyperlipidemia |
E.1.2 | System Organ Class | 100000004861 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10051615 |
E.1.2 | Term | Atherosclerotic cardiovascular disease |
E.1.2 | System Organ Class | 100000004866 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10057079 |
E.1.2 | Term | Heterozygous familial hypercholesterolemia |
E.1.2 | System Organ Class | 100000004850 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To characterize the safety and tolerability of long-term administration of bempedoic acid (ETC-1002) 180 mg |
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E.2.2 | Secondary objectives of the trial |
To characterize the efficacy of long-term administration of bempedoic acid 180 mg/day as assessed by changes in LDL C, HDL C, non-high-density lipoprotein cholesterol (non-HDL C), ApoB, total cholesterol (TC), TG and high-sensitivity C reactive protein (hs CRP) in patients with hyperlipidemia |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Each patient must meet the following criteria to be enrolled in this study:
1. Successfully completed the parent study (1002-040) and meet both of the following criteria:
• The patient was compliant with the parent study requirements including study visits, procedures, and investigational medicinal product (IMP) in the opinion of the principal investigator.
• The patient was able to tolerate IMP through the end of the parent study.
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E.4 | Principal exclusion criteria |
1. Female patient is not willing to use at least 1 acceptable method of birth control during treatment and for an additional 30 days after the end of treatment unless patient is sterilized or postmenopausal;
a. Menopause is defined as greater than 55 years and ≥1 year without menses, less than 55 years and ≥1 year without menses with follicle-stimulating hormone (FSH) ≥40.0 IU/L, or surgically sterile (including hysterectomy and/or bilateral oophorectomy);
b. Acceptable methods of birth control include: oral birth control medications; placement of an intrauterine device (IUD) with or without hormones; barrier methods including condom or occlusive cap with spermicidal foam or spermicidal jelly; vasectomized male partner who is the sole partner for this patient; or true abstinence where it is the preferred and usual lifestyle of the patient (not including periodic abstinence such as calendar, ovulation, symptothermal, postovulation methods, or withdrawal).
2. Patient is pregnant or breastfeeding, or might become pregnant during treatment and/ or within 30 days after the end of treatment
3. Unreliability as a study participant based on the investigator’s (or designee’s) knowledge of the patient (eg, inability or unwillingness to adhere to the protocol)
4. Experienced a treatment-related SAE that led to study drug discontinuation in the parent study
5. Disorder that would interfere with understanding and giving informed consent or compliance with protocol requirements
6. Have any medical condition that in the opinion of the investigator may affect patient safety or ability to complete scheduled assessments
7. Patient’s medical condition requires lipid measurement and/or adjustment of background lipid-regulating therapy during the first 12 weeks of study participation
8. Known sensitivity to any of the products to be administered during dosing
9. Currently enrolled in another investigational device or drug study (excluding ETC-1002-040), or less than 30 days since ending another investigational device or drug study(s),or receiving or planning to receive other investigational agent(s) during this study. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary efficacy endpoint for this study is patient incidence of AEs |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
At all clinic visits, investigators will review all safety information including vital signs, AEs, and concomitant medications and will ensure that the collected data are recorded into the appropriate eCRF. Additionally, clinical laboratory samples will be collected and sent for analysis and the investigator will review the results to ensure continued patient safety while participating in the study. |
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E.5.2 | Secondary end point(s) |
• Percent change from baseline in LDL C at Weeks 52 and 78
• Change from baseline in LDL C at Weeks 52 and 78
• Percent change from baseline in non-HDL C at Weeks 52 and 78
• Percent change from baseline in TC at Weeks 52 and 78
• Percent change from baseline in ApoB at Weeks 52 and 78
• Percent change from baseline in hs CRP at Weeks 52 and 78
• Percent change from baseline in TG at Weeks 52 and 78
• Percent change from baseline in HDL C at Weeks 52 and 78
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Baseline and Weeks 52 and 78 |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 11 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 59 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Canada |
Germany |
Netherlands |
Poland |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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The study will end when the last patient completes their Week 82 visit. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 7 |
E.8.9.2 | In all countries concerned by the trial days | 0 |