E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cardiovascular Diseases [C14] |
MedDRA Classification |
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 | 10051614 |
E.1.2 | Term | Arteriosclerotic cardiovascular disease |
E.1.2 | System Organ Class | 10047065 - Vascular disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10061653 |
E.1.2 | Term | Apheresis |
E.1.2 | System Organ Class | 10042613 - Surgical and medical procedures |
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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 |
•Demonstrate superiority of pelacarsen (TQJ230) compared to placebo in reducing the rate of lipoprotein apheresis sessions in patients with hyperlipoproteinemia(a) and established CVD during 52 weeks of treatment |
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E.2.2 | Secondary objectives of the trial |
•Demonstrate superiority of pelacarsen (TQJ230) compared to placebo in reducing the time to lipoprotein apheresis avoidance •Demonstrate superiority of pelacarsen (TQJ230) vs placebo in avoiding the performance of any lipoprotein apheresis in the last 40 weeks of the study •Demonstrate superiority of pelacarsen (TQJ230) vs placebo in lowering Lp(a) after 52 weeks
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
•Patients currently undergoing lipoprotein apheresis for isolated Lp(a) on a weekly schedule in Germany for ≥ 12 months prior to screening with at least 35 sessions within the past 52 weeks prior to randomization •Lipoprotein(a) (Lp(a))> 60 mg/dL at screening •Spontaneous prior myocardial infarction (MI): ≥ 3 months prior to the screening visit, and/or •Ischemic stroke: ≥ 3 months prior to the screening visit, and/or •Clinically significant symptomatic peripheral artery disease (PAD) •Clinically significant symptomatic coronary artery disease (CAD)
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E.4 | Principal exclusion criteria |
•Uncontrolled hypertension •Heart failure New York Heart Association (NYHA) class IV •History of malignancy of any organ system •History of hemorrhagic stroke or other major bleeding •Platelet count <140,000 per mm3 at screening •Active liver disease or hepatic dysfunction •Significant kidney disease •Pregnant or nursing women |
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E.5 End points |
E.5.1 | Primary end point(s) |
•Rate of lipoprotein apheresis sessions performed over 52 weeks normalized to the weekly lipoprotein apheresis schedule |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
•Time to lipoprotein apheresis avoidance (where lipoprotein apheresis avoidance is defined as at least 24 weeks of no lipoprotein apheresis until end of study) •Total avoidance of lipoprotein apheresis from week 12 to week 52 •Change from baseline to week 52 in the log-transformed Lp(a) (measured prior to planned lipoprotein apheresis) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
52 weeks for all endpoints |
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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 | Yes |
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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 13 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
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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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 | 5 |
E.8.9.1 | In the Member State concerned days | 10 |