A Multicenter Trial Assessing the Impact of Lipoprotein(a) Lowering With Pelacarsen (TQJ230) on the Rate of Weekly Lipoprotein Apheresis Sessions in Patients With Hyperlipoproteinemia(a) and Established Cardiovascular Disease in Germany

Study Overview
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Brief Summary
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Phase III study to test the hypothesis that treatment with pelacarsen (TQJ230) 80 mg Q4W compared to placebo significantly reduces the rate of lipoprotein apheresis in patients with hyperlipoproteinemia (a) and established cardiovascular disease currently undergoing lipoprotein apheresis in Germany on a weekly schedule.

Detailed Description
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Lipoprotein apheresis to date is the only approved therapeutic option for cardiovascular (CV) risk reduction in patients with severely elevated Lp(a) levels in Germany. Lipoprotein apheresis is an expensive, burdensome, and time-consuming procedure. The current study (CTQJ230A12302) will investigate if treatment with pelacarsen (TQJ230) 80 mg Q4W vs placebo reduces the rate of lipoprotein apheresis in patients with hyperlipoproteinemia(a) and established CV disease

Official Title
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A Randomized, Double-blind, Placebo-controlled, Multicenter Trial Assessing the Reduction of the Rate of Lipoprotein Apheresis After Treatment With Pelacarsen (TQJ230) Compared to Placebo in Patients With Hyperlipoproteinemia(a) and Established Cardiovascular Disease Undergoing Weekly Lipoprotein Apheresis in Germany

Conditions
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Hyperlipoproteinemia(a)

Intervention / Treatment
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* Drug: Pelacarsen (TQJ230) 80 mg s.c.
* Drug: Corresponding Placebo


Participation Criteria
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Eligibility Criteria
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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 40 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 from screening visit to ≤ 10 years prior to the screening visit, and/or Ischemic stroke: ≥ 3 months from screening visit to ≤ 10 years prior to the screening visit, and/or Clinically significant symptomatic peripheral artery disease (PAD) 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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Pelacarsen (TQJ230)<br> | Drug: Pelacarsen (TQJ230) 80 mg s.c.<br>* Pelacarsen (TQJ230) 80 mg s.c. Q4W<br>* Other names: TQJ230;|
| Placebo Comparator: Placebo<br> | Drug: Corresponding Placebo<br>* Placebo to Pelacarsen<br>* Other names: Placebo;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Rate of lipoprotein apheresis sessions performed over 52 weeks normalized to the weekly lipoprotein apheresis schedule | 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 | Over 52 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to lipoprotein apheresis avoidance (where lipoprotein apheresis avoidance is defined as at least 24 weeks of no lipoprotein apheresis until end of study) | Demonstrate superiority of pelacarsen (TQJ230) compared to placebo in reducing the time to lipoprotein apheresis avoidance | At least 24 weeks up to Week 52 | 
| Total avoidance of lipoprotein apheresis from week 12 to week 52 | Demonstrate superiority of pelacarsen (TQJ230) vs placebo in avoiding the performance of any lipoprotein apheresis in the last 40 weeks of the study | Week 12 to Week 52 | 
| Change from baseline to week 52 in the log-transformed Lp(a) (measured prior to planned lipoprotein apheresis) | Demonstrate superiority of pelacarsen (TQJ230) vs placebo in lowering Lp(a) after 52 weeks | 52 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Lipoprotein (a), cardiovascular disease, apheresis, TQJ230, pelacarsen