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

Phase 3

Active, not recruiting

• Conditions: Hyperlipoproteinemia(a)
• Interventions: Drug: Pelacarsen (TQJ230) 80 mg s.c.; Drug: Corresponding Placebo

• Registration Number: NCT05305664
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

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

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

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2022-03-14
• Study First Submitted QC: 2022-03-22
• Study First Posted: 2022-03-31
• Study Start: 2022-08-19
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-25
• Last Update Posted: 2024-12-27
• Primary Completion: 2025-01-31
• Study Completion: 2025-01-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 51

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)

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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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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Pelacarsen (TQJ230)	Pelacarsen (TQJ230) 80 mg s.c.	-
Placebo	Corresponding Placebo	-

Primary Outcome Measures

Name	Time	Method
Rate of lipoprotein apheresis sessions performed over 52 weeks normalized to the weekly lipoprotein apheresis schedule	Over 52 Weeks	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

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Novartis Investigative Site; 🇩🇪 Wuerzburg, Germany