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

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

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.

Study Overview

• Status: Completed
• Conditions: Hyperlipoproteinemia(a)
• Intervention / Treatment: Drug: Pelacarsen (TQJ230) 80 mg s.c.; Drug: Corresponding Placebo

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) investigated 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

• Study Type: Interventional
• Enrollment (Actual): 51
• Phase: Phase 3

Contacts and Locations

Study Locations

• Germany
  • Berlin, Germany, 13353
    • Novartis Investigative Site
  • Cloppenburg, Germany, 49661
    • Novartis Investigative Site
  • Erlangen, Germany, 91054
    • Novartis Investigative Site
  • Mainz, Germany, 55131
    • Novartis Investigative Site
  • Ulm, Germany, 89081
    • Novartis Investigative Site
  • Baden-Wurttemberg
    • Villingen-Schwenningen, Baden-Wurttemberg, Germany, 78052
      • Novartis Investigative Site
  • Bavaria
    • Munich, Bavaria, Germany, 81377
      • Novartis Investigative Site
    • Würzburg, Bavaria, Germany, 97080
      • Novartis Investigative Site
  • Hesse
    • Frankfurt am Main, Hesse, Germany, 60431
      • Novartis Investigative Site
  • Lower Saxony
    • Göttingen, Lower Saxony, Germany, 37075
      • Novartis Investigative Site
  • North Rhine-Westphalia
    • Düsseldorf, North Rhine-Westphalia, Germany, 42010
      • Novartis Investigative Site
    • Geilenkirchen, North Rhine-Westphalia, Germany, 52511
      • Novartis Investigative Site
  • Saxony
    • Dresden, Saxony, Germany, 01307
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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)
• Clinically significant symptomatic coronary 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Pelacarsen (TQJ230); Pelacarsen (TQJ230) 80 mg s.c. Q4W	Drug: Pelacarsen (TQJ230) 80 mg s.c.; Pelacarsen (TQJ230) 80 mg s.c. Q4W; Other Names: TQJ230
Placebo Comparator: Placebo; Placebo to Pelacarsen s.c. Q4W	Drug: Corresponding Placebo; Placebo to Pelacarsen; Other Names: Placebo

What is the study measuring?

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	Rate (proportion) of apheresis sessions was calculated as the number of actual lipoprotein apheresis (LA) sessions received, divided by the number of planned LA sessions during the 52-week period, which is 52 for patients who completed all study visits, or pro-rated for those who discontinued early. This rate could range from 0 to 1, with 0 indicating that the patient had skipped all planned LA sessions, and 1 indicating that the patient had received all planned sessions. Multiple imputation for missing Lp(a) data was performed, and missing apheresis data was imputed.	Up to Week 52

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to Lipoprotein Apheresis Avoidance	Lipoprotein apheresis avoidance is defined as at least 24 consecutive weeks of no lipoprotein apheresis until end of study.	From randomization up to Week 52
Number of Participants With Total Lipoprotein Apheresis Avoidance From Week 12 to Week 52	Total lipoprotein apheresis avoidance is defined as no apheresis performed from Week 12 to Week 52.	Week 12 up to Week 52
Change From Baseline to Week 52 in the Log-transformed Lp(a) Reported as mg/dL	Week 52 / Baseline ratio in Lp(a) of pelacarsen (TQJ230) vs placebo reported as particle mass (mg/dL). Baseline Lp(a) was defined as the last non-missing pre-lipoprotein apheresis assessment prior to the first dose of randomized study drug.	Baseline, week 52
Change From Baseline to Week 52 in the Log-transformed Lp(a) Reported as Nmol/L	Week 52 / Baseline ratio in Lp(a) of pelacarsen (TQJ230) vs placebo reported as molar concentration (nmol/L). Baseline Lp(a) was defined as the last non-missing pre-lipoprotein apheresis assessment prior to the first dose of randomized study drug.	Baseline, week 52

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rate of Lipoprotein Apheresis Sessions From Week 12 to Week 52 and Week 24 to Week 52 Normalized to the Weekly Lipoprotein Apheresis Schedule	Rate (proportion) of apheresis sessions is calculated using the following formula: the total number of apheresis sessions performed over the double-blinded treatment period/total weeks from Week12 to Week52 or total weeks from Week24 to Week52. Multiple imputation for missing Lp(a) data was performed and missing apheresis data were imputed.	Week 12 to 52, Week 24 to 52
Number of Participants With Total Lipoprotein Apheresis Avoidance From Week 24 to Week 52	Number of participants with no apheresis performed between week 24 to week 52.	Week 24 to Week 52
Time-averaged Lp(a) Levels Reported as mg/dL	Time averaged Lp(a) levels were calculated as CAVG = CMIN + 0.73 × (CMAX-CMIN), where CMAX and CMIN are the immediate pre- and post-apheresis Lp(a) levels.	Baseline, Week 52
Time-averaged Lp(a) Levels Reported as Nmol/L	Time averaged Lp(a) levels were calculated as CAVG = CMIN + 0.73 × (CMAX-CMIN), where CMAX and CMIN are the immediate pre- and post-apheresis Lp(a) levels.	Baseline, Week 52
Percentage Change in Total Cholesterol, LDL-C, High-density Lipoprotein-Cholesterol (HDL-C), Non-HDL-C, Very-low-density Lipoprotein-Cholesterol (VLDL-C), apoB and Triglycerides (Pre- Lipoprotein Apheresis) From Baseline to Week 52	Evaluate the change in expanded lipid profile parameters measured in mg/dL	Baseline, Week 52
Change From Baseline to Week 52 in the Physical Health Summary Score for the SF-36 Questionnaire	The Short Form-36 Physical Component Summary (SF-36 PCS) is an instrument to measure health-related quality of life among healthy patients and patients with acute and chronic conditions. It consists of eight subscales (domains) that can be scored individually: Physical Functioning, Role-Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role- Emotional, and Mental Health. The outcome of the questionnaires in eight scales results in two summary scores, physical component and mental component, both ranging from 0 - 100. Higher scores indicate a higher level of functioning. A positive change from baseline score indicates an improvement.	Baseline, Week 52
Change From Baseline to Week 52 in the Mental Health Summary Score for the SF-36 Questionnaire	The Short Form-36 Physical Component Summary (SF-36 PCS) is an instrument to measure health-related quality of life among healthy patients and patients with acute and chronic conditions. It consists of eight subscales (domains) that can be scored individually: Physical Functioning, Role-Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role- Emotional, and Mental Health. The outcome of the questionnaires in eight scales results in two summary scores, physical component and mental component, both ranging from 0 - 100. Higher scores indicate a higher level of functioning. A positive change from baseline score indicates an improvement.	Baseline, Week 52
Patient Preference Questionnaire	Participants were asked for their treatment preference between weekly lipoprotein apheresis or monthly self-injection. The number of participants that prefer each option at baseline and 52 weeks is reported.	Baseline, Week 52

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Publications and helpful links

General Publications

• Parhofer KG, Julius U, Herzog AL, Kruger T, Weinmann-Menke J, Heine GH, Cao H, Schorr J, Ferber P, Wang J, Cristante E, Muller-Edenborn B, Vogt A, Schettler V, Steinhagen-Thiessen E. Pelacarsen and lipoprotein(a) apheresis in secondary prevention: the Lp(a)FRONTIERS APHERESIS trial. Eur Heart J. 2026 Jul 1;47(25):3284-3294. doi: 10.1093/eurheartj/ehag073.

Study record dates

Study Major Dates

• Study Start (Actual): August 19, 2022
• Primary Completion (Actual): January 28, 2025
• Study Completion (Actual): January 28, 2025

Study Registration Dates

• First Submitted: March 14, 2022
• First Submitted That Met QC Criteria: March 22, 2022
• First Posted (Actual): March 31, 2022

Study Record Updates

• Last Update Posted (Actual): July 17, 2026
• Last Update Submitted That Met QC Criteria: July 16, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Keywords: cardiovascular disease; apheresis; TQJ230; Lipoprotein (a); pelacarsen
• Additional Relevant MeSH Terms: Metabolic Diseases; Dyslipidemias; Lipid Metabolism Disorders; Cardiovascular Diseases; pelacarsen
• Other Study ID Numbers: CTQJ230A12302; 2021-003059-41 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No