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Completed

NCT Number: NCT05305664

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 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.

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Key information

Conditions

Hyperlipoproteinemia(a) Cardiovascular Diseases

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Novartis Investigative Site, Villingen-Schwenningen, Baden-Wurttemberg, Germany

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About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Pelacarsen (TQJ230) 80 mg s.c.

Drug

Pelacarsen (TQJ230) 80 mg s.c. Q4W

Other names: TQJ230

Corresponding Placebo

Drug

Placebo to Pelacarsen

Other names: Placebo

Primary outcomes

  1. Rate of Lipoprotein Apheresis Sessions Performed Over 52 Weeks Normalized to the Weekly Lipoprotein Apheresis Schedule

    Time frame: Up to Week 52

    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.

Secondary outcomes

  1. Time to Lipoprotein Apheresis Avoidance

    Time frame: From randomization up to Week 52

    Lipoprotein apheresis avoidance is defined as at least 24 consecutive weeks of no lipoprotein apheresis until end of study.

  2. Number of Participants With Total Lipoprotein Apheresis Avoidance From Week 12 to Week 52

    Time frame: Week 12 up to Week 52

    Total lipoprotein apheresis avoidance is defined as no apheresis performed from Week 12 to Week 52.

  3. Change From Baseline to Week 52 in the Log-transformed Lp(a) Reported as mg/dL

    Time frame: Baseline, week 52

    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.

  4. Change From Baseline to Week 52 in the Log-transformed Lp(a) Reported as Nmol/L

    Time frame: Baseline, week 52

    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.

Other outcomes

  1. Rate of Lipoprotein Apheresis Sessions From Week 12 to Week 52 and Week 24 to Week 52 Normalized to the Weekly Lipoprotein Apheresis Schedule

    Time frame: Week 12 to 52, Week 24 to 52

    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.

  2. Number of Participants With Total Lipoprotein Apheresis Avoidance From Week 24 to Week 52

    Time frame: Week 24 to Week 52

    Number of participants with no apheresis performed between week 24 to week 52.

  3. Time-averaged Lp(a) Levels Reported as mg/dL

    Time frame: Baseline, Week 52

    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.

  4. Time-averaged Lp(a) Levels Reported as Nmol/L

    Time frame: Baseline, Week 52

    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.

  5. 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

    Time frame: Baseline, Week 52

    Evaluate the change in expanded lipid profile parameters measured in mg/dL

  6. Change From Baseline to Week 52 in the Physical Health Summary Score for the SF-36 Questionnaire

    Time frame: Baseline, Week 52

    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.

  7. Change From Baseline to Week 52 in the Mental Health Summary Score for the SF-36 Questionnaire

    Time frame: Baseline, Week 52

    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.

  8. Patient Preference Questionnaire

    Time frame: Baseline, Week 52

    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.

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2022
Primary completion
2025
Study completion
2025
First posted
Mar 31, 2022
Registry last updated
Jul 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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