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

Hyperlipoproteinemia(a) Clinical Trial

Official 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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05305664
• Other study ID #: CTQJ230A12302
• Secondary ID: 2021-003059-41
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: August 19, 2022
• Est. completion date: June 27, 2025

Study information

• Verified date: May 2024
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 51
• Est. completion date: June 27, 2025
• Est. primary completion date: June 27, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Cardiovascular Diseases
• Hyperlipidemias
• Hyperlipoproteinemia(a)
• Hyperlipoproteinemias

Intervention

Drug:
• Pelacarsen (TQJ230) 80 mg s.c.
Pelacarsen (TQJ230) 80 mg s.c. Q4W
• Corresponding Placebo
Placebo to Pelacarsen

Locations

Country	Name	City	State
Germany	Novartis Investigative Site	Berlin
Germany	Novartis Investigative Site	Cloppenburg
Germany	Novartis Investigative Site	Dresden
Germany	Novartis Investigative Site	Duesseldorf	North Rhine-Westphalia
Germany	Novartis Investigative Site	Erlangen
Germany	Novartis Investigative Site	Frankfurt
Germany	Novartis Investigative Site	Geilenkirchen	North Rhine-Westphalia
Germany	Novartis Investigative Site	Gottingen
Germany	Novartis Investigative Site	Mainz
Germany	Novartis Investigative Site	Muenchen
Germany	Novartis Investigative Site	Ulm
Germany	Novartis Investigative Site	Villingen-Schwenningen
Germany	Novartis Investigative Site	Wuerzburg

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Rate of lipoprotein apheresis sessions from week 12 to week 52 and week 24 to week 52 normalized to the weekly lipoprotein apheresis schedule	Evaluate rate of lipoprotein apheresis at defined periods of time	Week 12 to 52, Week 24 to 52
Other	Total avoidance of lipoprotein apheresis between week 24 to week 52	Evaluate avoidance of the performance of any lipoprotein apheresis in the last 28 weeks of the study	Week 24 to Week 52
Other	Time-averaged Lp(a) levels	Evaluate pre- and post-lipoprotein apheresis Lp(a) levels	52 weeks
Other	Percent 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	52 weeks
Other	Change from baseline to week 52 in the physical and mental health summary scores for the SF-36 questionnaire and a patient preference questionnaire	Evaluate quality of life	52 weeks
Primary	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	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
Secondary	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
Secondary	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