Clinical trial 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
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hyperlipoproteinemia(a)
Keywords: Lipoprotein (a)
cardiovascular disease
apheresis
TQJ230
pelacarsen
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2022-03-31
Enrollment: 51
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

Eligibility criteria: 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

Primary outcomes:
measure: Rate of lipoprotein apheresis sessions performed over 52 weeks normalized to the weekly lipoprotein apheresis schedule
timeFrame: Over 52 Weeks
Secondary outcomes:
measure: Time to lipoprotein apheresis avoidance (where lipoprotein apheresis avoidance is defined as at least 24 weeks of no lipoprotein apheresis until end of study)
timeFrame: At least 24 weeks up to Week 52
measure: Total avoidance of lipoprotein apheresis from week 12 to week 52
timeFrame: Week 12 to Week 52
measure: Change from baseline to week 52 in the log-transformed Lp(a) (measured prior to planned lipoprotein apheresis)
timeFrame: 52 weeks
Other outcomes:
measure: Rate of lipoprotein apheresis sessions from week 12 to week 52 and week 24 to week 52 normalized to the weekly lipoprotein apheresis schedule
timeFrame: Week 12 to 52, Week 24 to 52
measure: Total avoidance of lipoprotein apheresis between week 24 to week 52
timeFrame: Week 24 to Week 52
measure: Time-averaged Lp(a) levels
timeFrame: 52 weeks
measure: 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
timeFrame: 52 weeks
measure: Change from baseline to week 52 in the physical and mental health summary scores for the SF-36 questionnaire and a patient preference questionnaire
timeFrame: 52 weeks
Locations:
city: Duesseldorf
country: Germany
facility: Novartis Investigative Site
state: North Rhine-Westphalia
city: Geilenkirchen
country: Germany
facility: Novartis Investigative Site
state: North Rhine-Westphalia
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Cloppenburg
country: Germany
facility: Novartis Investigative Site
city: Dresden
country: Germany
facility: Novartis Investigative Site
city: Erlangen
country: Germany
facility: Novartis Investigative Site
city: Frankfurt
country: Germany
facility: Novartis Investigative Site
city: Gottingen
country: Germany
facility: Novartis Investigative Site
city: Mainz
country: Germany
facility: Novartis Investigative Site
city: Muenchen
country: Germany
facility: Novartis Investigative Site
city: Ulm
country: Germany
facility: Novartis Investigative Site
city: Villingen-Schwenningen
country: Germany
facility: Novartis Investigative Site
city: Wuerzburg
country: Germany
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: