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
Primary Purpose
Hyperlipoproteinemia(a)
Status
Recruiting
Phase
Phase 3
Locations
Germany
Study Type
Interventional
Intervention
Pelacarsen (TQJ230) 80 mg s.c.
Corresponding Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Hyperlipoproteinemia(a) focused on measuring Lipoprotein (a), cardiovascular disease, apheresis, TQJ230, pelacarsen
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
Sites / Locations
- Novartis Investigative SiteRecruiting
- Novartis Investigative SiteRecruiting
- Novartis Investigative SiteRecruiting
- Novartis Investigative SiteRecruiting
- Novartis Investigative SiteRecruiting
- Novartis Investigative SiteRecruiting
- Novartis Investigative SiteRecruiting
- Novartis Investigative SiteRecruiting
- Novartis Investigative SiteRecruiting
- Novartis Investigative SiteRecruiting
- Novartis Investigative SiteRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Pelacarsen (TQJ230)
Placebo
Arm Description
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
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
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
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
Full Information
NCT ID
NCT05305664
First Posted
March 14, 2022
Last Updated
September 26, 2023
Sponsor
Novartis Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT05305664
Brief Title
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
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 19, 2022 (Actual)
Primary Completion Date
February 12, 2025 (Anticipated)
Study Completion Date
February 12, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hyperlipoproteinemia(a)
Keywords
Lipoprotein (a), cardiovascular disease, apheresis, TQJ230, pelacarsen
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
60 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Pelacarsen (TQJ230)
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Pelacarsen (TQJ230) 80 mg s.c.
Other Intervention Name(s)
TQJ230
Intervention Description
Pelacarsen (TQJ230) 80 mg s.c. Q4W
Intervention Type
Drug
Intervention Name(s)
Corresponding Placebo
Other Intervention Name(s)
Placebo
Intervention Description
Placebo to Pelacarsen
Primary Outcome Measure Information:
Title
Rate of lipoprotein apheresis sessions performed over 52 weeks normalized to the weekly lipoprotein apheresis schedule
Description
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
Time Frame
Over 52 Weeks
Secondary Outcome Measure Information:
Title
Time to lipoprotein apheresis avoidance (where lipoprotein apheresis avoidance is defined as at least 24 weeks of no lipoprotein apheresis until end of study)
Description
Demonstrate superiority of pelacarsen (TQJ230) compared to placebo in reducing the time to lipoprotein apheresis avoidance
Time Frame
At least 24 weeks up to Week 52
Title
Total avoidance of lipoprotein apheresis from week 12 to week 52
Description
Demonstrate superiority of pelacarsen (TQJ230) vs placebo in avoiding the performance of any lipoprotein apheresis in the last 40 weeks of the study
Time Frame
Week 12 to Week 52
Title
Change from baseline to week 52 in the log-transformed Lp(a) (measured prior to planned lipoprotein apheresis)
Description
Demonstrate superiority of pelacarsen (TQJ230) vs placebo in lowering Lp(a) after 52 weeks
Time Frame
52 weeks
Other Pre-specified Outcome Measures:
Title
Rate of lipoprotein apheresis sessions from week 12 to week 52 and week 24 to week 52 normalized to the weekly lipoprotein apheresis schedule
Description
Evaluate rate of lipoprotein apheresis at defined periods of time
Time Frame
Week 12 to 52, Week 24 to 52
Title
Total avoidance of lipoprotein apheresis between week 24 to week 52
Description
Evaluate avoidance of the performance of any lipoprotein apheresis in the last 28 weeks of the study
Time Frame
Week 24 to Week 52
Title
Time-averaged Lp(a) levels
Description
Evaluate pre- and post-lipoprotein apheresis Lp(a) levels
Time Frame
52 weeks
Title
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
Description
Evaluate the change in expanded lipid profile parameters
Time Frame
52 weeks
Title
Change from baseline to week 52 in the physical and mental health summary scores for the SF-36 questionnaire and a patient preference questionnaire
Description
Evaluate quality of life
Time Frame
52 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Novartis Pharmaceuticals
Phone
+41613241111
Email
novartis.email@novartis.com
First Name & Middle Initial & Last Name or Official Title & Degree
Novartis Pharmaceuticals
Facility Information:
Facility Name
Novartis Investigative Site
City
Duesseldorf
State/Province
North Rhine-Westphalia
ZIP/Postal Code
42010
Country
Germany
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Geilenkirchen
State/Province
North Rhine-Westphalia
ZIP/Postal Code
52511
Country
Germany
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Berlin
ZIP/Postal Code
13353
Country
Germany
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Dresden
ZIP/Postal Code
01307
Country
Germany
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Frankfurt
ZIP/Postal Code
60431
Country
Germany
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Gottingen
ZIP/Postal Code
37075
Country
Germany
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Mainz
ZIP/Postal Code
55131
Country
Germany
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Muenchen
ZIP/Postal Code
81377
Country
Germany
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Ulm
ZIP/Postal Code
89081
Country
Germany
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Villingen-Schwenningen
ZIP/Postal Code
D78052
Country
Germany
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Wuerzburg
ZIP/Postal Code
97080
Country
Germany
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing 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
Learn more about this trial
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
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