An Open Label Extension (OLE) Study to Evaluate Long-term Safety and Tolerability of Pelacarsen (TQJ230) (OLE)

A Single Arm, Open Label Extension (OLE), Multicenter Study to Evaluate Long-term Safety and Tolerability of Pelacarsen (TQJ230) in Patients With Cardiovascular Disease Who Have Successfully Completed the Apheresis Parent Study.

This non-randomized, open-label extension study will provide post-trial access to pelacarsen (TQJ230) to participants in Germany with hyperlipoproteinemia(a) and established cardiovascular disease who have successfully completed the double-blind parent study (CTQJ230A12302).

Study Overview

• Status: Recruiting
• Conditions: Hyperlipoproteinemia (a)
• Intervention / Treatment: Drug: Pelacarsen

Detailed Description

This study will allow to evaluate a long-term safety and tolerability of pelacarsen (TQJ230) 80 mg QM in patients. All consenting participants from the parent study who meet the eligibility criteria will be participating in this open label extension study. The maximum duration of this study is approximately 5 years.

• Study Type: Interventional
• Enrollment (Estimated): 60
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals; Phone Number: +41613241111; Email: novartis.email@novartis.com
• Study Contact Backup: Name: Novartis Pharmaceuticals

Study Locations

• Germany
  • Berlin, Germany, 13353
    • Recruiting
    • Novartis Investigative Site
  • Dresden, Germany, 01307
    • Recruiting
    • Novartis Investigative Site
  • Mainz, Germany, 55131
    • Recruiting
    • Novartis Investigative Site
  • Muenchen, Germany, 81377
    • Recruiting
    • Novartis Investigative Site
  • Wuerzburg, Germany, 97080
    • Recruiting
    • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Signed informed consent
• Participant has successfully completed the parent study and is considered safe to participate by Investigator's clinical judgement.

Exclusion Criteria:

• Any medical condition(s) that may put the participant at risk in the investigator's opinion
• Pregnant or nursing (lactating) women
• Women of child-bearing potential, unless they are using effective methods of contraception during dosing and for 16 weeks (= 5 times the terminal half-life) after stopping of study medication

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Pelacarsen (TQJ230); open-label pelacarsen 80 mg	Drug: Pelacarsen; Pelacarsen 80mg s.c. monthly; Other Names: TQJ230

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Adverse events (AEs) or serious adverse events (SAEs), including changes in laboratory evaluations, vital signs qualifying and reported as AEs.	Evaluate long-term safety and tolerability of pelacarsen (TQJ230) in participants with elevated Lp(a) and established cardiovascular disease who have competed the parent study	60 months
Duration of drug exposure	Duration of drug exposure will be collected	60 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of lipoprotein apheresis sessions performed yearly for year 1, 2, 3, 4 and 5	Evaluate the long-term effect of pelacarsen on the need for lipoprotein apheresis.	60 months
Absolute change in Lp(a) compared to baseline of the parent study.	Evaluate the long-term effect of pelacarsen on Lp(a) change.	month 1, month 3, month 6, month 9, month 12, then every 6 months during OLE (up to 60 months)
Percentage change in Lp(a) compared to baseline of the parent study.	Evaluate the long-term effect of pelacarsen on Lp(a) change.	month 1, month 3, month 6, month 9, month 12, then every 6 months during OLE (up to 60 months)

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): September 29, 2023
• Primary Completion (Estimated): August 24, 2028
• Study Completion (Estimated): August 24, 2028

Study Registration Dates

• First Submitted: June 2, 2023
• First Submitted That Met QC Criteria: June 2, 2023
• First Posted (Actual): June 12, 2023

Study Record Updates

• Last Update Posted (Actual): March 26, 2024
• Last Update Submitted That Met QC Criteria: March 25, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: Cardiovascular disease; Lipoprotein(a); Apheresis; Post trial access; pelacarsen (TQJ230)
• Additional Relevant MeSH Terms: Metabolic Diseases; Lipid Metabolism Disorders; Dyslipidemias; Hyperlipidemias; Hyperlipoproteinemias
• Other Study ID Numbers: CTQJ230A12302E1; 2023-503611-16-00 (Registry Identifier: EUCTR)

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