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

Phase 3

Active, not recruiting

• Conditions: Hyperlipoproteinemia (a)
• Interventions: Drug: Pelacarsen

• Registration Number: NCT05900141
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

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).

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 Timeline

• Study First Submitted: 2023-06-02
• Study First Submitted QC: 2023-06-02
• Study First Posted: 2023-06-12
• Study Start: 2023-09-29
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-14
• Last Update Posted: 2025-04-18
• Primary Completion: 2028-08-24
• Study Completion: 2028-08-24

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 41

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Pelacarsen (TQJ230)	Pelacarsen	open-label pelacarsen 80 mg

Primary Outcome Measures

Name	Time	Method
Duration of drug exposure	60 months	Duration of drug exposure will be collected
Incidence of Adverse events (AEs) or serious adverse events (SAEs), including changes in laboratory evaluations, vital signs qualifying and reported as AEs.	60 months	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

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Novartis Investigative Site; 🇩🇪 Wuerzburg, Germany