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

Study Overview
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Brief Summary
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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
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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.

Official Title
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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.

Conditions
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Hyperlipoproteinemia (a)

Intervention / Treatment
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* Drug: Pelacarsen


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Open-label extension (OLE) study
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Pelacarsen (TQJ230)<br>open-label pelacarsen 80 mg | Drug: Pelacarsen<br>* Pelacarsen 80mg s.c. monthly<br>* Other names: TQJ230;|

What is the study measuring?
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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) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Lipoprotein(a), Cardiovascular disease, Apheresis, Post trial access, pelacarsen (TQJ230)