Hyperlipoproteinemia (a)
Conditions
Keywords
Lipoprotein(a), Cardiovascular disease, Apheresis, Post trial access, pelacarsen (TQJ230)
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.
Interventions
DRUGPelacarsen
Pelacarsen 80mg s.c. monthly
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Masking description
Open label study
Intervention model description
Open-label extension (OLE) study
Eligibility
Sex/Gender
ALL
Age
18 Years to 100 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
| Duration of drug exposure | 60 months | Duration of drug exposure will be collected |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
Germany
Outcome results
None listed