Pelacarsen for Cardiovascular Disease

Recruiting at 24 trial locations

Overseen By Novartis Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Novartis Pharmaceuticals

Must not be taking: Investigational drugs

Disqualifiers: Adverse events, Medical conditions, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

600 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial focuses on providing ongoing access to a treatment called pelacarsen for individuals with cardiovascular disease. It aims to continue supporting participants who completed earlier studies with pelacarsen, a medication for managing cardiovascular conditions. Suitable candidates include those who finished the previous studies without major issues or treatment discontinuation. As a Phase 3 trial, this is the final step before FDA approval, offering participants the opportunity to contribute to the potential availability of a new treatment.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

Is there any evidence suggesting that pelacarsen is likely to be safe for humans?

Research has shown that pelacarsen is generally safe for people. One study found pelacarsen to be safe and well-tolerated, even in participants with mild liver problems. Most side effects were mild, and no serious health issues were directly linked to the treatment. Another study demonstrated that pelacarsen effectively lowered lipoprotein(a) levels, a risk factor for heart disease, without causing major safety concerns. These findings suggest that pelacarsen is a promising treatment with a good safety record. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Pelacarsen is unique because it targets lipoprotein(a), a specific type of cholesterol particle that has been linked to cardiovascular disease, which is not directly addressed by current treatments like statins or PCSK9 inhibitors. Unlike these standard treatments that primarily focus on lowering LDL cholesterol, Pelacarsen works by reducing the levels of lipoprotein(a) in the blood, potentially offering a new avenue for patients who have high levels of this particle and are at risk for cardiovascular events. Researchers are excited about Pelacarsen because it could fill a gap in cardiovascular care, providing a targeted treatment option for those with elevated lipoprotein(a) who may not benefit fully from existing therapies.

What evidence suggests that pelacarsen might be an effective treatment for cardiovascular disease?

Research has shown that pelacarsen, the investigational treatment in this trial, can lower lipoprotein(a) levels, which is linked to a reduced risk of heart disease. In one study, pelacarsen significantly lowered lipoprotein(a) levels, with 98% of patients reaching safer levels for heart health. This reduction helps prevent heart problems like coronary heart disease. Another study confirmed that pelacarsen reduces heart risks compared to a placebo. These findings suggest that pelacarsen could help lower heart-related issues for people with high lipoprotein(a) levels. ^{2 3 6 7 8}

Are You a Good Fit for This Trial?

This trial is for individuals who have completed a previous pelacarsen study and received treatment until the end. They must consent to continue in this extension program, which provides ongoing access to pelacarsen.

Inclusion Criteria

I have given my consent to participate in the study.

I completed the initial study and received all planned treatments.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Open-label Extension

Participants receive open-label pelacarsen 80 mg once a month

36 months

Monthly visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Pelacarsen

Trial Overview

The trial is providing participants with continued access to pelacarsen (TQJ230), a medication being investigated for its potential benefits in treating cardiovascular diseases and preventing strokes.

How Is the Trial Designed?

Group I: Pelacarsen (TQJ230) Experimental Treatment 1 Intervention

open-label pelacarsen 80mg

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

Citations

1.

jacc.org

jacc.org/doi/10.1016/j.jacc.2021.12.032

Effect of Pelacarsen on Lipoprotein(a) Cholesterol and ...

Pelacarsen significantly lowers direct Lp(a)-C and has neutral to mild lowering of LDL-C corr. In patients with elevated Lp(a), LDL-C corr provides a more ...

2.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04023552

NCT04023552 | Assessing the Impact of Lipoprotein (a) ...

Demonstrate the superiority of pelacarsen (TQJ230) compared to placebo in reducing the risk of the composite of coronary heart disease (CHD) outcomes: death due ...

3.

ir.ionis.com

ir.ionis.com/news-releases/news-release-details/ionis-announces-enrollment-completion-phase-3-lpa-horizon

Ionis announces enrollment completion of Phase 3 Lp(a) ...

Data from a Phase 2 study showed pelacarsen reduced Lp(a) levels below the recommended threshold of risk for CVD events (<50 mg/dL, <125 nmol/L) in 98% of ...

4.

novartis.com

novartis.com/clinicaltrials/study/nct06813911

Lp(a) Lowering Study of Pelacarsen (TQJ230) With ...

The purpose of the study CTQJ230A12304, is to evaluate the efficacy, safety, and tolerability of pelacarsen (TQJ230) compared to placebo in ...

5.

natap.org

natap.org/2022/HIV/090322_04.htm

Antisense Drug Reduced LPa; in Phase 3 Study Assessing ...

Lp(a) HORIZON is evaluating the safety and efficacy of pelacarsen in reducing cardiovascular events in patients with cardiovascular disease and elevated levels ...

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04023552

NCT04023552 | Assessing the Impact of Lipoprotein (a) ...

This is a pivotal phase 3 study designed to support an indication for the reduction of cardiovascular risk in patients with established CVD and elevated Lp(a)

7.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12478598/

Pharmacokinetics and Safety of Pelacarsen, a GalNAc3

The results of this phase I study support that pelacarsen is safe and well tolerated in participants with mild HI. Additionally, mild HI had ...

8.

sciencedirect.com

sciencedirect.com/science/article/pii/S0002870325001012

Design and Rationale of Lp(a)HORIZON Trial: Assessing ...

Pelacarsen significantly reduced Lp(a) in a dose-dependent manner with mean percent decrease of 35% at 20 mg every 4 weeks, 56% for 40 mg every 4 weeks, 58% for ...