Pelacarsen for Cardiovascular Disease

(Lp(a)HORIZON Trial)

No longer recruiting at 835 trial locations

Overseen By Novartis Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Novartis Pharmaceuticals

Disqualifiers: Uncontrolled hypertension, Heart failure, Malignancy, others

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

Duration: 4 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called Pelacarsen to determine if it can lower the risk of heart problems in individuals with cardiovascular disease and high levels of Lp(a) cholesterol. Participants will receive either Pelacarsen or a placebo (a harmless shot resembling the real treatment) through monthly injections. The trial seeks individuals who have experienced a heart attack, stroke, or serious artery disease in the past 3 to 10 years and have high Lp(a) levels. Those with these conditions might be suitable candidates for the trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Do I need to stop my current medications to join the trial?

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

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

Research shows that pelacarsen, also known as TQJ230, is generally safe for people. Previous studies have found that most patients taking pelacarsen did not experience major side effects.

One study highlighted that pelacarsen is well-tolerated, meaning that while some people might have mild side effects, serious problems are rare. Overall, these findings suggest that pelacarsen is safe for most patients.

As this is a late-stage trial, strong evidence already supports pelacarsen's safety for humans. This trial aims to confirm its effectiveness in lowering the risk of heart disease for people with high levels of Lp(a), a type of cholesterol linked to heart issues. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for cardiovascular disease, which often focus on managing symptoms and reducing risk factors, Pelacarsen (TQJ230) specifically targets lipoprotein(a), a known contributor to heart disease that is not addressed by most current therapies. This treatment is unique because it uses an innovative approach to lower lipoprotein(a) levels through monthly subcutaneous injections, potentially reducing cardiovascular events more effectively. Researchers are excited about Pelacarsen because it offers a new way to target a specific risk factor that many traditional therapies overlook, potentially leading to better outcomes for patients with cardiovascular disease.

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

Research has shown that pelacarsen, which participants in this trial may receive, can significantly lower levels of lipoprotein(a), or Lp(a), a substance in the blood linked to a higher risk of heart disease. Studies have found that pelacarsen can reduce Lp(a) levels by 35% to 80% in people with heart disease due to high Lp(a). These reductions are promising because high Lp(a) is a known risk factor for heart problems. The treatment is also considered safe, allowing most people to use it without serious side effects. Early data suggests that pelacarsen could effectively lower the risk of heart-related issues in those with high Lp(a). ^{1 2 3 4 5}

Who Is on the Research Team?

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Are You a Good Fit for This Trial?

This trial is for people with cardiovascular disease who've had a heart attack or ischemic stroke between 3 months and 10 years ago, or have significant peripheral artery disease. They must also have high levels of Lp(a), a type of cholesterol particle, as confirmed by the central lab. It's not open to those with active liver disease, severe kidney issues, uncontrolled blood pressure, advanced heart failure, low platelets count, cancer history or recent major bleeding.

Inclusion Criteria

I have severe leg pain due to poor blood circulation.

I had a heart attack between 3 months and 10 years ago.

Lp(a) ≥ 70 mg/dL at the screening visit, measured at the Central laboratory

Exclusion Criteria

I have a serious kidney condition.

Platelet count ≤LLN

My heart condition severely limits my physical activity.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive monthly subcutaneous injections of TQJ230 or placebo

4 years

Monthly visits for injections

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Pelacarsen (TQJ230)

Trial Overview

The study tests Pelacarsen (TQJ230) to see if it can reduce the risk of serious heart events in patients with existing cardiovascular conditions and elevated Lp(a). Participants will either receive TQJ230 or a placebo without knowing which one they're getting to compare outcomes fairly.

How Is the Trial Designed?

Group I: TQJ230 Experimental Treatment 1 Intervention

TQJ230 80 mg injected monthly administered subcutaneously

Group II: Placebo Placebo Group 1 Intervention

Monthly subcutaneous injections.

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.

ir.ionis.com

ir.ionis.com/news-releases/news-release-details/phase-2-results-akcea-apoa-lrx-presented-late-breaking-clinical

Phase 2 Results on AKCEA-APO(a)-LRx Presented in Late ...

“These data show that AKCEA-APO(a)-LRx significantly reduces Lp(a) in patients with pre-existing cardiovascular disease due to elevated Lp(a) ...

2.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC7577764/

Pelacarsen for lowering lipoprotein(a)

Pelacarsen safely and dose-dependently decreased Lp(a) levels by 35–80% and a Phase 3 trial [Lp(a)HORIZON, NCT04023552] is planned to run from 2020 to 2024.

3.

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 ...

4.

ir.ionis.com

ir.ionis.com/news-releases/news-release-details/new-england-journal-medicine-publishes-results-phase-2-study

LRx in Patients with Lp(a)-driven Cardiovascular Disease

Patients treated with AKCEA-APO(a)-LRx received pre-specified endpoints for Lp(a) levels with favorable safety and tolerability profile.

5.

sciencedirect.com

sciencedirect.com/science/article/pii/S1933287425003228

Pelacarsen: Mechanism of action and Lp(a)-lowering effect

A phase 2b study of pelacarsen showed ≥80% reduction in Lp(a) concentration with a favorable safety profile in patients with established ASCVD.