Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A randomized, double-blind, placebo-controlled, multicenter trial assessing the reduction of the rate of lipoprotein apheresis after treatment with pelacarsen (TQJ230) compared to placebo in patients with hyperlipoproteinemia(a) and established cardiovascular disease undergoing weekly lipoprotein apheresis in Germany

This clinical trial is testing a new medicine, pelacarsen (TQJ230), for adults who have very high levels of a particular type of fat in their blood, called lipoprotein(a), or Lp(a). These high levels have already caused heart problems, and currently, these patients are receiving a regular blood-cleaning treatment called lipoprotein apheresis. The main goal of this study is to find out if taking pelacarsen can reduce how often people need to have their apheresis treatment over 52 weeks. Researchers also want to see if the medicine can help people stop needing apheresis completely for a period of time, and if it lowers the Lp(a) levels themselves. Participants will either receive the new medicine or a dummy medicine (placebo) to fairly compare its effects.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Novartis Pharma AG

Enrolment target

Start

24 Apr 2024

Apheresis

What is this study about?

This study is investigating a new medicine called pelacarsen, also known as TQJ230. It's designed for people who have a condition where they have very high levels of a specific type of fat in their blood, called lipoprotein(a) or Lp(a). Unfortunately, for these individuals, these high Lp(a) levels have already led to heart and blood vessel problems.

Currently, these patients are undergoing a regular treatment called lipoprotein apheresis. This is a special procedure, similar to dialysis, where their blood is filtered to remove the excess Lp(a). The main purpose of this study is to see if taking pelacarsen, in addition to their current care, can help reduce how often they need to have these apheresis sessions over a year. The researchers also want to find out if, and for how long, people might be able to stop needing apheresis altogether, and if the new medicine helps to lower their Lp(a) levels.

By carefully comparing the effects of pelacarsen to a dummy medicine (placebo), the study aims to understand if this new treatment could offer a significant benefit to people living with this condition. It's important research that could potentially improve their quality of life by reducing the burden of frequent treatments.

Key takeaways

This study tests a new medicine (pelacarsen) for people with high Lp(a) levels and heart disease.
It aims to reduce how often patients need regular lipoprotein apheresis treatment.
Participants will receive either the new medicine or a dummy medicine for about one year.
Regular clinic visits and blood tests will be part of the study.
The study helps researchers understand if this new medicine is safe and effective.
You can leave the study at any time without affecting your normal care.

Who may be eligible?

This study is for adults aged 18 and older. Both men and women can take part. To be considered, you must have very high levels of fats called lipoprotein(a) in your blood, and you must have already experienced heart or blood vessel problems because of this condition. You also need to be regularly receiving a special blood-filtering treatment called lipoprotein apheresis.

There will be other specific health requirements and checks to make sure the study is safe for you and that the results will be clear. For example, certain other medical conditions or medicines might mean you can't join.

The research team will carry out a thorough assessment to determine if this study is suitable for you. They will explain everything clearly and answer any questions you have about whether you meet the criteria.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have very high levels of Lp(a) in your blood?
Have you already had problems with your heart or blood vessels due to high Lp(a)?
Are you currently having regular lipoprotein apheresis treatment?
Are you generally able to attend regular clinic appointments for about a year?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you will be in the study for a total of 52 weeks, which is about one year. During this time, you will receive regular injections of either the new medicine (TQJ230) or a placebo (a dummy medicine that looks the same but contains no active drug). You won't know which one you are receiving, and neither will your doctor, which helps make the study fair.

You will continue your regular lipoprotein apheresis treatments as scheduled or as advised by the study team. You'll have regular visits to the clinic for assessments, which will include blood tests to check your Lp(a) levels and other health markers, and to monitor your general health. You'll also be asked questions about how you are feeling and if you've noticed any changes. The main thing being measured is how many apheresis sessions you have during the year. Some people might also be monitored to see if they can completely stop apheresis for a period.

Potential risks and benefits

Taking part in this study could potentially offer benefits, such as a reduction in how often you need your lipoprotein apheresis treatment, or it might even help lower your lipoprotein(a) levels. However, as with any new medicine, there could be potential risks or side effects that are not yet fully known. These will be carefully explained to you by the study team. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care, if you decide it's not right for you.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Germany

Common questions

What is Lp(a)?

Lp(a) is a type of fat and protein particle in your blood. High levels can increase your risk of heart disease, even if your other cholesterol levels are normal.

What is lipoprotein apheresis?

It's a special medical procedure, similar to kidney dialysis, where your blood is filtered to remove excess Lp(a) and other harmful fats. It's usually done regularly for people with very high levels.

Will I know if I'm getting the real medicine or the dummy one?

No, neither you nor your study doctor will know whether you are receiving the real medicine (pelacarsen) or the placebo (dummy medicine). This is called 'double-blind' and helps ensure the study results are fair.

How long will the study last if I join?

The main part of the study will last for 52 weeks, which is about one year.

What if I get side effects from the medicine?

The study team will closely monitor you throughout the trial. If you experience any side effects, you should tell them immediately so they can provide appropriate care.