Active not recruitingPHASE3INTERVENTIONAL

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)

This study is for people who have already taken part in a trial for a new medicine called Olezarsen (previously known as AKCEA-APOCIII-LRx). Olezarsen is being tested to see how it affects high triglyceride levels in people with a rare inherited condition called Familial Chylomicronemia Syndrome (FCS). High triglycerides can cause serious health problems. This new study is an extension of the previous one, meaning those who finished the first study and found it suitable can continue to receive Olezarsen. The aim is to gather more information on how safe the medicine is and how well it works over a longer period. It also allows patients to keep taking Olezarsen until it might become more widely available.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Ionis Pharmaceuticals, Inc.

Enrolment target

Start

18 Nov 2021

Estimated completion

01 Feb 2028

Familial Chylomicronemia Syndrome

What is this study about?

This study, called "A Study of Olezarsen in Participants With Familial Chylomicronemia Syndrome (FCS)", is focused on a genetic condition where the body has trouble breaking down certain fats, leading to very high levels of triglycerides in the blood. This can cause severe health issues like inflammation of the pancreas. The study is investigating a new medicine named Olezarsen, which aims to help lower these triglyceride levels.

The main goal of this particular study is to continue observing how Olezarsen affects triglyceride levels over time. It's an extension of a previous study, meaning it's designed for people who have already completed the initial trial. By continuing the treatment for a longer period, researchers can get a much clearer picture of its long-term safety and how well it continues to work.

Gathering more information from this extended study is important for several reasons. It helps researchers understand the medicine better before it might be considered for wider use. It also gives patients who found the treatment helpful a chance to keep taking Olezarsen while it's still being studied.

Key takeaways

The study is for people with Familial Chylomicronemia Syndrome (FCS).
It's testing a medicine called Olezarsen to lower high triglyceride levels.
Only participants from a previous Olezarsen study can join this one.
The study aims to gather more information on the long-term safety and effects of the medicine.
Participation lasts for about 201 weeks (nearly four years).
It provides continued access to Olezarsen for eligible patients.

Who may be eligible?

This study is specifically designed for adults aged 18 and over, of any gender, who have Familial Chylomicronemia Syndrome (FCS).

To be considered for this study, you must have successfully completed the previous study involving Olezarsen (known as ISIS 678354-CS3). This means you received all your scheduled doses in the earlier study and your doctor confirmed that it was safe for you to continue.

Unfortunately, you wouldn't be able to join this study if you have developed any new health condition, or if an existing condition has worsened, in a way that your doctor believes would make it unsafe for you or would stop you from taking part fully.

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 Familial Chylomicronemia Syndrome (FCS)?
Did you complete the previous Olezarsen study (NCT04568434) fully?
Did your doctor say it was safe for you to continue with the treatment after the previous study?
Have you developed any new health problems that might prevent you from taking part?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, the total time you'd be involved is about 201 weeks, which is roughly just under four years. This includes a short period of up to 31 days to confirm you're eligible.

Once you're in the study, you'll receive the Olezarsen medicine for about 157 weeks, which is just over three years. During this time, you'll have regular visits for medical checks and blood tests to see how you're doing and how the medicine is affecting you. After your treatment period ends, there will be a follow-up period of 13 weeks (about three months) to make sure everything is well and to gather any final information.

Potential risks and benefits

Taking part in this study may offer the potential benefit of continuing to receive Olezarsen, which could help manage your triglyceride levels if it was effective for you in the previous study. This could be particularly welcome until the medicine might become more broadly available. As with any medicine, there is always a chance of side effects, some of which you may have already experienced in the earlier study, and new ones could still emerge with longer use. Your health will be closely monitored throughout the study. Remember, your participation is completely voluntary, and you can withdraw from the study at any time without giving a reason, and this will not affect your medical care.

Locations (27)

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

Diabetes/Lipid Management & Research Center
Verified postcode
Huntington Beach, United States
Excel Medical Clinical Trials, LLC
Verified postcode
Boca Raton, United States
Department of Pharmacy
Verified postcode
Park Ridge, United States
University of Kansas Medical Center (KUMC)
Verified postcode
Kansas City, United States
CTSI Investigational Pharmacy
Verified postcode
New York, United States
Milstein Hospital
Verified postcode
New York, United States
IDS Central
Verified postcode
Philadelphia, United States
Ecogene-21
Verified postcode
Chicoutimi, Canada
Institute de Recherches Cliniques de Montreal
Verified postcode
Montreal, Canada
Nathalie Saint-Pierre
Verified postcode
Montreal, Canada
Clinique des Maladies Lipidiques de Quebec Inc.
Verified postcode
Québec, Canada
Groupement Hospitalier Est- Pharmacie Secteur Essais Clinique
Verified postcode
Bron, France

Common questions

What is Familial Chylomicronemia Syndrome (FCS)?

FCS is a rare genetic condition where your body has trouble breaking down certain fats, leading to very high levels of triglycerides in your blood. This can cause serious health problems.

What is Olezarsen?

Olezarsen is a new experimental medicine being tested to help lower very high triglyceride levels in people with FCS.

Who can join this study?

This study is for adults over 18 who have FCS and have successfully completed the previous Olezarsen study (ISIS 678354-CS3) and were found suitable to continue by their doctor.

How long will the study last?

If you join, you would be involved for about 201 weeks in total, which is nearly four years. This includes treatment time and follow-up after the treatment stops.

Why is this extended study happening?

This study is being done to gather more information on the long-term safety and effectiveness of Olezarsen, and to allow participants to continue receiving the medicine until it might become available outside of trials.