Not recruitingRare disease

Study on the Effects of ISIS 678354 in Patients with Familial Chylomicronemia Syndrome (FCS)

Verified siteInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare genetic disorder called Familial Chylomicronemia Syndrome (FCS). FCS is a condition that affects the body's ability to break down fats, leading to very high levels of fat in the blood. The treatment being tested in this study is an injection called ISIS 678354, also known as AKCEA-APOCIII-LRx. This medication is designed to help reduce the levels of triglycerides, which are a type of fat found in the blood.

The purpose of the study is to evaluate how well ISIS 678354 works in lowering triglyceride levels in patients with FCS. Participants in the study will receive the medication through a small injection under the skin, using a device called YpsoMate. This device is a single-use, disposable injection system that helps deliver the medication safely and effectively. The study will monitor changes in triglyceride levels over time, as well as the safety and tolerability of the treatment.

Throughout the study, participants will have their triglyceride levels checked at various intervals, such as at 6 months, 12 months, 24 months, and 36 months. The study will also look at other factors, like changes in other blood fats and the occurrence of any side effects. The goal is to see if the treatment can help achieve significant reductions in triglyceride levels and improve the overall health of patients with Familial Chylomicronemia Syndrome.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Participation begins after satisfactory completion of the previous study, ISIS 678354 CS3, with the last dose administered at Week 49. The safety profile must be acceptable according to the investigator's judgment.
A commitment to follow a diet with no more than 20 grams of fat per day is required during the study.
Step 2
Treatment administration
The medication used in this study is ISIS 678354, administered as an injection under the skin (subcutaneous use).
The primary goal is to evaluate the effect of ISIS 678354 on the change in fasting triglycerides (TG) levels from the start of the study.
Step 3
Monitoring and evaluation
The study will assess the safety and tolerability of the treatment with ISIS 678354.
Secondary evaluations include changes in fasting triglycerides and other blood components at various intervals: Month 6, 12, 24, and 36.
The study will also monitor the occurrence of acute pancreatitis events during the treatment period.
Step 4
Study duration
The study is expected to conclude by October 1, 2025.
The recruitment for the study began on July 1, 2022.

Who can join the trial?

4 criteria

Must have completed the previous study called ISIS 678354 CS3, including the last dose at Week 49, and have a safety profile that is considered acceptable by the study doctor.
Must be willing to follow a diet that includes **20 grams of fat or less** per day during the study.
Both **men and women** can participate.
Participants can be from a **vulnerable population**, which means they might need extra protection or care.

Who cannot join the trial?

9 criteria

Patients who have a different condition than Familial Chylomicronemia Syndrome (FCS) cannot participate. FCS is a rare genetic disorder that affects how the body processes fats.
Patients who are not within the specified age range cannot participate. The age range for this study is not specified here, but it is important for eligibility.
Patients who are not part of the specified clinical trial group cannot participate. This means the study is looking for a specific group of people based on certain characteristics.
Patients who are not willing to follow the study procedures or take the study medication cannot participate.
Patients who have other medical conditions that might interfere with the study cannot participate. This is to ensure the safety of the participants and the accuracy of the study results.
Patients who are pregnant or breastfeeding cannot participate. This is to protect the health of the mother and the baby.
Patients who are taking certain medications that might interfere with the study cannot participate. This is to avoid any interactions that could affect the study results.
Patients who have participated in another clinical trial recently cannot participate. This is to ensure that previous treatments do not affect the current study.
Patients who have a history of non-compliance with medical treatments cannot participate. This means they have not followed medical advice or treatment plans in the past.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Amsterdam UMC
Responsive
Amsterdam, The Netherlands
Unidade Local De Saude De Lisboa Ocidental E.P.E.
Responsive
Carnaxide, Portugal
Universita Degli Studi Di Roma La Sapienza
Responsive
Rome, Italy

Trial locations

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Investigated drugs

AKCEA-APOCIII-LRX is a medication being studied for its effects on patients with Familial Chylomicronemia Syndrome (FCS). This medication is administered through an injection under the skin. The main goal of the study is to see how this medication affects the levels of triglycerides, which are a type of fat found in the blood, when patients are fasting.

Isis 678354 Sodium Salt

What is already known about the treatment

AKCEA-APOCIII-LRX – This medication is administered through subcutaneous injection and is currently being studied in clinical trials for its effects on Familial Chylomicronemia Syndrome (FCS). It is not yet widely available in medical practice, as it is still under investigation. The main therapeutic indication for AKCEA-APOCIII-LRX is to reduce high levels of triglycerides in patients with FCS. At the molecular level, it works by targeting and reducing the production of apolipoprotein C-III, a protein that plays a role in lipid metabolism. This medication is classified as an antisense oligonucleotide, which is a type of drug designed to interfere with the expression of specific genes.

Investigated diseases

Familial Chylomicronemia Syndrome – This is a rare genetic disorder characterized by extremely high levels of triglycerides in the blood due to the body's inability to break down chylomicrons, which are particles that transport fats. The condition is caused by mutations in genes responsible for producing enzymes that help in fat metabolism. Individuals with this syndrome often experience symptoms such as abdominal pain, eruptive xanthomas (small, yellowish skin lesions), and recurrent episodes of pancreatitis. The disease typically manifests in childhood or early adulthood and can lead to complications if not managed properly. It is important for individuals with this condition to adhere to a strict low-fat diet to help manage symptoms.