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

1 1 1

What is this study about?

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.

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.

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.

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.

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 Study?

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 Study?

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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Azienda Ospedaliera Universitaria Federico II Di Napoli	Naples	Italy
Unidade Local De Saude De Lisboa Ocidental E.P.E.	Carnaxide	Portugal

Other Sites

Site Name	City	Country
Hospital Universitario 12 De Octubre	Madrid	Spain
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone	Palermo	Italy
Unidade Local De Saude Do Alto Ave E.P.E.	Guimaraes	Portugal
Virgen del Rocío University Hospital	Sevilla	Spain
Hospital Clinic De Barcelona	Barcelona	Spain
Centre Hospitalier Lyon Sud	Pierre Benite	France
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen	Gothenburg	Sweden
Mgzdkenwrxp cjquiwa Kxmmfmui Rdytqnea sfxldl	Bratislava	Slovakia
Clgoax Hsbbxizryjo Uqcxcibsuhgdk Ds Djpzj	Dijon	France
Autfbztuh Uts	Amsterdam	The Netherlands
Aqumojshaq Pvmfaguf Hspqsuvm Dy Mliwiaegd	Marseille	France
Uscurhybqy Dhkug Snead Do Rido Ls Sgqgyhso	Rome	Italy

Trial status

Country	Status	Recruitment Start
France	Not recruiting	01.07.2022
Italy	Not recruiting	01.07.2022
Portugal	Not recruiting	01.07.2022
Slovakia	Not recruiting	01.07.2022
Spain	Not recruiting	01.07.2022
Sweden	Not recruiting	01.07.2022
The Netherlands	Not recruiting	01.07.2022

Trial locations

Investigated drugs:

Isis 678354 Sodium Salt

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.

Investigated diseases:

Familial hypertriglyceridaemia

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.

Trial ID:

2023-509029-29-00

Protocol code:

ISIS 678354-CS13

NCT ID:

NCT05130450

Trial Phase:

Therapeutic confirmatory (Phase III)

Other Trials to Consider

#1 Clinical Trials Search in Europe

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

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?