RecruitingRare disease

Study of ISIS 678354 for Patients with Familial Chylomicronemia Syndrome Previously Treated with Volanesorsen

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 condition called Familial Chylomicronemia Syndrome (FCS). FCS is a genetic disorder that affects the body's ability to break down fats, leading to high levels of fat in the blood. The study involves a treatment called AKCEA-APOCIII-LRx, also known by its code name ISIS 678354. This treatment is given as an injection under the skin and is being tested in patients who have previously been treated with another medication called Volanesorsen (ISIS 304801).

The purpose of the study is to evaluate the safety and tolerability of ISIS 678354 in patients with FCS. Participants will receive the treatment over a period of time, and researchers will monitor them for any changes in their health. The study will look at how well patients tolerate the treatment and any side effects they may experience. This includes checking for changes in blood components like platelets and kidney function, as well as any bleeding events.

Throughout the study, researchers will also measure the levels of certain fats in the blood, such as triglycerides and cholesterol, to see how they change with the treatment. The study will last for several months, with regular check-ups to ensure the safety and well-being of the participants. The ultimate goal is to gather information that could help improve treatment options for people living with FCS.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, the patient will begin treatment with ISIS 678354, which is administered as an injection under the skin (subcutaneous).
The study is designed for patients with Familial Chylomicronemia Syndrome (FCS) who have been previously treated with a medication called volanesorsen.
Step 2
Treatment administration
The patient will receive ISIS 678354 injections at regular intervals. The specific dosage and frequency will be determined by the study protocol and the patient's individual needs.
The treatment aims to evaluate the safety and tolerability of ISIS 678354 over a period of time.
Step 3
Monitoring and assessments
Throughout the study, the patient will undergo regular monitoring to assess safety and tolerability. This includes checking for any changes in platelet count, kidney function, and liver enzymes.
The study will also monitor for any adverse events or side effects that may occur during the treatment period.
Step 4
Primary and secondary endpoints
The primary endpoints focus on safety, including changes in platelet count and kidney function, as well as any major or clinically relevant bleeding events.
Secondary endpoints include measuring the concentration of ISIS 678354 in the blood and changes in various lipid levels, such as triglycerides and cholesterol.
Step 5
Study duration
The study is expected to continue until March 1, 2024, with key assessments occurring at weeks 53, 105, and 157.
The patient will be required to attend regular study visits to ensure comprehensive monitoring and data collection.

Who can join the trial?

6 criteria

The patient must have Familial Chylomicronemia Syndrome (FCS), which is a condition that can be diagnosed through clinical evaluation or genetic testing.
The patient should currently be on or have previously been treated with volanesorsen, a medication also known as ISIS 304801.
If the patient is in a country where Waylivra (another name for volanesorsen) is available, they should not be deprived of this treatment unless it was stopped due to adverse events (AEs), which are unwanted side effects.
Other specific criteria defined in the study protocol must also be met.
Both male and female patients are eligible to participate.
The study includes patients who may be considered part of a vulnerable population, which means they might need special protection or consideration.

Who cannot join the trial?

10 criteria

Patients who have not been previously treated with a medication called volanesorsen.
Patients who have any other serious medical conditions that might interfere with the study.
Patients who are pregnant or breastfeeding.
Patients who are unable to follow the study procedures or attend study visits.
Patients who have a history of allergic reactions to similar medications.
Patients who are currently participating in another clinical trial.
Patients who have a history of drug or alcohol abuse.
Patients who have certain blood disorders or bleeding problems.
Patients who have severe liver or kidney disease.
Patients who have uncontrolled high blood pressure.

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Verified sites

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Karolinska University Hospital
Verified
Solna, Sweden

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Investigated drugs

AKCEA-APOCIII-LRX
is a medication being studied for its safety and tolerability in patients with Familial Chylomicronemia Syndrome (FCS). It is administered through an injection under the skin. This medication is designed to help manage the symptoms of FCS, a rare genetic disorder that affects the body's ability to break down fats.
Volanesorsen
is a medication that was previously used by patients in this study. It is also used to treat Familial Chylomicronemia Syndrome by reducing the levels of certain fats in the blood. The study aims to see how patients who have been treated with Volanesorsen respond to the new medication, AKCEA-APOCIII-LRX.

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 safety and tolerability in patients with Familial Chylomicronemia Syndrome (FCS) who have previously been treated with Volanesorsen. 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 manage FCS, a rare genetic disorder. At the molecular level, it works by reducing the production of apolipoprotein C-III, a protein that plays a role in lipid metabolism. It is classified pharmacologically as an antisense oligonucleotide.

Investigated diseases

Familial Chylomicronemia Syndrome – This is a rare genetic disorder characterized by the body's inability to break down fats properly, leading to extremely high levels of triglycerides in the blood. The condition is caused by mutations in genes responsible for the production or function of lipoprotein lipase, an enzyme crucial for fat metabolism. As a result, individuals with this syndrome often experience symptoms such as abdominal pain, recurrent episodes of pancreatitis, and eruptive xanthomas, which are small, yellowish skin lesions. Over time, the accumulation of chylomicrons, which are fat particles, can lead to complications affecting various organs. The disease typically manifests in childhood or early adulthood and requires ongoing management to prevent severe complications.