Not recruitingRare disease

Study on the Effects of ARO-APOC3 for Adults with Familial Chylomicronemia Syndrome

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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 condition known as Familial Chylomicronemia Syndrome (FCS). FCS is a disorder that affects the body's ability to break down fats, leading to extremely high levels of triglycerides in the blood. The study will evaluate a new treatment called ARO-APOC3, which is a solution for injection. This treatment is a synthetic molecule designed to target and reduce the production of a specific protein involved in fat metabolism, known as apolipoprotein C-III.

The purpose of the study is to assess how effective and safe ARO-APOC3 is for adults with FCS. Participants in the study will receive either the new treatment or a placebo, which is a substance with no active medication. The study will involve regular injections and monitoring over a period of time to observe changes in triglyceride levels and any potential side effects.

Throughout the study, participants will have their triglyceride levels checked at various intervals to see how they respond to the treatment. The study aims to provide valuable information on whether ARO-APOC3 can help manage FCS by lowering triglyceride levels and improving overall health outcomes for those affected by this condition.

The research process

The trial runs in 7 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, eligibility is confirmed based on specific criteria such as age and medical condition. Participants must be adults diagnosed with Familial Chylomicronemia Syndrome (FCS) and have a fasting triglyceride level of at least 10 mmol/L (880 mg/dL) that does not respond to standard lipid-lowering therapy.
Step 2
Initial assessment
An initial assessment is conducted to establish baseline measurements. This includes measuring fasting triglyceride levels and other relevant health indicators.
Step 3
Treatment administration
Participants receive the study medication, ARO-APOC3, which is a synthetic double-stranded siRNA oligonucleotide. It is administered as a subcutaneous injection. The exact dosage and frequency are determined by the study protocol.
Step 4
Ongoing monitoring
Throughout the study, regular monitoring is conducted to assess the efficacy and safety of the treatment. This includes periodic measurements of fasting triglyceride levels and other health parameters.
Step 5
Evaluation of primary endpoint
The primary endpoint is evaluated by measuring the percent change from baseline in fasting triglyceride levels at Month 10.
Step 6
Evaluation of secondary endpoints
Secondary endpoints include the percent change from baseline in fasting triglyceride levels at Months 10 and 12 (averaged), and the percent change in fasting APOC3 levels at Months 10 and 12.
Step 7
Completion of study
The study is estimated to conclude by May 3, 2026. Final assessments are conducted to gather comprehensive data on the treatment's efficacy and safety.

Who can join the trial?

3 criteria

Men and women who are not pregnant and are at least 18 years old. In some places, you need to be at least 19 years old.
Have a fasting triglyceride level of at least 10 mmol/L (which is the same as 880 mg/dL). This level should not improve with standard treatments that lower fats in the blood.
Have a diagnosis of Familial Chylomicronemia Syndrome (FCS), which is a specific medical condition.

Who cannot join the trial?

3 criteria

Patients who are not adults cannot participate. This means only people who are 18 years or older can join.
Patients who do not have **Familial Chylomicronemia Syndrome (FCS)** cannot participate. FCS is a rare genetic disorder that affects how the body processes fats.
Patients who are part of a vulnerable population may not be eligible. Vulnerable populations include groups like pregnant women, children, or people with certain disabilities.

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Verified sites

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

KBC Zagreb
Responsive
Zagreb, Croatia
Katholieke Universiteit te Leuven
Responsive
Leuven, Belgium
Universitair Ziekenhuis Gent
Responsive
Gent, Belgium

Trial locations

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Investigated drugs

ARO-APOC3 is a medication being studied for its effectiveness and safety in treating adults with Familial Chylomicronemia Syndrome (FCS). This condition is a rare genetic disorder that affects the body's ability to break down fats, leading to high levels of triglycerides in the blood. The medication aims to reduce these triglyceride levels, potentially improving symptoms and reducing the risk of complications associated with FCS.

Synthetic Double-Stranded Sirna Oligonucleotide Directed Against Apolipoprotein C-Iii Mrna And Covalently Linked To A Ligand Containing Three N-Acetylgalactosamine Residues

What is already known about the treatment

ARO-APOC3 – This medication is administered via injection and is currently being studied in clinical trials for its effectiveness and safety in treating Familial Chylomicronemia Syndrome (FCS). It is not yet widely available in medical practice, as it is still under investigation. The main therapeutic indication for ARO-APOC3 is to manage FCS, a rare genetic disorder that affects fat metabolism. At the molecular level, ARO-APOC3 works by targeting and reducing the production of a specific protein involved in fat metabolism, which helps lower triglyceride levels in the blood. It is classified pharmacologically as an antisense oligonucleotide therapy.

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 the production or function of lipoprotein lipase, an enzyme crucial for fat metabolism. Individuals with this syndrome often experience symptoms such as abdominal pain, recurrent episodes of pancreatitis, and eruptive xanthomas, which are small, yellowish skin lesions. 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 control triglyceride levels.