Ongoing Clinical Trials for Familial Hypertriglyceridaemia
This article provides information about 5 ongoing clinical trials investigating new treatments for familial hypertriglyceridaemia, a genetic condition characterized by extremely high levels of triglycerides (fats) in the blood. These studies are testing injectable medications designed to reduce triglyceride levels and are being conducted across multiple European countries.
Clinical trial locations
- Austria
- Belgium
- Bulgaria
- Croatia
- Czechia
- Denmark
- Finland
- France
- Study of ISIS 678354 for Patients with Severe High Triglycerides
- Study of Olezarsen (ISIS 678354) in Patients with Severe Hypertriglyceridemia: Testing Effects on Triglyceride Levels
- Study on the Effects of ARO-APOC3 for Adults with Familial Chylomicronemia Syndrome
- Study on the Effects of ISIS 678354 in Patients with Familial Chylomicronemia Syndrome (FCS)
- Germany
- Greece
- Hungary
- Ireland
- Italy
- Lithuania
- Netherlands
- Norway
- Poland
- Portugal
- Romania
- Slovakia
- Spain
- Study of ISIS 678354 for Patients with Severe High Triglycerides
- Study of Olezarsen (ISIS 678354) in Patients with Severe Hypertriglyceridemia: Testing Effects on Triglyceride Levels
- Study on the Effects of ARO-APOC3 for Adults with Familial Chylomicronemia Syndrome
- Study on the Effects of ISIS 678354 in Patients with Familial Chylomicronemia Syndrome (FCS)
- Sweden
- Study of ISIS 678354 for Patients with Familial Chylomicronemia Syndrome Previously Treated with Volanesorsen
- Study of ISIS 678354 for Patients with Severe High Triglycerides
- Study of Olezarsen (ISIS 678354) in Patients with Severe Hypertriglyceridemia: Testing Effects on Triglyceride Levels
- Study on the Effects of ISIS 678354 in Patients with Familial Chylomicronemia Syndrome (FCS)
Study of ISIS 678354 for Patients with Familial Chylomicronemia Syndrome Previously Treated with Volanesorsen
This trial is investigating AKCEA-APOCIII-LRx (also known as ISIS 678354), a medication designed to help people with Familial Chylomicronemia Syndrome. This is a rare genetic condition that prevents the body from breaking down fats properly, leading to dangerously high triglyceride levels in the blood.
Who can participate:
- Adults diagnosed with Familial Chylomicronemia Syndrome through clinical evaluation or genetic testing
- People currently taking or previously treated with volanesorsen (another medication for this condition)
- Both men and women are eligible
Who cannot participate:
- People who have never been treated with volanesorsen
- Pregnant or breastfeeding women
- People with serious medical conditions that might interfere with the study
- Those with blood disorders, bleeding problems, severe liver or kidney disease, or uncontrolled high blood pressure
- People unable to follow study procedures or attend regular visits
What the study involves: The medication is given as an injection under the skin at regular intervals. Throughout the study, researchers will monitor safety by checking blood components like platelets and kidney function, watching for bleeding events, and measuring changes in triglyceride and cholesterol levels. The study will last several months with regular check-ups to ensure participant safety and well-being.
Study of ISIS 678354 for Patients with Severe High Triglycerides
This clinical trial is testing olezarsen (ISIS 678354) in people with severe hypertriglyceridemia, a condition where triglyceride levels in the blood are dangerously high. Elevated triglycerides increase the risk of heart disease and other serious health problems.
Who can participate:
- Adults with fasting triglyceride levels of at least 500 mg/dL measured twice during screening
- People currently taking lipid-lowering medications that have been stable for at least 4 weeks
- Those willing to follow specific diet and lifestyle recommendations
- Both men and women are eligible
Who cannot participate: People with severe hypertriglyceridemia who do not meet the specific triglyceride threshold requirements for the study.
What the study involves: Participants will receive either olezarsen or a placebo (inactive substance) as injections under the skin. The study lasts approximately 53 weeks and includes regular monitoring of triglyceride levels and other health markers at baseline, month 6, and month 12. Researchers will assess how effectively olezarsen reduces triglyceride levels compared to placebo, while participants continue their current lipid-lowering treatments and follow the prescribed diet.
Study of Olezarsen (ISIS 678354) in Patients with Severe Hypertriglyceridemia: Testing Effects on Triglyceride Levels
This study is testing olezarsen, a new type of medication called an antisense oligonucleotide that works by influencing how genes function in the body. The trial aims to determine how well olezarsen reduces triglyceride levels in patients with severe hypertriglyceridemia.
Who can participate:
- Adults aged 18 to 75 years
- People with fasting triglyceride levels of at least 500 mg/dL
- Those currently on stable lipid-lowering medications for at least 4 weeks
- Individuals able to provide fasting blood samples
- Both men and women
Who cannot participate:
- Pregnant or breastfeeding women
- People who had acute pancreatitis within the past 6 months
- Those with uncontrolled diabetes, severe kidney or liver disease
- People who had a heart attack or stroke within the past 6 months
- Those with active cancer or recent cancer treatment
- People with alcohol or substance abuse issues within the past year
- Anyone participating in another clinical trial within the past 30 days
What the study involves: Participants will be randomly assigned to receive either olezarsen or placebo through subcutaneous injections. The treatment continues for approximately 12 months, with regular measurements of triglyceride levels taken at weeks 25 and 27, and assessments of other blood components including apolipoprotein C-III, remnant cholesterol, and non-HDL cholesterol. Liver fat content will also be monitored throughout the study period.
Study on the Effects of ARO-APOC3 for Adults with Familial Chylomicronemia Syndrome
This trial focuses on ARO-APOC3, an investigational treatment for Familial Chylomicronemia Syndrome. ARO-APOC3 is designed to target and reduce the production of apolipoprotein C-III, a specific protein involved in fat metabolism.
Who can participate:
- Adults aged 18 years or older (19 years in some locations)
- People with a diagnosis of Familial Chylomicronemia Syndrome
- Those with fasting triglyceride levels of at least 10 mmol/L (880 mg/dL) that do not respond to standard lipid-lowering therapy
- Both men and women who are not pregnant
Who cannot participate: People without a diagnosis of Familial Chylomicronemia Syndrome or those who do not meet the age requirements.
What the study involves: After initial assessments to establish baseline triglyceride levels, participants will receive ARO-APOC3 as subcutaneous injections according to the study protocol. Regular monitoring throughout the study will assess treatment efficacy and safety. The primary evaluation measures the percent change in fasting triglyceride levels at Month 10, with additional assessments at Month 12. Secondary evaluations include changes in fasting APOC3 levels. The study is expected to conclude by May 2026.
Study on the Effects of ISIS 678354 in Patients with Familial Chylomicronemia Syndrome (FCS)
This trial is studying AKCEA-APOCIII-LRx (ISIS 678354) in patients with Familial Chylomicronemia Syndrome, a rare genetic disorder that prevents proper fat breakdown. The medication is designed to reduce triglyceride levels in the blood.
Who can participate:
- People who successfully completed a previous study (ISIS 678354 CS3) with an acceptable safety profile
- Those willing to follow a strict diet with no more than 20 grams of fat per day
- Both men and women
Who cannot participate:
- People with conditions other than Familial Chylomicronemia Syndrome
- Pregnant or breastfeeding women
- Those unwilling to follow study procedures or take the study medication
- People with other medical conditions that might interfere with the study
- Those taking certain medications that could affect study results
- People who recently participated in another clinical trial
- Individuals with a history of not following medical advice or treatment plans
What the study involves: Treatment is administered as injections under the skin using a single-use device called YpsoMate. The study monitors changes in fasting triglyceride levels at multiple intervals: 6, 12, 24, and 36 months. Researchers will also assess the safety and tolerability of the treatment, measure changes in other blood fats, and monitor for any side effects. The study is expected to conclude by October 2025.
Summary
All five clinical trials focus on developing new injectable treatments for familial hypertriglyceridaemia, primarily testing two main investigational drugs: olezarsen (also known as ISIS 678354 or AKCEA-APOCIII-LRx) and ARO-APOC3. Both medications work by targeting specific proteins involved in fat metabolism to reduce triglyceride levels in the blood.
The trials are widely distributed across Europe, with the highest concentration of studies taking place in Sweden, France, Spain, and Italy. Many countries, including the Netherlands, Portugal, Slovakia, and Poland, are participating in multiple trials, providing broader access to these experimental treatments.
Most studies require participants to have severely elevated triglyceride levels, typically at least 500 mg/dL or higher, that do not respond adequately to standard treatments. The trials range from approximately 12 months to 36 months in duration, with regular monitoring of triglyceride levels and safety assessments throughout the treatment period. All participants must maintain their current lipid-lowering medications and follow specific dietary guidelines during the studies.
These trials represent important research into rare genetic conditions affecting fat metabolism, offering hope for improved treatment options for patients who have limited alternatives with currently available therapies.
