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Hyperchylomicronaemia – Trials in Disease

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Ongoing Clinical Trials for Hyperchylomicronaemia

There are currently 4 ongoing clinical trials investigating treatments for hyperchylomicronaemia, a rare genetic disorder affecting the body’s ability to break down fats. These studies are testing new medications designed to reduce dangerously high levels of triglycerides in the blood, with trials taking place across multiple European countries.

Clinical trial locations

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

This trial is testing a medication called AKCEA-APOCIII-LRx (also known as ISIS 678354) in patients with Familial Chylomicronemia Syndrome who have previously been treated with a drug called volanesorsen. The medication is given as an injection under the skin.

Who can join:

  • Patients with Familial Chylomicronemia Syndrome, confirmed either through clinical evaluation or genetic testing
  • Patients who are currently on or have previously taken volanesorsen (ISIS 304801)
  • In countries where Waylivra (another name for volanesorsen) is available, patients should only join if they had to stop this treatment due to side effects
  • Both men and women can participate

Who cannot join:

  • Patients who have never been treated with volanesorsen
  • Those with other serious medical conditions that could interfere with the study
  • Pregnant or breastfeeding women
  • People unable to follow study procedures or attend visits
  • Those with a history of allergic reactions to similar medications
  • Anyone currently in another clinical trial
  • People with a history of drug or alcohol abuse
  • Those with certain blood disorders, bleeding problems, severe liver or kidney disease, or uncontrolled high blood pressure

What the study focuses on: The main goal is to evaluate the safety and tolerability of ISIS 678354. Researchers will monitor how well patients tolerate the treatment and track any side effects, particularly changes in platelet count, kidney function, liver enzymes, and any bleeding events. The study will also measure changes in blood fat levels, including triglycerides and cholesterol, throughout the treatment period.

Treatment being tested: AKCEA-APOCIII-LRx is an antisense oligonucleotide that works by reducing the production of apolipoprotein C-III, a protein involved in fat metabolism. The treatment is given through regular injections under the skin.

Study of ISIS 678354 for Patients with Severe High Triglycerides

This trial is investigating olezarsen (ISIS 678354) for people with severe hypertriglyceridemia, a condition where there are very high levels of triglycerides (a type of fat) in the blood. The study compares olezarsen to a placebo (inactive substance) to see how well it reduces triglyceride levels.

Who can join:

  • Patients with severe hypertriglyceridemia
  • Fasting triglyceride levels of at least 500 mg/dL (5.65 mmol/L) at two specific measurement times
  • Currently taking lipid-lowering medications according to local health guidelines, with stable doses for at least 4 weeks before the study
  • Willing to follow specific diet and lifestyle recommendations
  • Both men and women can participate

Who cannot join: The trial excludes patients with severe hypertriglyceridemia who do not meet the specific criteria outlined above.

What the study focuses on: The primary goal is to see if olezarsen can lower fasting triglyceride levels compared to placebo. The study will also monitor other blood fat levels, such as different types of cholesterol, and track any health complications. Participants will be followed for approximately 53 weeks with regular health check-ups.

Treatment being tested: Olezarsen is a lipid-lowering agent given as an injection under the skin. It works by targeting specific molecules in the body to reduce triglyceride production, helping to lower fat levels in the bloodstream.

Study of Olezarsen (ISIS 678354) in Patients with Severe Hypertriglyceridemia: Testing Effects on Triglyceride Levels

This trial tests olezarsen (ISIS 678354) in patients with severe hypertriglyceridemia over a 12-month period. The medication is an antisense oligonucleotide that influences how genes work to reduce fat levels in the blood.

Who can join:

  • Adults aged 18 years or older
  • High triglyceride levels of at least 500 mg/dL when measured after fasting
  • Currently taking lipid-lowering medications according to standard medical guidelines
  • Stable dose of these medications for at least 4 weeks before screening
  • Both men and women can participate
  • Willing to continue current lipid-lowering medications throughout the study
  • Able to provide fasting blood samples

Who cannot join:

  • People below 18 or above 75 years of age
  • Pregnant or breastfeeding women
  • Recent history of acute pancreatitis (within 6 months)
  • Use of medications that significantly affect triglyceride levels
  • Uncontrolled diabetes or severe kidney or liver disease
  • Recent heart attack or stroke (within 6 months)
  • Active cancer or recent cancer treatment (within 5 years)
  • Recent participation in another trial (within 30 days)
  • Known allergies to the study medication
  • Alcohol or substance abuse within the past year
  • Mental conditions or unstable medical conditions that could affect study participation

What the study focuses on: The study measures how well olezarsen reduces triglyceride levels compared to placebo over 12 months. Researchers will also track changes in specific proteins involved in fat processing, different types of cholesterol, and liver fat content. Safety monitoring includes regular blood tests and health assessments.

Treatment being tested: Olezarsen represents a new approach to treating high triglyceride levels. It is an antisense oligonucleotide given by injection under the skin, targeting genetic factors that contribute to fat metabolism problems.

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

This trial studies ISIS 678354 (AKCEA-APOCIII-LRx) in patients with Familial Chylomicronemia Syndrome who have completed a previous study. The medication is delivered through a device called YpsoMate, a single-use injection system.

Who can join:

  • Patients who have completed the previous study called ISIS 678354 CS3, including the last dose at Week 49
  • Must have an acceptable safety profile according to the study doctor’s judgment
  • Willing to follow a diet with no more than 20 grams of fat per day during the study
  • Both men and women can participate

Who cannot join:

  • Patients with conditions other than Familial Chylomicronemia Syndrome
  • Those not willing to follow study procedures or take the medication
  • People with other medical conditions that might interfere with the study
  • Pregnant or breastfeeding women
  • Those taking certain medications that might interfere with the study
  • Recent participants in other clinical trials
  • People with a history of not following medical treatments

What the study focuses on: The main goal is to evaluate how ISIS 678354 affects fasting triglyceride levels over time. The study will monitor changes at 6, 12, 24, and 36 months. Researchers will also assess the safety and tolerability of the treatment and track the occurrence of acute pancreatitis (inflammation of the pancreas) during the treatment period.

Treatment being tested: AKCEA-APOCIII-LRx is an antisense oligonucleotide given as an injection under the skin. It works by targeting and reducing the production of apolipoprotein C-III, a protein involved in fat metabolism, to help lower triglyceride levels.

Summary

All four clinical trials focus on testing medications to reduce dangerously high levels of triglycerides in the blood. Three trials investigate the same medication under different names (olezarsen, ISIS 678354, or AKCEA-APOCIII-LRx), while one specifically targets patients who have already used volanesorsen.

The trials are widely distributed across Europe, with Sweden being the only country participating in all four studies. France, Italy, the Netherlands, Portugal, Slovakia, and Spain are involved in three trials each, showing significant research activity in these regions. The concentration of trials in Western and Northern European countries suggests these regions have established research infrastructure for rare metabolic disorders.

All studies use similar treatment methods, with medications delivered through injections under the skin. The trials require participants to have very high triglyceride levels (at least 500 mg/dL in most cases) and typically last between 12 and 36 months. Common safety concerns across all trials include monitoring blood cell counts, kidney and liver function, and the risk of pancreatitis.

The investigational drug being tested works by targeting apolipoprotein C-III, a protein involved in fat metabolism, representing a novel approach to treating these difficult-to-manage conditions. While these trials offer hope for new treatment options, they are still in the research phase and outcomes are not yet known.

Ongoing Clinical Trials on Hyperchylomicronaemia

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