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Doconexent

This article discusses a clinical trial investigating the use of Doconexent, a component of a fat emulsion, in pediatric patients with Long-chain Fatty Acid Oxidation Disorders (LC-FAOD). The study aims to compare the effects of triheptanoin, which contains Doconexent, with medium-chain triglycerides (MCT) on major clinical events in children with LC-FAOD. This research is crucial for understanding potential treatments for this rare metabolic condition.

Table of Contents

What is DOCONEXENT?

DOCONEXENT, also known as docosahexaenoic acid or DHA, is a type of omega-3 fatty acid that is being studied as a potential treatment for children with Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)[1]. It is part of a combination medication that includes other substances such as glycerol, icosapent (another omega-3 fatty acid), egg lecithin, and vitamin E (DL-alpha-tocopherol).

What are Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)?

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) are a group of rare genetic conditions that affect how the body breaks down fats for energy[1]. People with LC-FAOD have difficulty converting long-chain fatty acids into energy, which can lead to various health problems, especially during times of illness, fasting, or increased energy demands.

Some types of LC-FAOD include:

  • Carnitine palmitoyl transferase (CPT) I deficiency
  • CPT II deficiency
  • Carnitine/acylcarnitine translocase (CACT) deficiency
  • Very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency
  • Long-chain 3-hydroxyacyl-CoA dehydrogenase (LCHAD) deficiency
  • Mitochondrial trifunctional protein (TFP) deficiency

Clinical Trial Overview

A clinical trial is being conducted to evaluate the effectiveness of a medication containing DOCONEXENT (along with other substances) compared to medium-chain triglycerides (MCT) in children with LC-FAOD[1]. This study is:

  • Randomized: Participants are randomly assigned to either the DOCONEXENT group or the MCT group
  • Double-blind: Neither the participants nor the researchers know which treatment each child is receiving
  • Multicenter: The study is being conducted at multiple hospitals or research centers

Who Can Participate in the Study?

The study is designed for children with LC-FAOD. Some key eligibility criteria include[1]:

  • Children from birth to less than 18 years old
  • Confirmed diagnosis of LC-FAOD
  • Significant clinical symptoms of LC-FAOD, such as:
    • At least 2 severe episodes in the past year (or 3 in the past 2 years) requiring emergency care or hospitalization
    • Recurrent symptomatic hypoglycemia (low blood sugar)
    • Susceptibility to hypoglycemia after short fasting periods
    • Evidence of heart problems (cardiomyopathy) requiring ongoing medical management

There are additional criteria for participation, and some conditions may exclude a child from the study. It’s important to discuss with your doctor if you think your child might be eligible.

Study Objectives

The main goal of this study is to evaluate how well the DOCONEXENT-containing medication works compared to MCT in reducing the frequency of major clinical events (MCEs)[1]. MCEs are serious health problems that can occur in children with LC-FAOD, such as:

  • Rhabdomyolysis: A condition where muscle tissue breaks down rapidly, which can be dangerous for the kidneys
  • Cardiomyopathy: Heart muscle disease that can affect the heart’s ability to pump blood effectively
  • Hypoglycemia: Dangerously low blood sugar levels

The study will also look at other important factors, such as[1]:

  • How long these major clinical events last
  • Changes in overall health status and functioning
  • Effects on heart function
  • Impact on quality of life
  • Safety of the medication

Potential Benefits of DOCONEXENT

While the study is still ongoing and results are not yet known, researchers hope that the DOCONEXENT-containing medication may offer several potential benefits for children with LC-FAOD, such as[1]:

  • Reducing the frequency and duration of major clinical events
  • Improving overall health and functioning
  • Reducing the frequency of hypoglycemic events
  • Potentially improving heart function in children with cardiomyopathy
  • Enhancing quality of life for children with LC-FAOD and their families

Safety Considerations

As with any medical study, there are important safety considerations to keep in mind[1]:

  • The study will carefully monitor for any side effects or adverse events
  • Participants will be closely followed by medical professionals throughout the study
  • Some children may not be eligible to participate if they have certain medical conditions or are taking specific medications
  • The study drug’s long-term effects are not yet fully known

It’s crucial for parents and caregivers to discuss all potential risks and benefits with their child’s doctor before considering participation in this or any clinical trial.

Aspect Details
Study Type Randomized, double-blind, multicenter study
Participants Pediatric patients (0 to <18 years) with LC-FAOD
Intervention Triheptanoin (containing Doconexent) vs. Medium-chain Triglycerides (MCT)
Primary Outcome Annualized rate of Major Clinical Events (MCEs)
Secondary Outcomes Duration of MCEs, hypoglycemic events, cardiomyopathy events, quality of life measures
Treatment Duration Up to 24 months
Additional Component Liver Sub-study for participants >2 years old

FAQ

  • What is the main purpose of this clinical trial? The main purpose of this clinical trial is to evaluate the effect of triheptanoin (which contains Doconexent) compared to medium-chain triglycerides (MCT) on the frequency of major clinical events in pediatric patients with Long-chain Fatty Acid Oxidation Disorders (LC-FAOD).
  • Who can participate in this clinical trial? The trial is open to male and female patients from birth to under 18 years of age with a confirmed diagnosis of LC-FAOD. Participants must have significant clinical manifestations of LC-FAOD, such as severe episodes of metabolic decompensation or evidence of functional cardiomyopathy.
  • What are the primary outcomes being measured in this study? The primary outcome being measured is the annualized event rate of major clinical events (MCEs) during the double-blind treatment period. This will help determine if triheptanoin is more effective than MCT in reducing these events.
  • How long does the treatment period last? The maximum treatment period for this clinical trial is 24 months.
  • Are there any additional studies within this clinical trial? Yes, there is a Liver Sub-study for participants over 2 years of age with specific liver fat content criteria. This sub-study will evaluate the effect of triheptanoin versus MCT on hepatic lipids using specialized imaging techniques.

Ongoing Clinical Trials on Doconexent

  • Study on the Effects of Triheptanoin and Medium-chain Triglycerides in Children with Long-chain Fatty Acid Oxidation Disorders

    Not recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Czechia Germany Poland Spain

Glossary

  • Long-chain Fatty Acid Oxidation Disorders (LC-FAOD): A group of rare genetic metabolic disorders where the body is unable to properly break down long-chain fatty acids for energy, leading to various health complications.
  • Triheptanoin: An investigational drug being studied for its potential to treat LC-FAOD. It contains Doconexent and is compared to medium-chain triglycerides in this trial.
  • Medium-chain Triglycerides (MCT): A type of fat that is more easily digested and absorbed by the body, often used in the management of LC-FAOD.
  • Major Clinical Events (MCEs): Significant health episodes related to LC-FAOD, such as rhabdomyolysis, cardiomyopathy, and hypoglycemia, which are being measured in this study.
  • Rhabdomyolysis: A serious condition resulting from the breakdown of damaged muscle tissue, which can lead to kidney damage.
  • Cardiomyopathy: A group of diseases affecting the heart muscle, making it harder for the heart to pump blood to the rest of the body.
  • Hypoglycemia: Low blood sugar levels, which can be a dangerous complication in patients with LC-FAOD.
  • Double-blind study: A research design where neither the participants nor the researchers know which treatment is being administered, reducing potential bias.
  • Proton Magnetic Resonance Spectroscopy (1H-MRS): An imaging technique used in the Liver Sub-study to measure liver fat content non-invasively.
  • Doconexent: Also known as docosahexaenoic acid (DHA), it is an omega-3 fatty acid and a component of the investigational drug being studied in this trial.

References

  1. http://clinicaltrials.eu/trial/study-on-the-effects-of-triheptanoin-and-medium-chain-triglycerides-in-children-with-long-chain-fatty-acid-oxidation-disorders/