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

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What is this study about?

This clinical trial is focused on studying a group of rare genetic conditions known as Long-chain Fatty Acid Oxidation Disorders (LC-FAOD). These disorders affect the body’s ability to break down certain fats into energy, especially during periods without food. The study will compare the effects of a treatment called Triheptanoin, also known by its code name UX007 and marketed as DOJOLVI®, with another treatment involving Even-chain, Medium-chain Triglycerides (MCT). Triheptanoin is an oral liquid that provides an alternative energy source for patients with LC-FAOD.

The purpose of the study is to evaluate how Triheptanoin affects the frequency of major clinical events, such as episodes of low blood sugar or muscle breakdown, in children with LC-FAOD. Participants in the study will be randomly assigned to receive either Triheptanoin or MCT. The study will be conducted in a double-blind manner, meaning neither the participants nor the researchers will know which treatment is being given to each participant. This helps ensure that the results are not biased.

The study will last for up to 24 months, during which participants will take the assigned treatment orally. Throughout the study, participants will be monitored for any changes in their condition, including the occurrence of major clinical events. The study aims to provide valuable information on the effectiveness of Triheptanoin in managing LC-FAOD and improving the quality of life for affected individuals.

1 joining the study

Upon joining the study, the patient will be randomly assigned to receive either triheptanoin or medium-chain triglycerides (MCT).

The study aims to evaluate the effect of triheptanoin on major clinical events in patients with long-chain fatty acid oxidation disorders (LC-FAOD).

2 medication administration

The patient will take the assigned medication orally in the form of a liquid.

The dosage and frequency will be determined by the study protocol and adjusted as necessary by the study team.

3 monitoring and assessments

Throughout the study, the patient will undergo regular monitoring to assess the frequency and duration of major clinical events.

Additional assessments may include evaluations of heart function and liver fat content, depending on the patient’s participation in specific sub-studies.

4 completion of the study

The study is expected to continue until June 2027.

Upon completion, the patient’s health and response to the medication will be thoroughly evaluated to determine the overall effect of the treatment.

Who Can Join the Study?

Have a confirmed diagnosis of Long-chain Fatty Acid Oxidation Disorders (LC-FAOD). This includes specific conditions like 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, and mitochondrial trifunctional protein (TFP) deficiency. The diagnosis must be confirmed by specific medical tests or genetic analysis.
Be a male or female from birth (including newborns) up to less than 18 years old.
Have a caregiver who is willing and able to help with all study requirements.
Have a legally authorized representative who can provide written consent after understanding the study details. The participant should also be able to give written agreement appropriate for their age.
Have at least one of the following significant health issues related to LC-FAOD:
- At least 2 severe episodes of metabolic decompensation (such as low blood sugar, muscle breakdown, or worsening heart problems) in the past year, or 3 in the past 2 years, requiring emergency or hospital care.
- Repeated low blood sugar symptoms needing treatment.
- Prone to low blood sugar after short fasting periods (less than 4 to 12 hours, depending on age).
- Heart problems needing ongoing medical care or signs of heart failure.
- A sibling with the same genetic variant who has had major clinical events (MCEs).
- Genetic variants known or suspected to cause very low enzyme activity or severe disease symptoms.
Females who can have children and males who are fertile must agree to use highly effective birth control methods from the time of consent until 5 days after the last study drug dose. Females must agree not to become pregnant, and males must agree not to father a child or donate sperm.

Who Cannot Join the Study?

Individuals who do not have a diagnosis of Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) cannot participate. LC-FAOD is a condition where the body has trouble breaking down certain fats to use for energy.
Participants must be within a specific age range, which is not specified here, but generally means that very young children or older adults might not be eligible.
Both males and females can participate, so gender is not a reason for exclusion.
People who are considered part of a vulnerable population may not be eligible. Vulnerable populations can include groups like children, pregnant women, or those with certain disabilities, who might need extra protection in research studies.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Hospital Universitario 12 De Octubre	Madrid	Spain
Instytut Pomnik Centrum Zdrowia Dziecka	Warsaw	Poland
Universidade De Santiago De Compostela	Santiago De Compostela	Spain
Medical Center – University Of Freiburg	Freiburg Im Breisgau	Germany
Vseobecna Fakultni Nemocnice V Praze	Prague	Czechia
Uoyrkzaxbifumh Cyihtcu Keftcebeh	Gdansk	Poland

Trial status

Country	Status	Recruitment Start
Czechia	Not recruiting	28.02.2023
Germany	Not recruiting	28.02.2023
Poland	Not recruiting	28.02.2023
Spain	Not recruiting	28.02.2023

Trial locations

Investigated drugs:

Triheptanoin is a type of oil that is being studied to see if it can help reduce major clinical events in children with long-chain fatty acid oxidation disorders. These disorders affect how the body breaks down certain fats to produce energy, especially during periods without food. Triheptanoin is thought to provide an alternative energy source that might help improve the health of these patients.

Medium-chain Triglycerides (MCT) are a type of fat that is easier for the body to break down and use for energy. In this study, MCT is being compared to triheptanoin to see which one is more effective in reducing major clinical events in children with long-chain fatty acid oxidation disorders. MCT is commonly used in dietary supplements and medical foods for people with certain metabolic disorders.

Investigated diseases:

Fatty acid oxidation disorder

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) – These are a group of rare genetic conditions that affect the body’s ability to break down long-chain fatty acids into energy, especially during periods of fasting or increased energy demands. Individuals with LC-FAOD may experience muscle weakness, low blood sugar, and liver problems. The disorder can lead to episodes of metabolic crisis, which are characterized by symptoms such as extreme fatigue, muscle pain, and heart problems. These episodes can be triggered by illness, fasting, or intense exercise. Over time, repeated metabolic crises can cause damage to organs and tissues. Management of the condition often involves dietary adjustments to prevent energy deficits.

Trial ID:

2023-509809-76-00

Protocol code:

UX007-CL302

NCT ID:

NCT05933200

Trial Phase:

Therapeutic confirmatory (Phase III)

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

What is this study about?

Who Can Join the Study?

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