Active not recruitingPHASE3INTERVENTIONAL

A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD

This study aims to discover if a special oil called triheptanoin works better than a common oil called MCT oil for young people with a genetic condition called Long-chain Fatty Acid Oxidation Disorders (LC-FAOD). These disorders make it hard for the body to turn certain fats into energy, which can lead to serious health issues. The main goal is to see if triheptanoin can reduce how often these serious health problems happen. Participants will be randomly given one of the two oils, and the study will last for about 3.5 to 4 years. There's also a smaller part of the study looking at how these oils might affect liver fat in some participants.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Ultragenyx Pharmaceutical Inc

Enrolment target

Start

28 Feb 2023

Estimated completion

01 Aug 2027

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

What is this study about?

This study is for children and teenagers who have a rare genetic condition known as Long-chain Fatty Acid Oxidation Disorder (LC-FAOD). People with LC-FAOD have trouble breaking down certain types of fat to get energy. This can lead to serious health problems like low blood sugar, muscle weakness, or heart issues, often called Major Clinical Events (MCEs) by doctors. The study wants to find out if a specific oil called triheptanoin is more effective than another oil, called MCT oil, at preventing these serious events.

Researchers will compare these two oils to see if one can help patients stay healthier and have fewer problems. This is important because finding better ways to manage LC-FAOD can significantly improve the lives of young people with this condition. Understanding how these oils work can lead to better treatments in the future.

There's also a smaller, additional part of the study focused on the liver. It will look at whether triheptanoin or MCT oil can help reduce the amount of fat in the liver. Too much fat in the liver can cause problems, so this part of the study is trying to see if these oils can improve liver health for some participants.

Key takeaways

The study compares two oils, triheptanoin and MCT oil, for children and teens with LC-FAOD.
The main goal is to reduce serious health problems (Major Clinical Events).
It's a long-term study, lasting around 3.5 to 4 years.
Patients will be randomly assigned to one of the two oils, and neither they nor their doctors will know which one.
There's an optional liver study for some participants to check liver fat levels.

Who may be eligible?

To join the main part of this study, participants must be under 18 years old – from newborns up to 17 years. They must have a confirmed diagnosis of LC-FAOD and have a parent or guardian who can help them follow all study instructions. Importantly, they also need to have had specific health problems related to their LC-FAOD, such as frequent severe episodes requiring hospital visits, ongoing heart problems, or a history of very low blood sugar.

Some genetic information is also important. Participants may qualify if they have genetic changes known to cause severe LC-FAOD. Both males and females can take part. If females are old enough to have children or males can father children, they will need to agree to use effective birth control during and shortly after the study.

For the smaller liver study, participants must already be in the main study and be older than 2 years of age. They will also need to have a certain amount of fat in their liver, as shown by a special scan, and be able to stay still for this scan.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is the person under 18 years old (including newborns)?
Do they have a confirmed diagnosis of LC-FAOD?
Have they had specific serious health problems related to LC-FAOD recently?
Is a parent or guardian able and willing to help with study tasks?
For the liver study: Is the person over 2 years old and have a certain amount of fat in their liver?

Answer every question to see your result.

What does participation involve?

If you join this study, it will last for about 3.5 to 4 years. Initially, there will be a screening period to make sure you're a good fit. Then, you'll be randomly assigned to receive either the triheptanoin oil or the MCT oil. This is a "double-blind" study, meaning neither you nor your doctors will know which oil you're getting, so everyone can be as unbiased as possible.

During the study, there will be a phase to gradually adjust your dose (titration) and regular check-ups to see how you're doing. After the main treatment period ends, there will be a safety phone call to follow up on your health. Participants in the liver sub-study will also have special scans to measure liver fat. After the main part of the study, there might be an 'open access period' where all participants could potentially receive triheptanoin.

Throughout the study, you'll have doctors' visits for assessments, and you'll be taking the assigned oil regularly. The aim is to carefully monitor your health and see how the oils affect your condition over time.

Potential risks and benefits

Participating in this study might offer potential benefits, such as receiving treatment that could help reduce serious health problems related to LC-FAOD, and regular, close monitoring by specialists. However, there are also potential risks, such as side effects from the study oils, which will be carefully explained by the study team. You might not receive the active treatment, or the treatment might not work for you. It's important to remember that joining a study is always voluntary, and you have the right to withdraw at any time, for any reason, without it affecting your medical care.

Locations (15)

General University Hospital in Prague-GUH (Všeobecná fakultní nemocnice v Praze- VFN)
Verified postcode
Prague, Czechia
Universitätsklinikum Freiburg
Verified postcode
Freiburg im Breisgau, Germany
Juntendo University Hospital
City only
Bunkyo City, Japan
The Jikei University Hospital
Verified postcode
Minato, Japan
Instytut Pomnik-Centrum Zdrowia Dziecka
Verified postcode
Warsaw, Poland
Gdańksi Uniwersytet Medyczny
Verified postcode
Gdansk, Poland
King Faisal Specialist Hospital & Research Centre
Verified postcode
Riyadh, Saudi Arabia
Sant Joan de Deu Hospital (SJD)
Verified postcode
Barcelona, Spain
University Hospital Santiago de Compostela
Verified postcode
A Coruña, Spain
University Hospital 12 de Octubre
Verified postcode
Madrid, Spain
Cukurova University
Verified postcode
Adana, Turkey (Türkiye)
Gazi University
Verified postcode
Ankara, Turkey (Türkiye)

Common questions

What is LC-FAOD?

LC-FAOD stands for Long-chain Fatty Acid Oxidation Disorders. It's a genetic condition where the body has trouble turning certain fats into energy, which can cause serious health problems.

What's the difference between triheptanoin and MCT oil?

Triheptanoin is a special oil being tested, while MCT oil is a more common oil. Both are used to help people with LC-FAOD get energy, but this study is comparing which one works better for serious health problems.

Will I know which oil I'm taking?

No, this is a 'double-blind' study. Neither you nor the study doctors will know if you're taking triheptanoin or MCT oil. This helps make sure the results are fair.

How long will the study last?

The main study is expected to last for about 3.5 to 4 years from start to finish.

What about the liver part of the study?

Some participants older than 2 will also have special scans for a 'Liver Substudy' to see if either oil helps reduce fat in the liver. This is an optional part for those who qualify.