Triheptanoin
(Brand name Dojolvi)
Approved in the U.S. for children and adults with genetically confirmed LC-FAODs. Not yet approved in Australia.
Study details
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Paediatric Patients With LC-FAOD (NCT05933200).
Manufacturer
Ultragenyx Pharmaceutical Inc.
Types of mito
Long-chain fatty acid oxidation disorders (LC-FAODs)
Which people with mito might this therapy help?
Children and adults with genetically confirmed long-chain fatty acid oxidation disorders (LC-FAODs). These include:
- VLCAD deficiency ⓘ Very long-chain acyl-CoA dehydrogenase deficiency
- LCHAD deficiency ⓘ Long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency
- TFP deficiency ⓘ Trifunctional protein deficiency
- CPT I deficiency ⓘ Carnitine palmitoyltransferase I deficiency
- CPT II deficiency ⓘ Carnitine palmitoyltransferase II deficiency
- CACT deficiency ⓘ Carnitine-acylcarnitine translocase deficiency
Dojolvi is a prescription medicine taken by mouth. It is an oil that can be mixed with food or liquid. It gives the body a type of fat that can be used for energy. This may help people with LC-FAODs, because their bodies have trouble using long-chain fats for energy.
Treatment is managed by a metabolic specialist and dietitian. The dose is based on a person’s daily calorie needs.
What research has been done on this therapy?
Dojolvi has been studied in children and adults with LC-FAODs. The U.S. approval was based on evidence from 3 clinical studies. These studies looked at safety, side effects, and whether triheptanoin could help reduce serious LC-FAOD events.
A long-term extension study also looked at triheptanoin in 94 people with LC-FAODs. This study followed people who had used triheptanoin before, and people who had not used it before.
Common side effects include stomach pain, diarrhoea, vomiting and nausea.
Clinical trial information
Study name
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Paediatric Patients With LC-FAOD (NCT05933200).
Location
Multiple sites overseas, including Europe, Japan, Saudi Arabia, and Turkey. There are no Australian sites.
Who is funding the study?
Ultragenyx Pharmaceutical Inc.
This phase 3 study is comparing triheptanoin with standard medium-chain triglyceride oil, also called MCT oil.
The study is looking at whether triheptanoin can reduce major clinical events in children with LC-FAODs. These events may include serious low blood sugar, muscle breakdown, or heart problems that need urgent care or hospital care.
Important information
Find out more
Emerging therapies for mito
Learn about developing therapies and opportunities to take part in research.
Clinical trials explained
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