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Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program

Monitoring program for fatty acid oxidation disorders in clinic.

Recruiting
All
Phase N/A

LC-FAOD stands for Long-Chain Fatty Acid Oxidation Disorders, which are rare problems where the body can't break down certain fats, called long-chain fatty acids, for energy. This study is about watching over patients with LC-FAOD to see how safe a treatment called triheptanoin is in the long run. Triheptanoin is a special type of oil that helps provide energy to people with LC-FAOD.

The study lasts up to 10 years and will collect information from both adults and children with LC-FAOD. Patients can join if they have been diagnosed with any type of LC-FAOD and are willing to follow study rules. They can join even if they've used triheptanoin before or if they are getting it through special programs. Patients will be looked after by their doctors and may or may not take triheptanoin during the study. However, the study itself won't provide this treatment.

  • Study Duration: Up to 10 years.
  • Participants: Adults and children with LC-FAOD.
  • Treatment: Triheptanoin access through usual channels, not directly from the study.
Study details
    Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

NCT04632953

Ultragenyx Pharmaceutical Inc

26 July 2025

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