NCT04632953

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

Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program (LC-FAOD DMP)

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 150 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to assess the long-term safety, including pregnancy, infant, and lactation outcomes, of patients with LC-FAOD who are enrolled in the DMP.

Detailed description

The LC-FAOD Disease Monitoring Program (DMP) is an international, long-term, retrospective and prospective outcomes study aiming to collect safety and effectiveness data for triheptanoin and the natural history of LC-FAOD for a study duration of up to 10 years from adult and pediatric patients with LC-FAOD, with any previous disease management, regardless of prior treatment with triheptanoin, those who have previously participated in triheptanoin clinical trials, and those who received triheptanoin through an Expanded Access Program (EAP). Patients enrolling in the LC-FAOD DMP will be managed at the discretion of their physicians and may or may not be treated with triheptanoin during the course of the study. Patients will have access to triheptanoin only through authorized commercial use (if approved in their country) or available EAP but not from the LC-FAOD DMP itself.

Conditions

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

Interventions

Type	Name	Description
OTHER	No Intervention	No Intervention

Timeline

• Start date: 2021-11-30
• Primary completion: 2035-12-01
• Completion: 2035-12-01
• First posted: 2020-11-17
• Last updated: 2026-03-06

Locations

16 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study