Ultragenyx clinical studies in development for FAOD

UX007 for LC-FAOD

Currently, UX007—an investigational, pharmaceutical-grade, specially designed synthetic triglyceride compound—is being studied in patients with LC-FAOD. Supplement-grade medium-chain triglyceride (MCT) oil has been studied for over 15 years and has shown potential as an alternative energy source for the brain.

Clinical studies of UX007

Phase 2 study

Ultragenyx is currently conducting a 78-week, single-arm, open-label phase 2 study evaluating the safety and efficacy of UX007 in patients with LC-FAOD. It is composed of a 24-week interim study and a 54-week extension study. Patients are to be evaluated on several end points, including:

  • Cycle ergometer performance
  • 12-minute walk test (12MWT)
  • Liver disease/hypoglycemia
  • Cardiac disease
  • Quality of life (QOL)

For more information, visit: https://www.clinicaltrials.gov/ct2/show/NCT01886378.

Phase 3 study

A Phase 3 study of UX007 in LC-FAOD is in planning.

This treatment is investigational and has not yet been approved by any regulatory agency for use. Efficacy and safety have not been established. The information presented should not be construed as a recommendation for use.

If you are interested in clinical study opportunities for FAOD, talk to your doctor or
click here to sign up

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