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Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency clinical trials at UCSF
1 in progress, 0 open to new patients

  • Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies.

    Sorry, accepting new patients by invitation only

    This open-label long-term safety and efficacy study will provide an opportunity for LC-FAOD patients to be treated with UX007 for up to 5 years or until market approval, whichever occurs first, under a single standardized protocol. The subjects may have participated in other studies or treatment programs with UX007/triheptanoin but would be consolidated into one program for long-term maintenance and consistent safety monitoring. The study is designed to obtain long-term safety information and evaluate maintenance of efficacy in a diverse LC-FAOD population.

    San Francisco, California and other locations