Triheptanoin (UX007) to Treat Citrate Transporter Deficiency

NO_LONGER_AVAILABLE

Conditions studied: Citrate Transporter Deficiency, SLC13A5 Gene Mutation

In brief

The purpose of this study is to determine whether triheptanoin (UX007) is effective in the treatment of neurological symptoms related to citrate transporter deficiency (SLC13A5 gene mutation).

Key facts

Study ID

NCT02500082

Run by

Irina A Anselm

Last updated by the study team

2016-01-28

Who can join

Age: 4 and older, up to 9. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of citrate transporter deficiency due to mutations in the SLC13A5 gene.
• Presentation with severe global developmental delay and seizures.

You may not qualify if…

• Valproate is an AED that partially inhibits the TCA cycle via alpha-ketoglutarate dehydrogenase and should not be administered to subjects taking UX007.

Where it is running

• Department of Neurology, Boston Children's Hospital — Boston, Massachusetts, United States

Full record on ClinicalTrials.gov

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