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Triheptanoin (UX007) to Treat Citrate Transporter Deficiency

NCT02500082 · Status: NO_LONGER_AVAILABLE · Type: EXPANDED_ACCESS

Last updated 2016-01-28

No results posted yet for this study

Summary

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).

Conditions

  • Citrate Transporter Deficiency
  • SLC13A5 Gene Mutation

Interventions

DRUG

triheptanoin

Triheptanoin (UX007) is a medium chain triglyceride of three seven-carbon fatty acids (C7), on a glycerol backbone, with a molecular formula of C24H44O6. It is being evaluated as a substrate replacement therapy for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD) and for the treatment of seizures associated with Glut 1 DS. Triheptanoin is metabolized to provide substrate replacement for both fatty acid metabolism and anaplerosis (replacement of TCA cycle intermediates) required to restore the efficient generation of energy and the net production of glucose in patients. The mechanism of action of triheptanoin in restoring energy metabolism is dependent on its medium-chain length as well as its odd-carbon properties. Triheptanoin is a highly purified form intended for oral administration.

Sponsors & Collaborators

  • Irina A Anselm

    lead OTHER

Principal Investigators

  • Irina A Anselm, MD · Boston Children's Hospital

Eligibility

Min Age
4 Years
Max Age
9 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02500082 on ClinicalTrials.gov