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drugset / Trial / NCT02036853

An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome

NCT02036853 ↗

Phase 2 Completed 20 enrolled Adrian Lacy Ultragenyx Pharmaceutical Inc · collab
Non-randomizedSingle-groupOpen-labelTreatment

Summary

This study is being done to assess the safety and long-term efficacy of triheptanoin in pediatric patients with Glut1 DS over a 5-year treatment period. Glut 1 is a protein that helps transport glucose to the brain. Glucose is the brain's primary source of energy. Glut 1 DS prevents this protein from being effectively produced, causing deprivation of energy to the neurons of the of the brain. Glut1 DS is a severely debilitating disease characterized by seizures, developmental delay and movement disorder. There are currently no approved treatments specific to Glut1 DS. Treatment generally includes medications for control of seizures. The use of a ketogenic diet can be effective in controlling seizures when medications are ineffective or provide insufficient control. However, the ketogenic diet may be very difficult for patients to maintain for long periods of time, and there may be negative secondary long-term effects of ketogenic diet.. Triheptanoin is metabolized to molecules that can provide an alternative energy source to the brain, and appears to help in controlling seizures without many of the difficulties of the ketogenic diet. Eligible patients may be those who have been diagnosed with GLUT1 DS, and have discontinued or are not currently on ketogenic diet, or are able to tolerate triheptanoin if they have been treated or are currently being treated with triheptanoin and do not qualify for any other clinical trial.

Timeline

Start
2014-02-20
Primary completion
2019-06-30
Completion
2019-06-30

Publications

Drugs

EvaluationDrugModalityDoseRoute
Subject Triheptanoin Other / unclassified 1 g/kg —
Subject Triheptanoin Other / unclassified 4 g/kg —

Indications

GLUT1 deficiency syndrome