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.
Age range
1 Year – 50 Years
Sex
ALL
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Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Reported Change in Seizures Frequency From Baseline at 13 Weeks
Timeframe: Baseline and 13 weeks
Reported Change in Seizures Frequency From Baseline at 26 Weeks
Timeframe: Baseline and 26 weeks
Reported Change in Seizures Frequency From Baseline at 1 Year
Timeframe: Baseline and one yr
Reported Change in Seizures Frequency From Baseline at 18 Months
Timeframe: Baseline and 18 months
Reported Change in Seizures Frequency From Baseline at 2 Years
Timeframe: Baseline and two yrs
Reported Change in Seizures Frequency From Baseline at 3 Years
Timeframe: Baseline and three yrs
Reported Change in Seizure Frequency From Baseline at 4 Years
Timeframe: Baseline and four yrs
Reported Change in Seizure Frequency From Baseline at 5 Years
Timeframe: Baseline and five yrs