Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) Clinical Trial
Official title:
An Open-label Intermediate-size Treatment Protocol for the Urgent Treatment of Seriously Ill Patients With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) With Triheptanoin (UX007)
Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial.
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS): Available through Intermediate-Size Population Expanded Access in US only. The intermediate-size expanded access treatment protocol is intended to provide rapid access to triheptanoin for the treatment of seriously ill patients with Glut1 DS. Consideration for access is for patients with previous exposure to triheptanoin. Patients will be treated under this protocol for the duration of one year, with consideration on a yearly basis for extension of treatment based on the risk-benefit ratio assessed in the Treating Physician's quarterly progress reports. Patients may continue to receive triheptanoin under this intermediate-size treatment protocol until commercial availability of triheptanoin, should the drug receive regulatory approval for the specific disease indication. Long Chain Fatty Acid Oxidation Disorders (LC-FAOD) and Non-FAOD conditions: Expanded access may be available outside of the US in countries prior to approval by the local regulatory agencies. For full details, please visit the links provided below. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT01993186 -
Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
|
Phase 2 | |
| Terminated |
NCT02960217 -
Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
|
Phase 3 |