Clinical trials: Glut1 Deficiency Syndrome
Glucose Transporter Type 1 Deficiency Syndrome, GLUT1 Deficiency Syndrome
Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D)
The purpose of this trial is to determine if an alternative energy source will impact brain metabolism in a disorder characterized by glucose metabolism failure in the brain.
- Trial name
- NCT02018315
- Testing
- Triheptanoin
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Juan Pascual
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
The primary objectives of the study are to evaluate the efficacy of UX007 compared to placebo as measured by the reduction from randomization to Week 8 in frequency of seizures and to evaluate the safety of UX007 via adverse…
- Trial name
- NCT01993186
- Testing
- UX007
- Where
- 14 sites in United States, Australia and 5 more
- Volunteers
- 36 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)
An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome
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.
- Trial name
- GLUT1DS (NCT02036853)
- Testing
- Triheptanoin
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Adrian Lacy
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
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)
The primary objective of the study was to evaluate the efficacy and safety of UX007 in the treatment of disabling paroxysmal movement disorders associated with Glut1 DS.
- Trial name
- NCT02960217
- Testing
- UX007
- Where
- 12 sites in United States, France and 4 more
- Volunteers
- 44 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
GLUT1DS1, Epilepsy
Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)
Forty-five subjects receiving no dietary therapy with a proven G1D diagnosis will be enrolled. To evaluate the effect of C7 supplementation of a regular diet on a EEG activity in addition to IQ, language, working memory…
- Conditions
- GLUT1DS1, Epilepsy, Glut1 Deficiency Syndrome 1, Autosomal Recessive, Glucose Metabolism Disorders
- Trial name
- NCT03181399
- Testing
- Triheptanoin
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- University of Texas Southwestern Medical Center
Glucose Transporter Type 1 Deficiency Syndrome
Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)
The primary objective of the study is to evaluate the long-term safety of UX007 in Glut1 DS participants.
- Trial name
- NCT02599961
- Testing
- UX007
- Where
- 9 sites in United States, Australia and 3 more
- Volunteers
- 15 people
- Sponsor
- Ultragenyx Pharmaceutical Inc