Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)
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|ClinicalTrials.gov Identifier: NCT03181399|
Recruitment Status : Recruiting
First Posted : June 8, 2017
Last Update Posted : August 20, 2019
|Condition or disease||Intervention/treatment||Phase|
|GLUT1DS1 Epilepsy Glut1 Deficiency Syndrome 1, Autosomal Recessive Glucose Metabolism Disorders Glucose Transport Defect Glucose Transporter Type 1 Deficiency Syndrome Glucose Transporter Protein Type 1 Deficiency Syndrome||Drug: Triheptanoin||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||45 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||All subjects will receive supplementation at the maximum tolerated dose.|
|Masking:||None (Open Label)|
|Official Title:||Dietary Treatment of Glucose Transporter Type 1 Deficiency (G1D)|
|Actual Study Start Date :||April 18, 2018|
|Estimated Primary Completion Date :||May 30, 2020|
|Estimated Study Completion Date :||May 30, 2023|
This is a single arm study.
. Triheptanoin will be taken 4 times per day (approximately every 6 hours: prior to breakfast, lunch and dinner and a mid-afternoon snack) by mouth. It is dosed 4 times per day, divided evenly.
Other Name: C7
- Neuropsychological attention scores [ Time Frame: Medication taken daily for 6 months. ]To evaluate the impact of triheptanoin supplementation on measures of neuropsychological function primarily indicative of attention in G1D subjects receiving normal diet. These measures include one of two quantitative scales WPPSI-IV (Wechsler Preschool and Primary Scale of Intelligence; if younger than 7 years old), or WASI-II (Wechsler Abbreviated Scale of Intelligence; if older than 8 years old) depending on age.
- EEG changes: spike-wave activity duration in EEG (electroencephalogram) tracings [ Time Frame: Medication taken daily for 6 months. ]Expecting to find a greater than 30% decrease in spike-wave activity determined as percent duration of spike-wave activity over the total duration of EEG.
- Ataxia scores [ Time Frame: Medication taken daily for 6 months. ]
Ataxia is scored 0 (normal; no ataxia) to 30 (severe ataxia) per modified ICARS ( International Cooperative Ataxia Rating Scale) scale in Schmahmann, J. D., Gardner, R., MacMore, J. and Vangel, M. G. (2009), Development of a brief ataxia rating scale (BARS) based on a modified form of the ICARS.
Mov. Disord., 24: 1820-1828
- Global impression scale [ Time Frame: Medication taken daily for 6 months. ]Scores range from 1 (very much improved) through to 7 (very much worse)
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03181399
|Contact: Juan Pascual, MDfirstname.lastname@example.org|
|Contact: Adrian Avila, BSemail@example.com|
|Principal Investigator:||Juan Pascual, MD||Study Principal Investigator|