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

PHASE2CompletedINTERVENTIONAL
Enrollment

20

Participants

Timeline

Start Date

February 20, 2014

Primary Completion Date

June 30, 2019

Study Completion Date

June 30, 2019

Conditions
Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)
Interventions
DRUG

Triheptanoin

"Schedule A: Subjects previously treated with triheptanoin will continue to dose at approximately 35% of total daily calories (\~1-4g/kg/day, depending on age).~Schedule B: Subjects who are naïve to triheptanoin will begin a 2-week fixed titration schedule up until they have reached 35% of total daily calories (\~1-4 g/kg/day depending on age). If a subject has not reached the target of 35% of total daily calories, by the end of the 2-week fixed titration period, dose titration should continue until achieved or until the maximally tolerated dose has been established."

Trial Locations (1)

76104

Cook Childrens Medical Center, Fort Worth

All Listed Sponsors
collaborator

Ultragenyx Pharmaceutical Inc

INDUSTRY

lead

Adrian Lacy

OTHER

NCT02036853 - An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome | Biotech Hunter | Biotech Hunter