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GLUT1 DS

Phase 2

Glut1 Deficiency Syndrome | Small molecule | Other |Ultragenyx Pharmaceutical Inc.|Last Updated: Oct 3, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment20
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02014883Phase II Open Label Study Using Triheptanoin in Patients With Glucose Type 1 Transporter Deficiency GLUT1-DSPHASE2 COMPLETED 20Dec 4, 2013Jul 4, 2019Oct 3, 20251 France
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Study Endpoints
Primary Endpoints
Number of paroxystic events
6 months

The number of paroxystic events, in particular abnormal movements, will be collected during trihepatnoin treatment.

Secondary Endpoints
Safety
6 months
6 minutes walk test
6 months
9 hole Peg board
6 months
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
GLUT1 DSEXPERIMENTAL -
Interventions
NameTypeDescription
GLUT1 DSDRUG -
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Eligibility Criteria
Age Range3 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Mutation in SLC2A1 gene * Age \> 3 years * Patient with history/frequency of seizures or movement disorders documented at least 3 months prior to the beginning of the study * Covered by french social security * Patients who freely agree to participate in this study and underst...

Countries:France
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