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POST Triheptanoin

Phase 1

Frontal Lobe Hypometabolism | Small molecule | Other |Ultragenyx Pharmaceutical Inc.|Last Updated: Jul 23, 2020

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 Enrollment15
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02679235Brain Energy and Aging With TriheptanoinPHASE1 COMPLETED 15Apr 1, 2016Aug 8, 2018Jul 23, 20201 Canada
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Study Endpoints
Primary Endpoints
Global Change in Brain Glucose Uptake
28±2 days

Global change (average of cortex) in brain glucose uptake as measured by 18F-FDG PET scans PRE vs POST 28±2 days of supplementation

Global Change in Brain Ketone Uptake
28±2 days

Global change in brain ketone uptake as measured by 11C-acetoacetate PET scans

Secondary Endpoints
Change in Brain Volumes
28±2 days
Change in Cerebral Blood Flow
28±2 days
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
TriheptanoinEXPERIMENTALParticipant will undergo POST Triheptanoin suppementation 1g/kg body weight for 28 days (dose gradually increased each week, starting at, 0.25, 0.5, 0.75 and then 1 g) separated in 4 doses (with eah meal and before night) after a control PRE supplementation imaging protocol.
Interventions
NameTypeDescription
POST TriheptanoinDRUGThe target daily dose of triheptanoin will be 1 g/kg/d, or approximately 70 g/d during 28±2 days. It will be divided into four daily doses, one of which will be consumed at each meal and one with an evening snack.
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Eligibility Criteria
Age Range65 Years — N/A
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * Men and women ≥65 years old; * Score ≥26/30 on the Montreal Cognitive Assessment; -≥10% lower brain glucose uptake in the frontal cortex as determined by PET imaging. Exclusion Criteria: * Score \<26/30 on the Montreal Cognitive Assessment; * Medications likely to affect the...

Countries:Canada
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