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glucose

Phase 1

Glycogen Storage Disease Type IA | Monoclonal antibody | Metabolic |Ultragenyx Pharmaceutical Inc.|Last Updated: Feb 11, 2022

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
ACTIVE_CONTROLLEDBiomarker
Total Trials1
Total Enrollment20
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04311307Endogenous Glucose Production in Patients With Glycogen Storage Disease Type IaPHASE1 COMPLETED 20Jan 19, 2021Feb 7, 2022Feb 11, 20221 Netherlands
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Study Endpoints
Primary Endpoints
[6,6-2H2]glucose concentration in GSDIa patients
every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load

Endogenous glucose production (EGP) in GSDIa will be assessed through minimal model calculation

Secondary Endpoints
[6,6-2H2]glucose concentration in GSDIa patients and matched healthy participants
every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
[6,6-2H2]glucose concentration in severe and attenuated GSDIa patients
every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
[6,6-2H2]glucose concentration in the pre-prandial state versus the fed state in GSDIa patients
every 10 minutes within the first hour as well as 75, 90 and 120 minutes after the oral load
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeBASIC_SCIENCE
Treatment Arms
ArmTypeDescription
PatientsEXPERIMENTALGSDIa patients
ControlsACTIVE_COMPARATORHealthy volunteers
Interventions
NameTypeDescription
[6,6-2H2]glucoseBIOLOGICALEach subject will be administered two oral \[6,6-2H2\]glucose loads (pre-prandial, fed) in the hospital setting and one oral \[6,6-2H2\]glucose load at home setting (random time). Capillary blood samples will be collected on filter paper 10 times at specific time points after each oral \[6,6-2H2\]glucose load.
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Eligibility Criteria
Age Range16 Years — N/A
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * The diagnosis should be confirmed by G6PC mutation analysis * Age above 16 years * Stable medical condition before the start of the test procedures Exclusion Criteria: * Age \< 16 years * Intercurrent illness * Recent history of hospitalization due to hypoglycemia * Pregnanc...

Countries:Netherlands
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