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GR181413A/AT1001

Phase 1

Fabry Disease | Small molecule | Metabolic |Amicus Therapeutics, Inc.|Last Updated: Dec 18, 2013

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
RandomizedPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment14
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01853852A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese SubjectsPHASE1 COMPLETED 14Sep 1, 2011Dec 1, 2011Dec 18, 20131 Australia
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Study Endpoints
Primary Endpoints
Profile of plasma pharmacokinetics
0, 0.5, 1, 1.5, 2, 3, 3.5, 4, 5, 6, 8, 10, 12h post dose

AUC, Cmax, tmax, Tlast , t1/2, CL/F, Vz/F

Number of Participants with adverse events
up to 24 hr
Change from baseline in clinical laboratory tests (hematology, chemistry and urinalysis)
0, 24h post dose
Change from baseline in vital signs (blood pressure and heart rate)
0 ,1 , 2, 3, 4, 5, 6, 24h post dose
Change from baseline in 12-lead ECG
0, i, 2, 3, 6, 24h post dose
Profile of urine pharmacokinetics
0-4, 4-10, 10-12, 12-24h post dose

Ae, CLr, %Fx

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Study Design & Arms
AllocationRANDOMIZED
MaskingSINGLE
ModelCROSSOVER
Treatment Arms
ArmTypeDescription
50 mgEXPERIMENTALGR181413A/AT1001
150 mgEXPERIMENTALGR181413A/AT1001
450 mgEXPERIMENTALGR181413A/AT1001
PlaceboPLACEBO_COMPARATORplacebo
Interventions
NameTypeDescription
GR181413A/AT1001 solutionDRUGPowder for reconstitution
GR181413A/AT1001 capsuleDRUGSize 2, hard gelatin capsule, white opaque / blue opaque
Potable waterOTHERMatched, Size 2, hard gelatin capsule, white opaque/blue opaque
Placebo capsuleDRUGSolution matched
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Eligibility Criteria
Age Range20 Years — 55 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: 1. Healthy as determined by a responsible and experienced physician, based on a medical evaluation including medical history, physical examination, laboratory tests and cardiac monitoring. A subject with a clinical abnormality or laboratory parameters outside the reference range...

Countries:Australia
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