Recent Updates
Recently added Catalysts

A

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
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
RandomizedCONTROLLED
Total Trials1
Total Enrollment20
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01489995Migalastat Food Effect StudyPHASE1 COMPLETED 20 — — Oct 1, 2011Dec 1, 2011Dec 18, 20131 United States
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Maximum observed plasma concentration of migalastat HCl after a single dose
5 weeks (60 PK timepoints)

Cmax

Time of occurence of maximum observed plasma concentration of migalastat HCl after a single dose
5 weeks (60 PK timepoints)

tmax

Area under the plasma concentration-time curve of migalastat HCl after a single dose from time 0 (before dosing) to infinity
5 weeks (60 PK timepoints)

AUC (0 to infinity)

Terminal phase half life of migalastat HCl after a single dose
5 weeks (60 PK timepoints)

t1/2

Apparent clearance following oral dosing of migalastat HCl after a single dose
5 weeks (60 PK timepoints)

CL/F

Area under the plasma concentration-time curve of migalastat HCl after a single dose from time 0 (before dosing) to the time of the last quantifiable concentration
5 weeks (60 PK timepoints)

AUC (0 to t)

Secondary Endpoints
Adverse Events
5 weeks
Clinical Laboratory Tests
5 weeks
Vital Signs
5 weeks
Unlock Study Endpoints
Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelCROSSOVER
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ReferenceEXPERIMENTALFasted
Glucose DrinkEXPERIMENTALFed
Before High Fat MealEXPERIMENTALFed
Before Light MealEXPERIMENTALFed
After Light MealEXPERIMENTALFed
Interventions
NameTypeDescription
A (migalastat)DRUG150 mg migalastat HCl in the fasting state (reference arm)
B (migalastat)DRUG150 mg migalastat HCl with simultaneous consumption of a glucose drink
C (migalastat)DRUG150 mg migalastat HCl 1 hour before consumption of a high fat meal
D (migalastat)DRUG150 mg migalastat HCl 1 hour before consumption of a light meal
E (migalastat)DRUG150 mg migalastat HCl 1 hour after consumption of a light meal
Unlock Study Design Details
Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * Male or Female aged 18 to 65 years inclusive * Healthy, as determined by study physician * Capable of giving informed consent Exclusion Criteria: * Positive for HIV or Hepatitis B and/or C viruses * History of drug or alcohol abuse or addiction within 2 years * Smoker or con...

Countries:United States
Unlock Eligibility Criteria