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Genz-682452

Phase 1

Healthy | Monoclonal antibody | Other |Sanofi|Last Updated: Mar 19, 2014

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
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment36
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01710826A Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Genz-682452 in Healthy VolunteersPHASE1 COMPLETED 36Oct 1, 2012Feb 1, 2013Mar 19, 20141 United States
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Study Endpoints
Primary Endpoints
Participants with Treatment-Emergent Adverse Events (TEAEs)
Day 1 through Day 14
Secondary Endpoints
Pharmacokinetics as measure by plasma parameters
Day 1 through Day 14
Pharmacodynamics as measure by Plasma concentrations of Glucosylceramide (GL-1), Globotriaosylceramide (GL-3) and GM3 ganglioside (GM3)
Day 1, through Day 14
Pharmacokinetics Urine parameter as measure by assessment of Genz-682452 in Urine, Ae0-t, fe0-t (Exploratory)
Day 1 through Day 14
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeBASIC_SCIENCE
Treatment Arms
ArmTypeDescription
Genz-682452EXPERIMENTALThis study will include three cohorts for doses of Genz-682452: Dose 1, Dose 2, Dose 3. Each cohort will include 12 participants, 9 of whom will be administered Genz-682452 and 3 will be administered placebo.
PlaceboPLACEBO_COMPARATORPlacebo comparator taken by participants randomized to the placebo arm in each of the three cohorts. Each cohort will include 12 participants, 9 of whom will be administered Genz-682452 and 3 will be administered placebo.
Interventions
NameTypeDescription
Genz-682452BIOLOGICALCapsules for oral administration.
PlaceboBIOLOGICALPlacebo capsules matching the Genz-682452 capsules.
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Eligibility Criteria
Age Range18 Years — 45 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * Body weight between 50.0 and 95.0 kg, inclusive, if male and between 40.0 and 85.0 kg inclusive, if female, body mass index between 18.0 and 30.0 kg/m\^2, inclusive. * Certified as healthy by a comprehensive clinical assessment. Exclusion Criteria: * Any history or presence ...

Countries:United States
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