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Genz-112638

Phase 1

Gaucher's Disease Type I | Small molecule | Metabolic |Sanofi|Last Updated: Nov 22, 2023

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
CONTROLLEDBiomarker
Total Trials1
Total Enrollment10
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06143904A Study to Evaluate Absolute Bioavailability, Absorption, Metabolism, and Excretion of Genz-112638 in Healthy Male ParticipantsPHASE1 COMPLETED 10 — — Jun 3, 2009Jul 5, 2009Nov 22, 2023 - —
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Study Endpoints
Primary Endpoints
Assessment of pharmacokinetic (PK) parameter: Cmax
Multiple timepoints up to Day 26

Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

Assessment of pharmacokinetic (PK) parameter: Tmax
Multiple timepoints up to Day 26

Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

Assessment of pharmacokinetic (PK) parameter: AUC0-∞
Multiple timepoints up to Day 26

Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

Assessment of pharmacokinetic (PK) parameter: AUC0 -τ
Multiple timepoints up to Day 26

Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

Assessment of pharmacokinetic (PK) parameter: AUC0-∞/D
Multiple timepoints up to Day 26

Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

Assessment of pharmacokinetic (PK) parameter: AUC0-τ/D
Multiple timepoints up to Day 26

Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

Assessment of pharmacokinetic (PK) parameter: F
Multiple timepoints up to Day 26

Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

Assessment of pharmacokinetic (PK) parameter: CL/F
Multiple timepoints up to Day 26

Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

Assessment of pharmacokinetic (PK) parameter: t½
Multiple timepoints up to Day 26

Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints

Pharmacokinetic (PK) parameter: Absolute bioavailability (F) of single-dose oral versus single-dose IV administration
Multiple timepoints up to Day 26
Assessment of pharmacokinetic (PK) parameter: Total radioactivity excreted in urine and feces
Multiple timepoints up to Day 26

Total radioactivity excreted in urine and feces will be converted to percentage of radioactive dose.

Assessment of pharmacokinetic (PK) parameter: Total radioactivity in whole blood and plasma
Multiple timepoints up to Day 26

Total radioactivity in whole blood and plasma will be converted to ngEq/g Genz-99067 concentration for whole blood and ngEq/mL for plasma, based on the dose specific activity.

Assessment of pharmacokinetic (PK) parameter: % relative abundance of each component in samples of plasma or excreta
Multiple timepoints up to Day 26

It will be estimated as \[(radioactivity for the HPLC peak)/(total radioactivity injected per HPLC run) x 100\].

Assessment of pharmacokinetic (PK) parameter: The percentage of the administered dose attributed to each component in samples of urine or feces
Multiple timepoints up to Day 26

It will be estimated as \[(% relative abundance)/100 x (percentage of radioactive dose in the sample)\].

Assessment of pharmacokinetic (PK) parameter: The radioactivity of [14C]-Genz-99067 and each major metabolite in plasma, as identified by radio-profiling
Multiple timepoints up to Day 26

It will be converted to equivalent concentrations as \[(% relative abundance)/100 x (equivalent concentration of total radioactivity in the sample)\].

Noncompartmental PK parameters: AUC0-t
Multiple timepoints up to Day 26
Noncompartmental PK parameters: AUC0-∞
Multiple timepoints up to Day 26
Noncompartmental PK parameters: Cmax
Multiple timepoints up to Day 26
Noncompartmental PK parameters: Tmax
Multiple timepoints up to Day 26
Noncompartmental PK parameters: t½
Multiple timepoints up to Day 26
Noncompartmental PK parameters: Vz/F
Multiple timepoints up to Day 26
Noncompartmental PK parameters: CL/F
Multiple timepoints up to Day 26
Noncompartmental PK parameters for urine and feces: Cum Ae
Multiple timepoints up to Day 26
Noncompartmental PK parameters for urine and feces: % dose
Multiple timepoints up to Day 26
Renal clearance (CLR) for total plasma radioactivity and Genz-99067
Multiple timepoints up to Day 26
PK parameters [AUC0-τ, AUC0-∞, Cmax, Tmax, t½] and metabolite ratio for metabolite(s) of Genz-99067
Multiple timepoints up to Day 26
Secondary Endpoints
Number of participants with treatment-emergent adverse events (TEAEs), serious adverse event (SAEs), and adverse event of special interest (AESI)
Up to Day 33
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeBASIC_SCIENCE
Treatment Arms
ArmTypeDescription
Treatment Period 1 -2EXPERIMENTALSingle-dose Intravenous (IV) Genz-112638 Day 1 followed by single dose GenZ-112638 oral capsules Day 8
Treatment Period 1-4EXPERIMENTALSingle-dose Intravenous (IV) Genz-112638 Day 1; single dose GenZ-112638 oral capsules Day 8; oral capsule Genz-112638 Days 9-14; single dose \[14C\]-Genz-112638 oral solution Day 15
Interventions
NameTypeDescription
Genz-112638DRUGPharmaceutical form:Solution-Route of administration:IV
[14C]-Genz-112638DRUGPharmaceutical form:Solution-Route of administration:Oral
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Eligibility Criteria
Age Range18 Years — 45 Years
SexMALE
Healthy VolunteersYes

Inclusion Criteria: Having given written informed consent prior to undertaking any study-related procedure The subject has a body weight of 50 to 100 kg \[110 to 220 pounds (lb)\] with a body mass index (BMI) less than 30 kilograms per square meter (kg/m2) at Screening. The subject's physical exami...

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