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Genz-112638

Phase 1

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

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment10

FDA Designations

No designations recorded

Clinical trial landscape

Genz-112638 · 1 trial · 1 indication

Phase 1 1
NCT06143904A Study to Evaluate Absolute Bioavailability, Absorption, Metabolism, and Excretion of Genz-112638 in Healthy Male ParticipantsGaucher's Disease Type I
COMPLETED10 Analytics
PHASE1COMPLETED
A Study to Evaluate Absolute Bioavailability, Absorption, Metabolism, and Excretion of Genz-112638 in Healthy Male Participants
Gaucher's Disease Type IUnlock trial analytics

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 to 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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Frequently asked questions about Genz-112638

What is Genz-112638 used for?

Genz-112638 is an investigational small molecule being developed for the treatment of Gaucher's Disease Type I, a rare genetic disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The drug is being studied to evaluate its absorption, metabolism, and excretion in the body.

Who makes Genz-112638?

Genz-112638 is being developed by Sanofi, a multinational pharmaceutical company listed on the stock exchange under the ticker symbol SNY. The drug is in early-stage clinical development for Gaucher's Disease Type I, a rare disease indication.

What phase is Genz-112638 in?

Genz-112638 is currently in Phase 1 clinical development. It is an investigational drug and has not yet been approved for commercial use. The ongoing research is focused on understanding the drug's pharmacokinetics, including its bioavailability, absorption, metabolism, and excretion in healthy volunteers.

What clinical trials is Genz-112638 in?

Genz-112638 has one completed Phase 1 clinical trial registered under the identifier NCT06143904. This study evaluated the absolute bioavailability, absorption, metabolism, and excretion of the drug in healthy male participants aged 18 years and older. The trial enrolled 10 participants and was a controlled study.

Is Genz-112638 the same as eliglustat?

Genz-112638 is the developmental code name for the drug also known as eliglustat. It is being developed by Sanofi for the treatment of Gaucher's Disease Type I. The drug is currently in Phase 1 clinical trials to assess its pharmacokinetic properties.