Not provided
Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| U1111-1294-7994 | Registry Identifier | ICTRP |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Objectives:
To determine pharmacokinetic (PK) variables, including absolute bioavailability (F), of Genz-99067, the free base of the L-tartaric acid salt of Genz-112638 as it exists in plasma, after a single intravenous (IV) dose and after a single oral dose of Genz-112638 (unlabeled).
To determine the PK, total recovery, routes and rates of excretion, and the metabolic profile of Genz-99067 after 5 days of BID oral dosing with unlabeled Genz-112638 followed by a single dose of [14C]-Genz-112638.
The maximum study duration for an individual subject was approximately 65 days from the beginning of Screening through the Safety Follow-up visit.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Period 1 -2 | Experimental | Single-dose Intravenous (IV) Genz-112638 Day 1 followed by single dose GenZ-112638 oral capsules Day 8 |
|
| Treatment Period 1-4 | Experimental | Single-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 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Genz-112638 | Drug | Pharmaceutical form:Solution-Route of administration:IV |
|
| Measure | Description | Time Frame |
|---|---|---|
| Assessment of pharmacokinetic (PK) parameter: Cmax | Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints | Multiple timepoints up to Day 26 |
| Assessment of pharmacokinetic (PK) parameter: Tmax | Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints | Multiple timepoints up to Day 26 |
| Assessment of pharmacokinetic (PK) parameter: AUC0-∞ | Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints | Multiple timepoints up to Day 26 |
| Assessment of pharmacokinetic (PK) parameter: AUC0 -τ | Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints | Multiple timepoints up to Day 26 |
| Assessment of pharmacokinetic (PK) parameter: AUC0-∞/D | Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints | Multiple timepoints up to Day 26 |
| Assessment of pharmacokinetic (PK) parameter: AUC0-τ/D | Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints | Multiple timepoints up to Day 26 |
| Assessment of pharmacokinetic (PK) parameter: F | Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints |
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with treatment-emergent adverse events (TEAEs), serious adverse event (SAEs), and adverse event of special interest (AESI) | Up to Day 33 |
Not provided
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 examination results, vital signs, laboratory assessments, and cardiac assessments are within normal limits or, if abnormal, are not clinically significant at Screening and Day -1. Exclusion Criteria: Participants are excluded from the study if any of the following criteria apply:
Prolonged QTc interval (eg, repeated demonstration of a QTc interval ≥450 msec), family history of long QT or Brugada Syndrome, and/or history of sudden death in a first-degree relative.
The subject receives an immunization within 30 days of providing informed consent.
The subject has a history of drug allergies (eg, significant rash, hives, etc in response to antibiotics).
The above information is not intended to contain all considerations relevant to the potential participation in a clinical trial.
Not provided
Not provided
Not provided
Not provided
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D005776 | Gaucher Disease |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
Not provided
Not provided
| ID | Term |
|---|---|
| C522917 | eliglustat |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Genz-112638 | Drug | Pharmaceutical form:Capsule-Route of administration:Oral |
|
|
| [14C]-Genz-112638 | Drug | Pharmaceutical form:Solution-Route of administration:Oral |
|
|
| Multiple timepoints up to Day 26 |
| Assessment of pharmacokinetic (PK) parameter: CL/F | Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints | Multiple timepoints up to Day 26 |
| Assessment of pharmacokinetic (PK) parameter: t½ | Plasma concentrations of Genz-99067 will be collected at pre-specified timepoints | Multiple timepoints up to Day 26 |
| 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 | Total radioactivity excreted in urine and feces will be converted to percentage of radioactive dose. | Multiple timepoints up to Day 26 |
| Assessment of pharmacokinetic (PK) parameter: Total radioactivity in whole blood and plasma | 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. | Multiple timepoints up to Day 26 |
| Assessment of pharmacokinetic (PK) parameter: % relative abundance of each component in samples of plasma or excreta | It will be estimated as [(radioactivity for the HPLC peak)/(total radioactivity injected per HPLC run) x 100]. | Multiple timepoints up to Day 26 |
| Assessment of pharmacokinetic (PK) parameter: The percentage of the administered dose attributed to each component in samples of urine or feces | It will be estimated as [(% relative abundance)/100 x (percentage of radioactive dose in the sample)]. | Multiple timepoints up to Day 26 |
| Assessment of pharmacokinetic (PK) parameter: The radioactivity of [14C]-Genz-99067 and each major metabolite in plasma, as identified by radio-profiling | It will be converted to equivalent concentrations as [(% relative abundance)/100 x (equivalent concentration of total radioactivity in the sample)]. | Multiple timepoints up to Day 26 |
| 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 |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |