A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth (Acronym)

An Open-label Study to Assess the Absorption Through the Mouth After Three Repeated 50 mg Doses of Eliglustat Solution, Separated by 2-hour Intervals, Held in the Mouth for 30 Seconds With Swishing But Without Ingestion, in Healthy Subjects

A study to assess the absorption of eliglustat through the mouth in healthy subjects and the safety of any systemic exposure resulting from oral surface absorption of eliglustat in healthy subjects.

Study Overview

• Status: Completed
• Conditions: Gaucher's Disease
• Intervention / Treatment: Drug: Eliglustat

Detailed Description

Duration of the study for each subject, not including screening, will be 3 days including follow-up.

• Study Type: Interventional
• Enrollment: 6
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Indiana
    • Evansville, Indiana, United States, 47710
      • Covance Clinical Researsh Unit

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

Body weight between 50.0 and 100.0 kg, inclusive, for males, and between 40.0 and 90.0 kg, inclusive, for females, body mass index between 18.0 and 30.0 kg/m2, inclusive.

Having given written informed consent prior to undertaking any study-related procedure.

Certified as healthy by a comprehensive clinical assessment (detailed medical history, complete physical examination, laboratory parameters, and ecg).

Exclusion Criteria:

Participants are excluded from the study if any of the following criteria apply:

Any history or presence of clinically relevant cardiovascular, pulmonary, gastrointestinal, hepatic, renal, metabolic, hematological, neurological, osteomuscular, articular, psychiatric, systemic, ocular, gynecologic (if female), or infectious disease, or signs of acute illness.

Any subject who, in the judgment of the Investigator, is likely to be noncompliant during the study, or unable to cooperate because of a language problem or poor mental development.

The above information is not intended to contain all considerations relevant to the potential participation in a clinical trial.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Basic Science
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Eliglustat; Three repeated doses of eliglustat solution, separated by 2-hour intervals, held in the mouth for 30 seconds with swishing but without ingestion	Drug: Eliglustat; Pharmaceutical form:solution -Route of administration: Oral wihtout ingestion; Other Names: GZ385660; CERDELGA

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Plasma pharmacokinetic (PK) parameter: Cmax	Maximum plasma concentration observed (Cmax)	Multiple timepoints on Day 1
Plasma pharmacokinetic (PK) parameter tmax	Time to reach Cmax (tmax)	Multiple timepoints on Day 1
Plasma pharmacokinetic (PK) parameter AUClast	Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to the real time tlast (AUClast)	Multiple timepoints on Day 1
Plasma pharmacokinetic (PK) parameter AUC 0-2h	Area under the plasma concentration versus time curve calculated using the trapezoidal method from time zero to 2 hours (h) post dose (AUC0-2h)	Multiple timepoints on Day 1
Plasma pharmacokinetic (PK) parameter AUC 2-4h	Area under the plasma concentration versus time curve calculated using the trapezoidal method from time 2 hours post dose to 4 hours post dose (AUC2-4h)	Multiple timepoints on Day 1
Plasma pharmacokinetic (PK) parameter AUC 4-6h	Area under the plasma concentration versus time curve calculated using the trapezoidal method from time 4 hours post dose to 6 hours post dose (AUC4-6h)	Multiple timepoints on Day 1
Plasma pharmacokinetic (PK) parameter tlast		Multiple timepoints on Day 1

Secondary Outcome Measures

Outcome Measure	Time Frame
Treatment emergent adverse events during the study	Up to Day 3

Collaborators and Investigators

• Sponsor: Sanofi

Study record dates

Study Major Dates

• Study Start (Actual): August 25, 2014
• Primary Completion (Actual): September 21, 2014
• Study Completion (Actual): September 21, 2014

Study Registration Dates

• First Submitted: December 21, 2023
• First Submitted That Met QC Criteria: December 21, 2023
• First Posted (Actual): January 5, 2024

Study Record Updates

• Last Update Posted (Actual): January 5, 2024
• Last Update Submitted That Met QC Criteria: December 21, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Lipidoses; Lipid Metabolism, Inborn Errors; Gaucher Disease; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Eliglustat
• Other Study ID Numbers: PKM14187; U1111-1294-8055 (Registry Identifier: ICTRP)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No