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

NCT06193304 | Completed Phase 1 FDA Drug

• 2 other identifiers: PKM14187U1111-1294-8055
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Gaucher's Disease

Outcome Measures

Primary Outcomes (7)

• 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 Outcomes (1)

• Treatment emergent adverse events during the study

  Up to Day 3

Study Arms (1)

Eliglustat

EXPERIMENTAL

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

Interventions

Eliglustat DRUG

Pharmaceutical form:solution -Route of administration: Oral wihtout ingestion

Also known as: GZ385660, CERDELGA

Eliglustat

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (1)

Covance Clinical Researsh Unit

Evansville, Indiana, 47710, United States

Location

MeSH Terms

Conditions

Gaucher Disease

Interventions

eliglustat

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2023
• First Posted: January 5, 2024
• Study Start: August 25, 2014
• Primary Completion: September 21, 2014
• Study Completion: September 21, 2014
• Last Updated: January 5, 2024

Record last verified: 2023-12

Data Sharing

• IPD Sharing: Will share

Locations

US (1)