A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate

Phase 1

Completed

• Conditions: Gaucher Disease
• Interventions: Drug: eliglustat

• Registration Number: NCT02536911
• Lead Sponsor: Genzyme, a Sanofi Company

Brief Summary

Primary Objective:

To study the effect of mild and moderate hepatic impairment on the pharmacokinetics (PK) of eliglustat.

Secondary Objective:

To assess the tolerability of eliglustat tartrate given as a single dose in subjects with mild and moderate hepatic impairment in comparison with matched subjects with normal hepatic function.

Detailed Description

The total study duration from screening period is approximately 31 days.

Study Timeline

• Study First Submitted: 2015-08-28
• Study First Submitted QC: 2015-08-28
• Study Start: 2015-09
• Study First Posted: 2015-09-01
• Primary Completion: 2016-12
• Study Completion: 2016-12
• Status Verified: 2017-02
• Last Update Submitted: 2017-02-09
• Last Update Posted: 2017-02-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
GZ385660 (healthy subjects)	eliglustat	Single dose of eliglustat tartrate will be given under fed conditions
GZ385660 (subjects with moderate hepatic impairment)	eliglustat	Single dose of eliglustat tartrate will be given under fed conditions
GZ385660 (subjects with mild hepatic impairment)	eliglustat	Single dose of eliglustat tartrate will be given under fed conditions

Primary Outcome Measures

Name	Time	Method
Assessment of PK parameter: Maximum plasma concentration observed (Cmax)	3 days
Assessment of PK parameter: Area under the plasma concentration (AUC)	3 days

Secondary Outcome Measures

Name	Time	Method
Assessment of PK parameter: Area under the plasma concentration versus time curve (AUClast)	3 days
Assessment of PK parameter: Apparent total body clearance (CL/F)	3 days
Assessment of PK parameter: Apparent volume of distribution during the terminal phase (Vz/F)	3 days
Assessment of PK parameter: Terminal half-life (t1/2z)	3 days
Change from baseline in ECG parameter	Baseline, Up to 10 days
Number of adverse events	Up to 10 days
Assessment of PK parameter: Predicted accumulation ratio (Rac,pred)	3 days

Trial Locations

Locations (2)

Investigational Site Number 840002; 🇺🇸 Miami, Florida, United States

Investigational Site Number 840001; 🇺🇸 Knoxville, Tennessee, United States