Ignite Creation Date:
2025-12-25 @ 12:13 AM
Ignite Modification Date:
2026-03-05 @ 12:49 AM
Study NCT ID:
NCT05718258
Status:
COMPLETED
Last Update Posted:
2025-09-15
First Post:
2023-01-30
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study in Adults to Investigate the Impact of Mild, Moderate, and Severe Hepatic Impairment on Pharmacokinetics of Venglustat Compared to Participants With Normal Hepatic Function
Sponsor:
Sanofi
Organization:
Study Overview
Official Title:
A Phase 1, Multi-center, Parallel, Open-label, Pharmacokinetic, Safety, and Tolerability Study of Venglustat Given as a Single Dose in Adult Participants With Mild, Moderate, and Severe Hepatic Impairment and in Matched Participants With Normal Hepatic Function
Status:
COMPLETED
Status Verified Date:
2025-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a parallel, Phase 1, four arm, open-label, single dose, multicenter study to evaluate the impact of hepatic impairment on venglustat exposure following treatment with venglustat.
The purpose of this study is to assess the effect of mild, moderate, and severe hepatic impairment on PK, safety, and tolerability of venglustat compared with normal hepatic function in male and female participants aged 18 to 79 years.
Study details include:
* The total study duration per participant will be up to 42 days, including up to 21 days for screening and approximately 21 days from institutionalization to the end of study (EOS).
* Institutionalization is mandatory until the activities on D5 have been completed.
* Each participant will receive a single dose of venglustat.
* For hepatically impaired participants there will be a screening visit, a multi-day institutionalization visit, and 7 site visits after D5 discharge, including the end of study (EOS) visit.
* For healthy volunteers there will be a screening visit, a multi-day institutionalization visit and 3 site visits after D5 discharge, including the end of study (EOS) visit.
The purpose of this study is to assess the effect of mild, moderate, and severe hepatic impairment on PK, safety, and tolerability of venglustat compared with normal hepatic function in male and female participants aged 18 to 79 years.
Study details include:
* The total study duration per participant will be up to 42 days, including up to 21 days for screening and approximately 21 days from institutionalization to the end of study (EOS).
* Institutionalization is mandatory until the activities on D5 have been completed.
* Each participant will receive a single dose of venglustat.
* For hepatically impaired participants there will be a screening visit, a multi-day institutionalization visit, and 7 site visits after D5 discharge, including the end of study (EOS) visit.
* For healthy volunteers there will be a screening visit, a multi-day institutionalization visit and 3 site visits after D5 discharge, including the end of study (EOS) visit.
Detailed Description:
The duration of the study for a participant in any arm will be up to 42 days. The treatment and follow up periods will last for a combined total of approximately 20 days, while the screening period will be up to 3 weeks, but may be shorter on an individual basis.
Screening period: up to 3 weeks (Days -21 to -2).
Open-label treatment period with compulsory institutionalization (Days -1 to 5).
Follow up period lasting until approximately day 20 ± 2 days post administration. This will include 7 additional site visits (including the EOS visit) for hepatically impaired groups. The group with normal hepatic function will only need to return D6 and D7 for study visit after institutionalization is completed.
Screening period: up to 3 weeks (Days -21 to -2).
Open-label treatment period with compulsory institutionalization (Days -1 to 5).
Follow up period lasting until approximately day 20 ± 2 days post administration. This will include 7 additional site visits (including the EOS visit) for hepatically impaired groups. The group with normal hepatic function will only need to return D6 and D7 for study visit after institutionalization is completed.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?: