Hepatic Function AbnormalPhase 1NCT05718258
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
A Phase 1 study testing Venglustat (GZ402671) in people with Hepatic Function Abnormal, run by Sanofi at 6 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 79 years
- Conditions the study is for
- Hepatic Function Abnormal
- What is being tested?
- Venglustat (GZ402671)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 6 sitesin 2 countries
- Study dates
- 18 Jan 2023to 29 Jan 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 79 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant must be 18 to 79 years of age inclusive, at the time of signing the informed consent.
- Body weight between 50.0 and 115.0 kg, inclusive, if male; between 40.0 and 100 kg, inclusive, if female, and body mass index (BMI) within the range 18 to 40 kg/m2, inclusive, at screening.
- Contraception (with double contraception methods) for male and female participants; not pregnant or breastfeeding for female participants; no sperm donation for male participants.
- Female participants must refrain from donating ova/ovum.
- Capable of giving signed informed consent.
- Participants with mild, moderate, and severe hepatic impairment
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- Stable chronic liver disease assessed by medical history, physical examination, and laboratory values. Hepatic impairment will be assessed according to the Child-Pugh classification, using CPS (Mild hepatic impairment is defined as a total score ranging from 5 to 6, inclusive (CPS-A); Moderate hepatic impairment is defined as a total score ranging from 7 to 9, inclusive (CPS-B); and severe hepatic impairment is defined as a total score ranging from 10 to 15, inclusive (CPS-C).
- Laboratory parameters within the acceptable range for participants with hepatic impairment; however, serum creatinine should be strictly at or below the upper laboratory norm and eGFR should be >= 60 mL/min.
- Participants with normal hepatic function
- Certified as healthy by a comprehensive clinical assessment (detailed medical history and complete physical examination).
- Laboratory parameters within the reference range for healthy participants.
Who cannot join
- Participant who had severe course of COVID-19 (i.e., hospitalization, extracorporeal membrane oxygenation, mechanically ventilated) within the past 8 weeks.
- History of current recreational drugs or alcohol abuse.
- Smoking more than 15 cigarettes or equivalent per day.
- Symptomatic postural hypotension, whatever the decrease in blood pressure, or asymptomatic postural hypotension defined as a decrease in SBP ≥30 mmHg within 3 minutes when changing from supine to standing position at screening and Day 1.
- Blood donation within 2 months before inclusion.
- Positive result on drug or alcohol screen.
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- Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study.
- Any consumption of citrus fruits (grapefruit, orange, etc) or their juices within 5 days before inclusion).
- Any participant who cannot comply with the following study restrictions: refraining from drinking alcohol, tea, coffee, chocolate, quinine, or caffeine-containing beverages from 1 day before institutionalization and throughout the study duration; following a stable lifestyle with no intensive physical activity from 1 day prior to institutionalization throughout the study duration until after collection of the final PK sample.
- Positive result on anti-human immunodeficiency virus (HIV) or COVID-19 test.
- Participants with mild, moderate, and severe hepatic impairment
- Hepatocellular carcinoma.
- Acute hepatitis.
- Uncontrolled or decompensated hepatic encephalopathy. Encephalopathy grade > 2.
- Any drug (within 14 days before inclusion or within 5 times the elimination half-life or pharmacodynamic half-life of the medication) which could impact by any mechanism of action, the pharmacokinetics of the investigational medicinal product, including moderate and strong CYP3A4 inhibitors or inducers.
- Any participant enrolled in or having participated in this or any other clinical study involving an IMP or in any other type of medical research according to applicable regulations within the past 14 days or 5 half-lives before screening, whichever is longer, per local regulations.
- Participants with normal hepatic functions
- Any history or presence of clinically relevant hepatic or renal disease.
- Any medication (including St John's Wort) within 14 days before inclusion or within 5 times the elimination half-life or pharmacodynamic half-life of the medication, with the exception of hormonal contraception or menopausal hormone replacement therapy; any vaccination within the last 28 days and any biologics (antibody or its derivatives) given within 4 months before inclusion.
- Current enrollment OR past participation in another investigational study in which an investigational intervention (eg, drug, vaccine, invasive device) was administered within the last 14 days or 5 half-lives days before screening, whichever is longer per local regulations.
- The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Trial locations and contacts
6 sites in 2 countries.
6 sites in 2 countries
United States5 sites
Volunteer Research Group-NOCCR Site Number : 8400003
Knoxville, Tennessee, 37920
Clinical Pharmacology of Miami Site Number : 8400002
Miami, Florida, 33014
Inland Empire Liver Foundation Site Number : 8400004
Rialto, California, 92377
Nucleus Network Site Number : 8400001
Saint Paul, Minnesota, 55114
American Research Corporation Site Number : 8400005
San Antonio, Texas, 78215
About this study
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
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.
Read more
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.
Read the 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.
- Conditions
- Hepatic Function Abnormal
- Being tested
- Venglustat (GZ402671)
- Medical subject headings
- Liver Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Venglustat (GZ402671)Drug
Pharmaceutical form: tablet
Route of administration: oral
| Study group | Role | What they receive |
|---|---|---|
| Participants with mild hepatic impairment | Experimental | Venglustat single dose on Day 1 Receives: Drug: Venglustat (GZ402671) |
| Participants with normal hepatic function | Experimental | Venglustat single dose on Day 1 Receives: Drug: Venglustat (GZ402671) |
| Participants with moderate hepatic impairment | Experimental | Venglustat single dose on Day 1 Receives: Drug: Venglustat (GZ402671) |
| Participants with severe hepatic impairment | Experimental | Venglustat single dose on Day 1 Receives: Drug: Venglustat (GZ402671) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum venglustat plasma concentration observed (Cmax)
Measured: Baseline to Day 20
Area under the plasma concentration versus time curve extrapolated to infinity (AUC)
Measured: Baseline to Day 20
Secondary outcomes (2)
Time to maximum venglustat plasma concentration (tmax)
Measured: Baseline to Day 20
Number of participants with adverse event (AE), treatment emergent adverse events (TEAEs), serious adverse events (SAEs) and adverse events of special interest (AESI)
Measured: Baseline to Day 20
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Feb 2023
- Study start18 Jan 2023
- Primary completion29 Jan 2024
- Study completion29 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hepatic Function AbnormalStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05718258
- Sponsor's protocol ID
- POP16551
- Other IDs
- U1111-1261-7500, 2022-000945-34
- Status last verified
- Sep 2025
- First submitted
- 30 Jan 2023
- Last updated
- 15 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05718258
- Acronym / sponsor ID
- POP16551
- Conditions
- Hepatic Function Abnormal
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 18 Jan 2023 (actual)
- Primary completion
- 29 Jan 2024 (actual)
- Study completion
- 29 Jan 2024 (actual)
- First posted
- 8 Feb 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Participants with mild hepatic impairment | Experimental | Drug: Venglustat (GZ402671)Venglustat single dose on Day 1 |
| Participants with normal hepatic function | Experimental | Drug: Venglustat (GZ402671)Venglustat single dose on Day 1 |
| Participants with moderate hepatic impairment | Experimental | Drug: Venglustat (GZ402671)Venglustat single dose on Day 1 |
| Participants with severe hepatic impairment | Experimental | Drug: Venglustat (GZ402671)Venglustat single dose on Day 1 |
Who may qualify
- Ages
- 18 to 79 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Participant must be 18 to 79 years of age inclusive, at the time of signing the informed consent.
- Body weight between 50.0 and 115.0 kg, inclusive, if male; between 40.0 and 100 kg, inclusive, if female, and body mass index (BMI) within the range 18 to 40 kg/m2, inclusive, at screening.
- Contraception (with double contraception methods) for male and female participants; not pregnant or breastfeeding for female participants; no sperm donation for male participants.
- Female participants must refrain from donating ova/ovum.
- Capable of giving signed informed consent.
Key exclusion criteria (21 listed)
- Participant who had severe course of COVID-19 (i.e., hospitalization, extracorporeal membrane oxygenation, mechanically ventilated) within the past 8 weeks.
- History of current recreational drugs or alcohol abuse.
- Smoking more than 15 cigarettes or equivalent per day.
- Symptomatic postural hypotension, whatever the decrease in blood pressure, or asymptomatic postural hypotension defined as a decrease in SBP ≥30 mmHg within 3 minutes when changing from supine to standing position at screening and Day 1.
- Blood donation within 2 months before inclusion.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Venglustat (GZ402671)Pharmaceutical form: tablet Route of administration: oral | Drug | Participants with mild hepatic impairment, Participants with moderate hepatic impairment, Participants with normal hepatic function, Participants with severe hepatic impairment |
Primary outcome
Maximum venglustat plasma concentration observed (Cmax)
Time frame: Baseline to Day 20
Area under the plasma concentration versus time curve extrapolated to infinity (AUC)
Time frame: Baseline to Day 20
2 secondary outcomes
Time to maximum venglustat plasma concentration (tmax)
Time frame: Baseline to Day 20
Number of participants with adverse event (AE), treatment emergent adverse events (TEAEs), serious adverse events (SAEs) and adverse events of special interest (AESI)
Time frame: Baseline to Day 20
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Sep 2025.
- POP16551 Plain Language Results SummaryLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
6 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Venglustat (GZ402671) in people with Hepatic Function Abnormal, run by Sanofi at 6 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States, Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum venglustat plasma concentration observed (Cmax) (time frame: Baseline to Day 20). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Sep 2025.