Hepatic EncephalopathyPhase 2NCT06987968
A Study to See if an Investigational Medicine Called VS-01 Can Help and How Safe it is in the Treatment of Patients With Overt Hepatic Encephalopathy
A Phase 2 study testing VS-01 on top of SOC (Active Treatment Group) and SOC in people with Hepatic Encephalopathy and Acute on Chronic Liver Failure (ACLF), run by Genfit at 6 sites in 3 countries.
Trial summary
A Phase 2, Randomized, Controlled, Open-Label, Adaptive Dose Design, Proof-of-Concept Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Two Different Dwell Times of VS-01 on Top of Standard of Care versus Standard of Care Alone in Patients with Overt Hepatic Encephalopathy Read more about this study
- Who is it for?
- Ages 18 to 79 years
- Conditions the study is for
- Hepatic Encephalopathy, Acute on Chronic Liver Failure (ACLF), Decompensated Cirrhosis
- What is being tested?
- VS-01 on top of SOC (Active Treatment Group) and SOC
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 6 sitesin 3 countries
- Study dates
- 25 Aug 2025to 15 Sep 2025
- Phase
- Phase 2
- 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
- 1)Patients with liver cirrhosis of any underlying etiology (liver cirrhosis diagnosed by standard clinical criteria, imaging findings and/or histology), who are diagnosed with Overt hepatic encephalopathy (OHE) (according to Hepatic Encephalopathy Grading Instrument) in the presence of:
- Acute decompensation (AD)(defined as the onset or worsening of ascites, hepatic encephalopathy, gastrointestinal (GI) bleeding or any combination of them with or without infection); or
- Acute on chronic liver failure (ACLF) grade 1 according to European Association for the Study of the Liver(EASL-CLIF) criteria; 2)Presence of ascites requiring diagnostic or therapeutic paracentesis; 3)Fasting blood ammonia > upper limit of normal (ULN) at baseline (BL); 4)Patients with a dry body weight ≥40 kg and <140 kg; 5)Male and female patients ≥18 to <80 years of age on the day of signing the informed consent form (ICF); 6)Patients willing and able to provide written informed consent. If the patient is unable to fully understand or sign the written informed consent based on the Investigator's judgment, the ICF must be signed by a legal representative of the patient according to local regulation.
Who cannot join
- ACLF grade 2 or higher as defined by European Association for the Study of the Liver-Chronic Liver Failure-Chronic Liver Failure (EASL-CLIF) criteria;
- Presence of spontaneous or secondary bacterial peritonitis (i.e., neutrophil counts >250/mm3 in ascitic fluid);
- Contraindication for paracentesis according to the European Association for the Study of the Liver (EASL) Clinical Practice Guidelines 2018, and American Association for the Study of Liver Diseases (AASLD)Guideline on the Treatment of Ascites, Spontaneous Bacterial Peritonitis, and Hepatorenal Syndrome 2021;
- Alfapump® in place to manage ascites;
- Known hypersensitivity to liposomes, history of mastocytosis, multiple hypersensitivities or similar diseases known to be associated with an increased risk of allergic/anaphylactoid reactions;
- Upper GI bleeding within the last 7 days prior to BL, acute bleeding or bleeding upon paracentesis at Screening (SCR) or Baseline (BL);
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- Poorly controlled seizure disorder;
- Respiratory failure requiring invasive mechanical ventilation;
- Severe circulatory failure requiring the use of high dose vasopressors (e.g., dopamine >15 μg/kg/min, or epinephrine >0.1 μg/kg/min, or norepinephrine >0.1 μg/kg/min); the use of terlipressin or low-dose norepinephrine to treat hepatorenal syndrome is not an exclusion criterion;
- Uncontrolled severe infection with hemodynamic instability or shock; patients may be enrolled provided anti-infectives have been administered for at least 48 hours prior to BL with an appropriate response as assessed by the Principal Investigator (PI);
- Need for Renal replacement therapy (RRT) or any extracorporeal liver support device;
- Any significant disease considered to be potentially detrimental or would preclude the patient from participating in and completing the study as assessed by the PI. This includes but is not limited to hepatocellular carcinoma outside Milan criteria, cholangiocarcinoma, extrahepatic cancer over the past 2 years, or people who inject drugs;
- Individuals for whom the PI deems that study participation would be unsafe or not in the interest of the patient;
- Pregnancy or lactation;
- Women of childbearing potential and non-sterile male patients who are not willing to use adequate contraception from SCR to 30 days after the final dose of investigational medicinal product (IMP);
- Participation in another interventional clinical trial within 30 days of SCR.
Trial locations and contacts
6 sites in 3 countries.
6 sites in 3 countries
Georgia4 sites
Georgian Clinics" Kutaisi Referral Hospital 2, Otskheli str.
Kutaisi, 4600
LEPL The First University Clinic of Tbilisi State, Medical University, Gudamakari str.4,,LEPL The First University Clinic
Tbilisi, 0141
LLC Geo Hospitals Tbilisi, Multiprofile Medical Center, Tsinandali ,str. 9/3
Tbilisi, 0144
Tbilisi State Medical University and Ingorokva, High Medical Technology University Clinic, Tsinandali Street
Tbilisi, 0144
United States1 site
96 Jonathan Lucas St, CSB (Main Hospital), Suite 908
Charleston, South Carolina, 29425
France1 site
Nouvel Hôpital Civil, Service de Medecine Intensive Reanimation, 1, place de l'Hôpital
Strasbourg, 67091
About this study
Official title: A Phase 2, Randomized, Controlled, Open-Label, Adaptive Dose Design, Proof-of-Concept Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Two Different Dwell Times of VS-01 on Top of Standard of Care Versus Standard of Care Alone in Patients With Overt Hepatic Encephalopathy
A Phase 2, Randomized, Controlled, Open-Label, Adaptive Dose Design, Proof-of-Concept Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Two Different Dwell Times of VS-01 on Top of Standard of Care versus Standard of Care Alone in Patients with Overt Hepatic Encephalopathy
- Being tested
- VS-01 on top of SOC (Active Treatment Group), SOC
- Keywords
- Liver, ammonia, toxic substances, chronic liver disease, cirrhosis, VS-01, Brain, Injury, Overt hepatic encephalopathy (OHE), Acute Decompensation (AD), Standard of Care (SOC), Acute-on-Liver Failure (ACLF)
- Medical subject headings
- Hepatic Encephalopathy, Acute-On-Chronic Liver Failure, Fibrosis, Wounds and Injuries
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VS-01 on top of SOC (Active Treatment Group)Drug
Patients will received VS-01 intraperitoneally up to four consecutive days on top of SOC
SOCDrug
Patients will received SOC
| Study group | Role | What they receive |
|---|---|---|
| VS-01 with 3 hours Dwell time+ Standard of Care (SOC) | Experimental | Patients randomized to Active Treatment group will receive VS-01 3 hours Dwell time on top of SOC Receives: Drug: VS-01 on top of SOC (Active Treatment Group) |
| VS-01 with 4 hours Dwell time+SOC | Experimental | Patients randomized to Active Treatment group will receive VS-01 4 hours Dwell time on top of SOC Receives: Drug: VS-01 on top of SOC (Active Treatment Group) |
| SOC | Other | Patients randomized to Control group will receive SOC alone Receives: Drug: SOC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to improvement of OHE as assessed by HEGI
Measured: from randomization and up to Day 5 morning
Secondary outcomes (3)
The incidence and severity ([NCI CTCAE] v5.0 grade) of treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs), comprising the evaluation of vital signs, ECG parameters, and laboratory parameters
[NCI CTCAE] v5.0 grade: National Cancer Institute Common Terminology Criteria for Adverse Events
Measured: from randomization and up to Day 14 (more or less 3 days)
Pharmacokinetics (PD) (for all arms): change from Baseline (BL) in fasting blood and peritoneal fluid ammonia levels.
Measured: from randomization up to Day 14 (more or less 3 days)
Pharmacokinetics (PK) (for active treatment arms): blood and peritoneal fluid concentration-time profile of baseline (BL) corrected citric acid and lipids in patients treated with VS-01.
Measured: From randomization up to Day 5 or EOT (only for patients treated during 4 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 May 2025
- Study start25 Aug 2025
- Primary completion8 Sep 2025
- Study completion15 Sep 2025
Why it stopped: Sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hepatic EncephalopathyStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genfit
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pejvack MOTLAGH, M.D, M.Sc, Study director, Genfit
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06987968
- Sponsor's protocol ID
- VS01_P2_24_2
- Other IDs
- 2025-521029-34-00, 2025
- Status last verified
- Dec 2025
- First submitted
- 7 May 2025
- Last updated
- 12 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Controlled, Open-Label, Adaptive Dose Design, Proof-of-Concept Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Two Different Dwell Times of VS-01 on Top of Standard of Care Versus Standard of Care Alone in Patients With Overt Hepatic Encephalopathy
- ClinicalTrials.gov ID
- NCT06987968
- Acronym / sponsor ID
- VS01_P2_24_2
- Keywords
- Liver, ammonia, toxic substances, chronic liver disease, cirrhosis, VS-01, Brain, Injury
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision
- Enrolment
- 3 participants (actual)
- Lead sponsor
- GenfitIndustry (pharma, biotech, devices)
- Study start
- 25 Aug 2025 (actual)
- Primary completion
- 8 Sep 2025 (actual)
- Study completion
- 15 Sep 2025 (actual)
- First posted
- 23 May 2025 (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.
A Phase 2, Randomized, Controlled, Open-Label, Adaptive Dose Design, Proof-of-Concept Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Two Different Dwell Times of VS-01 on Top of Standard of Care versus Standard of Care Alone in Patients with Overt Hepatic Encephalopathy
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| VS-01 with 3 hours Dwell time+ Standard of Care (SOC) | Experimental | Drug: VS-01 on top of SOC (Active Treatment Group)Patients randomized to Active Treatment group will receive VS-01 3 hours Dwell time on top of SOC |
| VS-01 with 4 hours Dwell time+SOC | Experimental | Drug: VS-01 on top of SOC (Active Treatment Group)Patients randomized to Active Treatment group will receive VS-01 4 hours Dwell time on top of SOC |
| SOC | Other | Drug: SOCPatients randomized to Control group will receive SOC alone |
Who may qualify
- Ages
- 18 to 79 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 1)Patients with liver cirrhosis of any underlying etiology (liver cirrhosis diagnosed by standard clinical criteria, imaging findings and/or histology), who are diagnosed with Overt hepatic encephalopathy (OHE) (according to Hepatic Encephalopathy Grading Instrument) in the presence of:
Key exclusion criteria (16 listed)
- ACLF grade 2 or higher as defined by European Association for the Study of the Liver-Chronic Liver Failure-Chronic Liver Failure (EASL-CLIF) criteria;
- Presence of spontaneous or secondary bacterial peritonitis (i.e., neutrophil counts >250/mm3 in ascitic fluid);
- Contraindication for paracentesis according to the European Association for the Study of the Liver (EASL) Clinical Practice Guidelines 2018, and American Association for the Study of Liver Diseases (AASLD)Guideline on the Treatment of Ascites, Spontaneous Bacterial Peritonitis, and Hepatorenal Syndrome 2021;
- Alfapump® in place to manage ascites;
- Known hypersensitivity to liposomes, history of mastocytosis, multiple hypersensitivities or similar diseases known to be associated with an increased risk of allergic/anaphylactoid reactions;
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 |
|---|---|---|
| VS-01 on top of SOC (Active Treatment Group)Patients will received VS-01 intraperitoneally up to four consecutive days on top of SOC | Drug | VS-01 with 3 hours Dwell time+ Standard of Care (SOC), VS-01 with 4 hours Dwell time+SOC |
| SOCPatients will received SOC | Drug | SOC |
Primary outcome
Time to improvement of OHE as assessed by HEGI
Time frame: from randomization and up to Day 5 morning
3 secondary outcomes
The incidence and severity ([NCI CTCAE] v5.0 grade) of treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs), comprising the evaluation of vital signs, ECG parameters, and laboratory parameters
Time frame: from randomization and up to Day 14 (more or less 3 days)
Pharmacokinetics (PD) (for all arms): change from Baseline (BL) in fasting blood and peritoneal fluid ammonia levels.
Time frame: from randomization up to Day 14 (more or less 3 days)
Pharmacokinetics (PK) (for active treatment arms): blood and peritoneal fluid concentration-time profile of baseline (BL) corrected citric acid and lipids in patients treated with VS-01.
Time frame: From randomization up to Day 5 or EOT (only for patients treated during 4 days)
Results and safety
The study was stopped early (reason given: Sponsor decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jan 2026.
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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.
Pejvack MOTLAGH, M.D, M.Sc study director
Genfit
6 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing VS-01 on top of SOC (Active Treatment Group) and SOC in people with Hepatic Encephalopathy and Acute on Chronic Liver Failure (ACLF), run by Genfit at 6 sites in 3 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 3 inclusion and 16 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: stopped early, as of the sponsor's last update on 12 Jan 2026. 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 Georgia, United States, France. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 was stopped early (reason given: Sponsor decision). Results are not available in the source record.
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?
Time to improvement of OHE as assessed by HEGI (time frame: from randomization and up to Day 5 morning). 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 12 Jan 2026; AzTrials last refreshed its copy on 7 Nov 2026.