End Stage Liver DiseasePhase 2NCT04496479
Allogenic Hepatocyte Transplantation Into Periduodenal Lymph Nodes
A Phase 2 study testing LYG-LIV0001 in people with End Stage Liver Disease, run by LyGenesis, Inc. at 2 sites in United States.
Trial summary
This Phase 2a clinical trial is a dose escalation study of the safety, tolerability, and efficacy of hepatocyte transplantation into lymph nodes via endoscopic ultrasound among subjects with end-stage liver disease. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- End Stage Liver Disease
- What is being tested?
- LYG-LIV0001
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesUnited States
- Study dates
- 11 Mar 2022to 30 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Have read, understood, and signed the informed consent form (ICF).
- Adults of either gender and ages 18 to 70 years old with a diagnosis of ESLD due to alcohol, chronic hepatitis B virus (HBV) and/or hepatitis C virus (HCV) infections, autoimmune hepatitis, primary sclerosis cholangitis, primary biliary cirrhosis (cholangitis), cirrhosis as the result of Wilson disease, hemochromatosis, sarcoidosis and alpha 1 antitrypsin deficiency, cryptogenic cirrhosis, and nonalcoholic steatohepatitis cirrhosis with a MELD-Na score >10 and <25 at screening.
- Subjects must have a body mass index (BMI) <35.
- Subjects with HCV associated ESLD must have been treated and demonstrate 24 weeks of negative HCV ribonucleic acid (RNA).
- Subjects with HBV must be on stable therapy for 6 months and have HBV deoxyribonucleic acid <500 c/mL.
- Women of childbearing potential (WOCBP) or sexual partners of male subjects who are WOCBP must be able and willing to use at least 1 highly effective method of contraception during the study and for 1 month after the last study visit. A female subject is considered to be a WOCBP after menarche and until she is in a postmenopausal state for 12 months or otherwise permanently sterile (for which acceptable methods include hysterectomy, bilateral salpingectomy, and bilateral oophorectomy; HMA, 2014). For the definition and list of highly effective methods of contraception, see Appendix 1.
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- Has stable control of portal hypertension and upper gastrointestinal bleeding with medical therapy and/or endoscopic therapy.
- If the subject has undergone a TIPS procedure for the clinical management of portal hypertension, they must be stable after the successful TIPS procedure, and not experiencing serious complications from the TIPS procedure itself (e.g., infection and intractable hepatic encephalopathy).
- Has blood urea nitrogen (BUN) <80 mg/dL.
- Has an estimated glomerular filtration rate (eGFR) ≥45 mL/min/1.73 m2.
- Agrees to avoid alcohol consumption during the study.
- Is willing and able to comply with all requirements of the study protocol.
Who cannot join
- Has primary hepatic neoplasms (hepatocellular carcinoma and cholangiocarcinoma).
- Has active and/or uncontrolled severe infections requiring hospitalization and prolonged antimicrobial therapy.
- Has severe coagulopathy (international normalized ratio [INR] >2, and/or platelet count <50,000/μL).
- Has psychiatric and/or social issues that could lead to noncompliance.
- Has an extrahepatic neoplastic disease requiring active chemotherapy, immunotherapy, and/or surgical resection.
- Has previously treated neoplastic disease with less than a 2-year cancer free period.
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- Pregnant and lactating women should not be in the study.
- Known hypersensitivity to human serum albumin.
- Subjects with uncontrolled hypertension (defined as a diastolic blood pressure of 110 mmHg or higher).
- Has recurrent/intractable ascites refractory to diuretics and requiring periodic large volume paracentesis.
- Has primary alcoholic liver disease and has not demonstrated abstinence for at least 24 weeks (6 months) prior to enrollment while attending mandatory rehab programs (e.g., Alcoholics Anonymous) and psychotherapy.
- Has grade 3 esophageal varices requiring the continuous use of propranolol and cannot afford to have this medication withheld and/or discontinued.
- Has a Child-Turcotte-Pugh (CTP) Class of C.
- Is receiving or plans to receive treatment with another investigational product or device.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 1 recruiting
- Withdrawn
Tufts Medical Center
Boston, Massachusetts, 02111
- Recruiting
Houston Methodist Hospital
Houston, Texas, 77030
Constance Mobley, MD
About this study
Official title: A Phase 2a, Open Label, Dose Escalation Study for Safety, Tolerability, and Efficacy of Hepatocyte Transplantation Into Periduodenal Lymph Nodes Among Subjects With End-Stage Liver Disease
This Phase 2a clinical trial is a dose escalation study of the safety, tolerability, and efficacy of hepatocyte transplantation into lymph nodes via endoscopic ultrasound among subjects with end-stage liver disease.
Read the detailed description
This safety, tolerability, and efficacy study includes an open-label dose-escalation phase for up to 12 subjects with end-stage liver disease (ESLD).
- Conditions
- End Stage Liver Disease
- Being tested
- LYG-LIV0001
- Keywords
- Allogeneic, Organogenesis, Hepatocyte, Transplantation, Liver Disease
- Medical subject headings
- End Stage Liver Disease, Liver Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LYG-LIV0001Biological
Allogenic hepatocytes suspended in a buffered cell preservation solution with increasing number of lymph nodes being transplanted for the dose escalation. Subjects will also receive immune suppression, including tacrolimus capsules to follow the dose prescribed by the investigator as well as a short course of prednisone.
| Study group | Role | What they receive |
|---|---|---|
| LYG-LIV0001 | Experimental | Open label group of subjects with end stage liver disease receiving increasing doses of the experimental therapy. Receives: Biological: LYG-LIV0001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dosage Selection
The primary objective of the dose escalation is to confirm the optimal dose of transplanted hepatocytes to safely achieve adequate allogeneic hepatocyte (AH) engraftment
Measured: Week 12
Safety of Engraftment of Hepatocytes in to Lymph Nodes
The primary safety objective of the dose escalation is to determine whether AH engraftments into the periduodenal lymph nodes in subjects with end-stage liver disease is safe as determined by the number/severity of adverse events
Measured: Week 12
Efficacy of Engraftment of Hepatocytes in to Lymph Nodes
The primary efficacy objective of the dose escalation is to determine whether AH engraftments into the periduodenal lymph nodes in subjects with end-stage liver disease is efficacious in addressing some of the signs and symptoms of end-stage liver disease
Measured: Week 12
Secondary outcomes (1)
Effectiveness of Selected Treatment to Modify the Liver Function Panel
Evaluate the effectiveness of hepatocyte transplants in modifying the liver function panel (total serum bilirubin, ammonia, prothrombin time, international normalized ratio, sodium, blood urea nitrogen, and creatinine) as measured through changes in laboratory biomarkers caused by end-stage liver disease
Measured: Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Aug 2020
- Study start11 Mar 2022
- Primary completion20 Aug 2027 (estimated)
- Study completion30 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for End Stage Liver DiseaseStudies 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
- Not applicable
- Intervention model
- Sequential
- Model description
- Open-Label Dose Escalation of Three Increasing Dosages
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- LyGenesis, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Paulo Fontes, MD, Study chair, LyGenesis, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04496479
- Sponsor's protocol ID
- LYG-LIV-02-01
- Status last verified
- Aug 2025
- First submitted
- 22 Jul 2020
- Last updated
- 8 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a, Open Label, Dose Escalation Study for Safety, Tolerability, and Efficacy of Hepatocyte Transplantation Into Periduodenal Lymph Nodes Among Subjects With End-Stage Liver Disease
- ClinicalTrials.gov ID
- NCT04496479
- Acronym / sponsor ID
- LYG-LIV-02-01
- Conditions
- End Stage Liver Disease
- Keywords
- Allogeneic, Organogenesis, Hepatocyte, Transplantation, Liver Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- LyGenesis, Inc.Industry (pharma, biotech, devices)
- Study start
- 11 Mar 2022 (actual)
- Primary completion
- 20 Aug 2027 (estimated)
- Study completion
- 30 Dec 2027 (estimated)
- First posted
- 3 Aug 2020 (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 Phase 2a clinical trial is a dose escalation study of the safety, tolerability, and efficacy of hepatocyte transplantation into lymph nodes via endoscopic ultrasound among subjects with end-stage liver disease.
Detailed description
This safety, tolerability, and efficacy study includes an open-label dose-escalation phase for up to 12 subjects with end-stage liver disease (ESLD).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-Label Dose Escalation of Three Increasing Dosages
| Arm | Type | Receives |
|---|---|---|
| LYG-LIV0001 | Experimental | Biological: LYG-LIV0001Open label group of subjects with end stage liver disease receiving increasing doses of the experimental therapy. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Have read, understood, and signed the informed consent form (ICF).
- Adults of either gender and ages 18 to 70 years old with a diagnosis of ESLD due to alcohol, chronic hepatitis B virus (HBV) and/or hepatitis C virus (HCV) infections, autoimmune hepatitis, primary sclerosis cholangitis, primary biliary cirrhosis (cholangitis), cirrhosis as the result of Wilson disease, hemochromatosis, sarcoidosis and alpha 1 antitrypsin deficiency, cryptogenic cirrhosis, and nonalcoholic steatohepatitis cirrhosis with a MELD-Na score >10 and <25 at screening.
- Subjects must have a body mass index (BMI) <35.
- Subjects with HCV associated ESLD must have been treated and demonstrate 24 weeks of negative HCV ribonucleic acid (RNA).
- Subjects with HBV must be on stable therapy for 6 months and have HBV deoxyribonucleic acid <500 c/mL.
Key exclusion criteria (14 listed)
- Has primary hepatic neoplasms (hepatocellular carcinoma and cholangiocarcinoma).
- Has active and/or uncontrolled severe infections requiring hospitalization and prolonged antimicrobial therapy.
- Has severe coagulopathy (international normalized ratio [INR] >2, and/or platelet count <50,000/μL).
- Has psychiatric and/or social issues that could lead to noncompliance.
- Has an extrahepatic neoplastic disease requiring active chemotherapy, immunotherapy, and/or surgical resection.
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 |
|---|---|---|
| LYG-LIV0001Allogenic hepatocytes suspended in a buffered cell preservation solution with increasing number of lymph nodes being transplanted for the dose escalation. Subjects will also receive immune suppression, including tacrolimus capsules to follow the dose prescribed by the investigator as well as a short course of prednisone. | Biological | LYG-LIV0001 |
Primary outcome
Dosage Selection
Time frame: Week 12
The primary objective of the dose escalation is to confirm the optimal dose of transplanted hepatocytes to safely achieve adequate allogeneic hepatocyte (AH) engraftment
Safety of Engraftment of Hepatocytes in to Lymph Nodes
Time frame: Week 12
The primary safety objective of the dose escalation is to determine whether AH engraftments into the periduodenal lymph nodes in subjects with end-stage liver disease is safe as determined by the number/severity of adverse events
Efficacy of Engraftment of Hepatocytes in to Lymph Nodes
Time frame: Week 12
The primary efficacy objective of the dose escalation is to determine whether AH engraftments into the periduodenal lymph nodes in subjects with end-stage liver disease is efficacious in addressing some of the signs and symptoms of end-stage liver disease
1 secondary outcome
Effectiveness of Selected Treatment to Modify the Liver Function Panel
Time frame: Week 52
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Aug 2025.
- Individual participant dataSharing planned: No
Related trials for End Stage Liver Disease
Not yet recruiting
Double Plasma Separation and Adsorption in Acute-on-Chronic Liver Failure (DPMAS-ACLF Trial)
Acute-on-Chronic Liver Failure (ACLF)
Not yet recruiting
Vitamin C for Acute Kidney Injury in ACLF With Septic Shock: A Randomized Controlled Trial
Acute-on-Chronic Liver Failure (ACLF)
Active, not recruiting
LiverPAL: A Trial of Inpatient Palliative Care for Patients With Advanced Liver Disease
Liver Disease Chronic, End Stage Liver DIsease
RecruitingObservational study
Establishing a Reference Framework for Outcomes After Machine-Preserved Liver Transplantation in Europe
End-stage Liver Disease (ESLD), Acute Liver Failure
RecruitingObservational study
CytOSorb TreatMent Of Critically Ill PatientS Registry
Septic Shock, Acute Respiratory Distress Syndrome
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.
Paulo Fontes, MD study chair
LyGenesis, Inc.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing LYG-LIV0001 in people with End Stage Liver Disease, run by LyGenesis, Inc. at 2 sites in United States.
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 12 inclusion and 14 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: looking for participants now, as of the sponsor's last update on 8 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Paulo Fontes, MD (412-860-3599, fontesp@lygenesis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dosage Selection (time frame: Week 12). The study lists 3 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 8 Aug 2025.