Donor-Alloantigen-Reactive Regulatory T Cell (darTregs) in Liver Transplantation

NCT ID: NCT02188719

Last Updated: 2020-09-22

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-12-17

Study Completion Date

2019-06-18

Brief Summary

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The purpose of this study is look at the safety of:

* Taking a specific combination of immunosuppressant drugs after liver transplantation
* Receiving one of three different doses of donor-alloantigen-reactive regulatory T cells (darTregs) while taking this specific combination of drugs

Detailed Description

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After liver transplantation, immunosuppressants must be taken every day to prevent the body from injuring the transplanted liver by a process called rejection. People who take these drugs may experience side effects.

Studies show that some of body's cells, called T regulatory cells (Tregs), may play a part in accepting the transplanted liver. The investigators are learning about whether scientists can take Tregs from the blood of a liver transplant recipient and teach them to protect the transplanted liver from rejection. In the laboratory, the recipient Tregs are exposed to cells from the liver donor. Research data suggests that giving these "donor reactive" Tregs back to the transplant recipient might allow a liver transplant recipient to take lower doses of immunosuppressants, or perhaps to stop them altogether, without rejecting the liver.

Conditions

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Liver Transplantation

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1 - Treg-supportive IS only

3 subjects (Cohort 1a) at site 1 (UCSF) and 3 subjects (Cohort 1b) from site 2 (Mayo Rochester) will receive Treg-Supportive immunosuppression (IS) regimen and will not receive Donor-Alloantigen-Reactive T Regulatory Cells (darTregs). Progression from one cohort to the next will depend on the cumulative incidence of sentinel adverse events.

Group Type EXPERIMENTAL

Anti-Thymocyte Globulin - Rabbit

Intervention Type BIOLOGICAL

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Subjects will be given a dose range of 3.0-4.5 mg/kg total, in divided doses of 1.5 mg/kg/day. Subjects who meet eligibility criteria for Thymoglobulin® administration will be given 1.5 mg/kg intravenously (IV) on post-operative day 3, within 72 hours of transplantation. Additional doses of 1.5 mg/kg IV will be administered until CD3 count is \<50/mm\^3 or when the maximal dose of 4.5/mg/kg has been given.

Everolimus

Intervention Type DRUG

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Subjects meeting eligibility criteria for Treg-supportive IS regimen will begin EVR no sooner than 30 days after liver transplantation and no later than 44 days after transplantation; with target trough levels of 6-8 μg/L.EVR target trough levels will be further reduced to 4-6 μg/L 24 - 26 weeks after transplantation.

Tacrolimus

Intervention Type DRUG

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Between 30 and 44 days following transplant: Subjects who are not eliminated by Exclusion Criteria C1 (Protocol Section 14.3.1) will proceed in the study and receive either TAC-based or EVR- based IS, based on eligibility Criteria C2 (Section 4.3.4 )

* TAC-based IS: reduce TAC trough level to 3-8 μg/dL; continue MMF
* EVR-based IS: reduce TAC trough level to 3-8 μg/dL; EVR target trough level of 6-8 μg/dL; decrease then discontinue MMF

Mycophenolate mofetil

Intervention Type DRUG

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. 1000 mg total daily dose. MMF will be initiated within 24 hours of transplantation. MMF must be discontinued as soon as target EVR trough levels have been achieved.

Prednisone

Intervention Type DRUG

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Solumedrol 500 mg will be given IV on the day of transplantation. Additional Solumedrol will be prescribed according to site-specific standard of care. Oral prednisone should be initiated once oral medication is tolerated.

Anti-Infective Prophylaxis

Intervention Type DRUG

Intravenous ganciclovir and/or oral Valcyte will be administered for the prophylaxis of cytomegalovirus (CMV) and Epstein-Barr Virus (EBV) for at least six months after liver transplantation.

Blood draws

Intervention Type PROCEDURE

Blood draws are necessary to carefully and frequently evaluate allograft function after liver transplantation and treatment with Treg-supportive IS as well as after darTreg infusion. Peripheral blood samples will be collected and analyzed per protocol throughout subject participation in this study.

Liver biopsies

Intervention Type PROCEDURE

Subjects will have a liver biopsy for this study 12-14 weeks after transplantation. For subjects receiving darTregs, a second biopsy will be performed 7-10 days after darTregs infusion.

Liver transplantation

Intervention Type PROCEDURE

Inclusion in this trial is in the setting of subjects defined as having end-stage liver disease and listed for primary solitary liver transplant.

Cohort 2 - darTreg infusion,50 million(range 25 to 60 million)

At least 3 subjects will receive a single infusion of 50 million darTregs. Progression from one cohort to the next will depend on the cumulative incidence of sentinel adverse events.

Group Type EXPERIMENTAL

Anti-Thymocyte Globulin - Rabbit

Intervention Type BIOLOGICAL

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Subjects will be given a dose range of 3.0-4.5 mg/kg total, in divided doses of 1.5 mg/kg/day. Subjects who meet eligibility criteria for Thymoglobulin® administration will be given 1.5 mg/kg intravenously (IV) on post-operative day 3, within 72 hours of transplantation. Additional doses of 1.5 mg/kg IV will be administered until CD3 count is \<50/mm\^3 or when the maximal dose of 4.5/mg/kg has been given.

darTreg Infusion

Intervention Type BIOLOGICAL

A single dose darTreg infusion (Cohorts 2 - 4) will be received as per protocol.

Everolimus

Intervention Type DRUG

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Subjects meeting eligibility criteria for Treg-supportive IS regimen will begin EVR no sooner than 30 days after liver transplantation and no later than 44 days after transplantation; with target trough levels of 6-8 μg/L.EVR target trough levels will be further reduced to 4-6 μg/L 24 - 26 weeks after transplantation.

Tacrolimus

Intervention Type DRUG

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Between 30 and 44 days following transplant: Subjects who are not eliminated by Exclusion Criteria C1 (Protocol Section 14.3.1) will proceed in the study and receive either TAC-based or EVR- based IS, based on eligibility Criteria C2 (Section 4.3.4 )

* TAC-based IS: reduce TAC trough level to 3-8 μg/dL; continue MMF
* EVR-based IS: reduce TAC trough level to 3-8 μg/dL; EVR target trough level of 6-8 μg/dL; decrease then discontinue MMF

Mycophenolate mofetil

Intervention Type DRUG

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. 1000 mg total daily dose. MMF will be initiated within 24 hours of transplantation. MMF must be discontinued as soon as target EVR trough levels have been achieved.

Prednisone

Intervention Type DRUG

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Solumedrol 500 mg will be given IV on the day of transplantation. Additional Solumedrol will be prescribed according to site-specific standard of care. Oral prednisone should be initiated once oral medication is tolerated.

Acetaminophen

Intervention Type DRUG

Pre-medication for single dose darTreg infusion (Cohorts 2 - 4). 650mg of acetaminophen will be administered intravenously or by mouth 30-60 minutes prior to the darTreg infusion.

Diphenhydramine

Intervention Type DRUG

Pre-medication for single dose darTreg infusion (Cohorts 2 - 4). 25-50mg of diphenhydramine will be administered intravenously or by mouth 30-60 minutes prior to the darTreg infusion.

Anti-Infective Prophylaxis

Intervention Type DRUG

Intravenous ganciclovir and/or oral Valcyte will be administered for the prophylaxis of cytomegalovirus (CMV) and Epstein-Barr Virus (EBV) for at least six months after liver transplantation.

Leukapheresis

Intervention Type PROCEDURE

Leukapheresis is necessary to ensure collection of adequate numbers of autologous Tregs to support ex vivo expansion of darTregs for infusion after liver transplantation.

Participants enrolled in Cohorts 3 and 4 will undergo leukapheresis. Participants enrolled in Cohort 2 will have either whole blood collection or leukapheresis for the purpose of isolating autologous Tregs for later manufacture. If a cohort 2 subject has a hemoglobin level \>/=10.5 gm/dL, he or she will undergo phlebotomy. If the patient has a hemoglobin level \</=10.5 gm/dL and remains eligible for the study, the patient will undergo leukapheresis.

Blood draws

Intervention Type PROCEDURE

Blood draws are necessary to carefully and frequently evaluate allograft function after liver transplantation and treatment with Treg-supportive IS as well as after darTreg infusion. Peripheral blood samples will be collected and analyzed per protocol throughout subject participation in this study.

Liver biopsies

Intervention Type PROCEDURE

Subjects will have a liver biopsy for this study 12-14 weeks after transplantation. For subjects receiving darTregs, a second biopsy will be performed 7-10 days after darTregs infusion.

Liver transplantation

Intervention Type PROCEDURE

Inclusion in this trial is in the setting of subjects defined as having end-stage liver disease and listed for primary solitary liver transplant.

Cohort 3 - darTreg infusion,200 million(range100-240 million)

At least 3 subjects will receive a single infusion dose of 200 million darTregs. Progression from one cohort to the next will depend on the cumulative incidence of sentinel adverse events.

Group Type EXPERIMENTAL

Anti-Thymocyte Globulin - Rabbit

Intervention Type BIOLOGICAL

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Subjects will be given a dose range of 3.0-4.5 mg/kg total, in divided doses of 1.5 mg/kg/day. Subjects who meet eligibility criteria for Thymoglobulin® administration will be given 1.5 mg/kg intravenously (IV) on post-operative day 3, within 72 hours of transplantation. Additional doses of 1.5 mg/kg IV will be administered until CD3 count is \<50/mm\^3 or when the maximal dose of 4.5/mg/kg has been given.

darTreg Infusion

Intervention Type BIOLOGICAL

A single dose darTreg infusion (Cohorts 2 - 4) will be received as per protocol.

Everolimus

Intervention Type DRUG

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Subjects meeting eligibility criteria for Treg-supportive IS regimen will begin EVR no sooner than 30 days after liver transplantation and no later than 44 days after transplantation; with target trough levels of 6-8 μg/L.EVR target trough levels will be further reduced to 4-6 μg/L 24 - 26 weeks after transplantation.

Tacrolimus

Intervention Type DRUG

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Between 30 and 44 days following transplant: Subjects who are not eliminated by Exclusion Criteria C1 (Protocol Section 14.3.1) will proceed in the study and receive either TAC-based or EVR- based IS, based on eligibility Criteria C2 (Section 4.3.4 )

* TAC-based IS: reduce TAC trough level to 3-8 μg/dL; continue MMF
* EVR-based IS: reduce TAC trough level to 3-8 μg/dL; EVR target trough level of 6-8 μg/dL; decrease then discontinue MMF

Mycophenolate mofetil

Intervention Type DRUG

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. 1000 mg total daily dose. MMF will be initiated within 24 hours of transplantation. MMF must be discontinued as soon as target EVR trough levels have been achieved.

Prednisone

Intervention Type DRUG

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Solumedrol 500 mg will be given IV on the day of transplantation. Additional Solumedrol will be prescribed according to site-specific standard of care. Oral prednisone should be initiated once oral medication is tolerated.

Acetaminophen

Intervention Type DRUG

Pre-medication for single dose darTreg infusion (Cohorts 2 - 4). 650mg of acetaminophen will be administered intravenously or by mouth 30-60 minutes prior to the darTreg infusion.

Diphenhydramine

Intervention Type DRUG

Pre-medication for single dose darTreg infusion (Cohorts 2 - 4). 25-50mg of diphenhydramine will be administered intravenously or by mouth 30-60 minutes prior to the darTreg infusion.

Anti-Infective Prophylaxis

Intervention Type DRUG

Intravenous ganciclovir and/or oral Valcyte will be administered for the prophylaxis of cytomegalovirus (CMV) and Epstein-Barr Virus (EBV) for at least six months after liver transplantation.

Leukapheresis

Intervention Type PROCEDURE

Leukapheresis is necessary to ensure collection of adequate numbers of autologous Tregs to support ex vivo expansion of darTregs for infusion after liver transplantation.

Participants enrolled in Cohorts 3 and 4 will undergo leukapheresis. Participants enrolled in Cohort 2 will have either whole blood collection or leukapheresis for the purpose of isolating autologous Tregs for later manufacture. If a cohort 2 subject has a hemoglobin level \>/=10.5 gm/dL, he or she will undergo phlebotomy. If the patient has a hemoglobin level \</=10.5 gm/dL and remains eligible for the study, the patient will undergo leukapheresis.

Blood draws

Intervention Type PROCEDURE

Blood draws are necessary to carefully and frequently evaluate allograft function after liver transplantation and treatment with Treg-supportive IS as well as after darTreg infusion. Peripheral blood samples will be collected and analyzed per protocol throughout subject participation in this study.

Liver biopsies

Intervention Type PROCEDURE

Subjects will have a liver biopsy for this study 12-14 weeks after transplantation. For subjects receiving darTregs, a second biopsy will be performed 7-10 days after darTregs infusion.

Liver transplantation

Intervention Type PROCEDURE

Inclusion in this trial is in the setting of subjects defined as having end-stage liver disease and listed for primary solitary liver transplant.

Cohort 4 - darTreg infusion,800 million(range 400-960 million)

Six subjects will receive a single infusion of 800 million darTregs.

Group Type EXPERIMENTAL

Anti-Thymocyte Globulin - Rabbit

Intervention Type BIOLOGICAL

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Subjects will be given a dose range of 3.0-4.5 mg/kg total, in divided doses of 1.5 mg/kg/day. Subjects who meet eligibility criteria for Thymoglobulin® administration will be given 1.5 mg/kg intravenously (IV) on post-operative day 3, within 72 hours of transplantation. Additional doses of 1.5 mg/kg IV will be administered until CD3 count is \<50/mm\^3 or when the maximal dose of 4.5/mg/kg has been given.

darTreg Infusion

Intervention Type BIOLOGICAL

A single dose darTreg infusion (Cohorts 2 - 4) will be received as per protocol.

Everolimus

Intervention Type DRUG

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Subjects meeting eligibility criteria for Treg-supportive IS regimen will begin EVR no sooner than 30 days after liver transplantation and no later than 44 days after transplantation; with target trough levels of 6-8 μg/L.EVR target trough levels will be further reduced to 4-6 μg/L 24 - 26 weeks after transplantation.

Tacrolimus

Intervention Type DRUG

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Between 30 and 44 days following transplant: Subjects who are not eliminated by Exclusion Criteria C1 (Protocol Section 14.3.1) will proceed in the study and receive either TAC-based or EVR- based IS, based on eligibility Criteria C2 (Section 4.3.4 )

* TAC-based IS: reduce TAC trough level to 3-8 μg/dL; continue MMF
* EVR-based IS: reduce TAC trough level to 3-8 μg/dL; EVR target trough level of 6-8 μg/dL; decrease then discontinue MMF

Mycophenolate mofetil

Intervention Type DRUG

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. 1000 mg total daily dose. MMF will be initiated within 24 hours of transplantation. MMF must be discontinued as soon as target EVR trough levels have been achieved.

Prednisone

Intervention Type DRUG

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Solumedrol 500 mg will be given IV on the day of transplantation. Additional Solumedrol will be prescribed according to site-specific standard of care. Oral prednisone should be initiated once oral medication is tolerated.

Acetaminophen

Intervention Type DRUG

Pre-medication for single dose darTreg infusion (Cohorts 2 - 4). 650mg of acetaminophen will be administered intravenously or by mouth 30-60 minutes prior to the darTreg infusion.

Diphenhydramine

Intervention Type DRUG

Pre-medication for single dose darTreg infusion (Cohorts 2 - 4). 25-50mg of diphenhydramine will be administered intravenously or by mouth 30-60 minutes prior to the darTreg infusion.

Anti-Infective Prophylaxis

Intervention Type DRUG

Intravenous ganciclovir and/or oral Valcyte will be administered for the prophylaxis of cytomegalovirus (CMV) and Epstein-Barr Virus (EBV) for at least six months after liver transplantation.

Leukapheresis

Intervention Type PROCEDURE

Leukapheresis is necessary to ensure collection of adequate numbers of autologous Tregs to support ex vivo expansion of darTregs for infusion after liver transplantation.

Participants enrolled in Cohorts 3 and 4 will undergo leukapheresis. Participants enrolled in Cohort 2 will have either whole blood collection or leukapheresis for the purpose of isolating autologous Tregs for later manufacture. If a cohort 2 subject has a hemoglobin level \>/=10.5 gm/dL, he or she will undergo phlebotomy. If the patient has a hemoglobin level \</=10.5 gm/dL and remains eligible for the study, the patient will undergo leukapheresis.

Blood draws

Intervention Type PROCEDURE

Blood draws are necessary to carefully and frequently evaluate allograft function after liver transplantation and treatment with Treg-supportive IS as well as after darTreg infusion. Peripheral blood samples will be collected and analyzed per protocol throughout subject participation in this study.

Liver biopsies

Intervention Type PROCEDURE

Subjects will have a liver biopsy for this study 12-14 weeks after transplantation. For subjects receiving darTregs, a second biopsy will be performed 7-10 days after darTregs infusion.

Liver transplantation

Intervention Type PROCEDURE

Inclusion in this trial is in the setting of subjects defined as having end-stage liver disease and listed for primary solitary liver transplant.

Interventions

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Anti-Thymocyte Globulin - Rabbit

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Subjects will be given a dose range of 3.0-4.5 mg/kg total, in divided doses of 1.5 mg/kg/day. Subjects who meet eligibility criteria for Thymoglobulin® administration will be given 1.5 mg/kg intravenously (IV) on post-operative day 3, within 72 hours of transplantation. Additional doses of 1.5 mg/kg IV will be administered until CD3 count is \<50/mm\^3 or when the maximal dose of 4.5/mg/kg has been given.

Intervention Type BIOLOGICAL

darTreg Infusion

A single dose darTreg infusion (Cohorts 2 - 4) will be received as per protocol.

Intervention Type BIOLOGICAL

Everolimus

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Subjects meeting eligibility criteria for Treg-supportive IS regimen will begin EVR no sooner than 30 days after liver transplantation and no later than 44 days after transplantation; with target trough levels of 6-8 μg/L.EVR target trough levels will be further reduced to 4-6 μg/L 24 - 26 weeks after transplantation.

Intervention Type DRUG

Tacrolimus

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Between 30 and 44 days following transplant: Subjects who are not eliminated by Exclusion Criteria C1 (Protocol Section 14.3.1) will proceed in the study and receive either TAC-based or EVR- based IS, based on eligibility Criteria C2 (Section 4.3.4 )

* TAC-based IS: reduce TAC trough level to 3-8 μg/dL; continue MMF
* EVR-based IS: reduce TAC trough level to 3-8 μg/dL; EVR target trough level of 6-8 μg/dL; decrease then discontinue MMF

Intervention Type DRUG

Mycophenolate mofetil

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. 1000 mg total daily dose. MMF will be initiated within 24 hours of transplantation. MMF must be discontinued as soon as target EVR trough levels have been achieved.

Intervention Type DRUG

Prednisone

Liver transplantation/Treg-supportive immunosuppression (IS) treatment. Solumedrol 500 mg will be given IV on the day of transplantation. Additional Solumedrol will be prescribed according to site-specific standard of care. Oral prednisone should be initiated once oral medication is tolerated.

Intervention Type DRUG

Acetaminophen

Pre-medication for single dose darTreg infusion (Cohorts 2 - 4). 650mg of acetaminophen will be administered intravenously or by mouth 30-60 minutes prior to the darTreg infusion.

Intervention Type DRUG

Diphenhydramine

Pre-medication for single dose darTreg infusion (Cohorts 2 - 4). 25-50mg of diphenhydramine will be administered intravenously or by mouth 30-60 minutes prior to the darTreg infusion.

Intervention Type DRUG

Anti-Infective Prophylaxis

Intravenous ganciclovir and/or oral Valcyte will be administered for the prophylaxis of cytomegalovirus (CMV) and Epstein-Barr Virus (EBV) for at least six months after liver transplantation.

Intervention Type DRUG

Leukapheresis

Leukapheresis is necessary to ensure collection of adequate numbers of autologous Tregs to support ex vivo expansion of darTregs for infusion after liver transplantation.

Participants enrolled in Cohorts 3 and 4 will undergo leukapheresis. Participants enrolled in Cohort 2 will have either whole blood collection or leukapheresis for the purpose of isolating autologous Tregs for later manufacture. If a cohort 2 subject has a hemoglobin level \>/=10.5 gm/dL, he or she will undergo phlebotomy. If the patient has a hemoglobin level \</=10.5 gm/dL and remains eligible for the study, the patient will undergo leukapheresis.

Intervention Type PROCEDURE

Blood draws

Blood draws are necessary to carefully and frequently evaluate allograft function after liver transplantation and treatment with Treg-supportive IS as well as after darTreg infusion. Peripheral blood samples will be collected and analyzed per protocol throughout subject participation in this study.

Intervention Type PROCEDURE

Liver biopsies

Subjects will have a liver biopsy for this study 12-14 weeks after transplantation. For subjects receiving darTregs, a second biopsy will be performed 7-10 days after darTregs infusion.

Intervention Type PROCEDURE

Liver transplantation

Inclusion in this trial is in the setting of subjects defined as having end-stage liver disease and listed for primary solitary liver transplant.

Intervention Type PROCEDURE

Other Intervention Names

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ATG (Anti-Thymocyte Globulin) Thymoglobulin® Donor-Alloantigen-Reactive Regulatory T Cell Infusion EVR Zortress® Prograf® MMF Cellcept Myfortic Solumedrol Tylenol® Diphenhydramine Hydrochloride Antiviral Prophylaxis ganciclovir Cytovene Valganciclovir Valcyte Leukocytapheresis Venipuncture Phlebotomy Liver biopsy samples Liver transplant

Eligibility Criteria

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Inclusion Criteria

* Subjects who meet all of the following criteria are eligible for enrollment as study participants:

* Able to understand and provide informed consent
* End-stage liver disease and listed for primary solitary liver transplant
* Have a calculated Model for End Stage Liver Disease (MELD) score ≤ 25 at the time of study entry/consent
* Female and male subjects with reproductive potential must agree to use effective methods of birth control for the duration of the study.
* If history of Hepatitis C Virus (HCV), have completed or are in current treatment for HCV AND have no detectable HCV RNA.
* Subjects with HCC meeting Milan criteria.

Exclusion Criteria

* End stage liver disease secondary to autoimmune etiology (autoimmune hepatitis, primary biliary cirrhosis, or primary sclerosing cholangitis)
* History of less than 5 years remission of malignancy, except for 1) HCC or 2) history of adequately treated in-situ cervical carcinoma, or adequately treated basal or squamous cell carcinoma of the skin.
* History of previous organ, tissue or cell transplant
* Serologic evidence of human immunodeficiency (HIV) 1 or -2 infection
* Epstein-Barr Virus (EBV) or cytomegalovirus (CMV) sero-negativity (EBV or CMV naïve candidates)
* Chronic use of systemic glucocorticoids or other Immunosuppression (IS), or biologic immunomodulators
* Chronic condition requiring anti-coagulation after liver transplantation
* Any chronic illness or prior treatment which, in the opinion of the investigator, precludes study participation
* Participation in any other studies that involved investigational drugs or regimens in the preceding year
* Received any vaccination within 28 days prior to leukapheresis or blood collection for Treg manufacture
* Hemoglobin \<9.0 g/dL within 10 days prior to screening
* Neutrophils \<1,500/μL within 10 days prior to screening
* Platelets \<40,000/μL within 10 days prior to screening


* Calculated Model for End Stage Liver Disease (MELD) score \>25 at the time of deceased donor liver transplant
* Last alpha-fetoprotein (AFP) obtained prior to liver transplantation \>400 μg/L for candidates with Hepatocellular Carcinoma (HCC)
* Unacceptable Peripheral Blood Mononuclear Cells (PBMC) product for participants enrolled in Cohorts 2, 3, or 4 per the UCSF The Human Islet and Cellular Transplant Facility (HICTF) manufacturing specifications
* Absence of donor spleen for any participants
* Human leukocyte antigen (HLA)-DR (DR is one of class II antigens) matched to donor at both loci
* Subject is \< 21 or \>70 years of age at the time of transplantation
* Located in the intensive care unit 72 hours after transplantation
* Hemoglobin \<8.0 g/dL
* Absolute neutrophil count \<1,200/μL
* Platelets \<40,000/μL
* Positive pregnancy test for females of child bearing potential
* Unexpected histopathology on back table liver biopsy that contraindicates the initiation of Treg supportive IS regimen.
* Development of a condition requiring chronic anti-coagulation.
* Hypersensitivity to rabbit proteins or any excipient in Thymoglobulin®.
* Detectable HCV RNA or less than six months after end of treatment for HCV at the time of transplantation (i.e., does not meet criteria for SVR).

* Explanted liver with evidence of increased risk of recurrent cancer risk (hepatocellular (HCC) tumor burden exceeding the Milan criteria; presence of vascular invasion; cholangiocarcinoma morphology)
* Insufficient depletion of recipient T cells, defined as a nadir CD3 count ≥50 cells μ/L (50 cells /mcL) or total lymphocyte count ≥ 0.1x 109/L if CD3 count is unavailable
* Development of a condition requiring chronic anti-coagulation.
* Clinical evidence of biliary obstruction
* Alanine Aminotransferase (ALT) \>2.0 x upper limit of normal (ULN)
* Inability to taper off corticosteroids by 44 days (+/- 2 days) after transplant
* Detectable circulating HCV RNA.

Everolimus Conversion Criteria C2 (assessed prior to conversion to EVR based IS regimen; EVR cannot be initiated prior to 30 days after liver transplantation). Subjects with any of the following will remain on TAC-based IS regimen.

* Evidence of hepatic artery stenosis or thrombosis by Doppler examination or angiography within 7 days prior to conversion
* Urine protein/creatinine ratio \>1.0 within 7 days prior to conversion
* Calculated GFR less than 30 ml/min per MDRD4 (Modification of Diet in Renal Disease Study) equation within 7 days prior to conversion
* Physical examination documentation of abnormal wound healing or uncontrolled wound infection
* Hemoglobin \<8.0 g/dL within 7 days prior to conversion
* Absolute neutrophil count \<1,200/μL within 7 days prior to conversion
* Platelets \<50,000/μL within 7 days prior to conversion

* Inability or unwillingness of participant to give additional written informed consent
* Unacceptable darTreg product
* Detectible circulating Epstein-Barr Virus (EBV) or cytomegalovirus (CMV) DNA within 10 days prior to darTreg infusion
* Detectible Hepatitis B Virus (HBV) DNA within 10 days prior to darTreg infusion
* Detectable circulating HCV RNA within 10 days prior to darTreg infusion.
* Alanine Aminotransferase (ALT) \>1.5x upper limit of normal within 10 days of darTreg infusion
* Most recent, but not greater than 10 days prior to darTreg infusion,12 hour TAC trough levels of \> 8 μg/L for all subjects
* Most recent, but not greater than 10 days prior to darTreg infusion,12 hour EVR trough levels of \< 5 μg/L for subjects on EVR
* For subjects on EVR-based IS, received Mycophenolate Mofetil (MMF) within 10 days prior to darTreg infusion
* Evidence of acute rejection or chronic rejection according to Banff criteria on protocol allograft biopsy based on local assessment
* Received any vaccination within 14 days prior to darTreg infusion
* Positive pregnancy test for females of child bearing potential
* Inability or unwillingness of participant to comply with study protocol or procedures.
* Calculated glomerular filtration rate (eGFR) less than 40 ml/min per MDRD4 equation within 10 days prior to infusion.
Minimum Eligible Age

21 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Rho Federal Systems Division, Inc.

INDUSTRY

Sponsor Role collaborator

National Institute of Allergy and Infectious Diseases (NIAID)

NIH

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Sandy Feng, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

University of California, San Francisco

Jeffrey Bluestone, PhD

Role: PRINCIPAL_INVESTIGATOR

University of California, San Francisco

Sang-Mo Kang, MD, FACS

Role: PRINCIPAL_INVESTIGATOR

University of California, San Francisco

Qizhi Tang, PhD

Role: PRINCIPAL_INVESTIGATOR

University of California, San Francisco

Locations

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University of California, San Francisco

San Francisco, California, United States

Site Status

Northwestern University

Chicago, Illinois, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

Countries

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United States

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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https://www.niaid.nih.gov/

National Institute of Allergy and Infectious Diseases (NIAID) website

Other Identifiers

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R34AI095135

Identifier Type: NIH

Identifier Source: secondary_id

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U01AI110658

Identifier Type: NIH

Identifier Source: secondary_id

View Link

NIAID CRMS ID#: 20182

Identifier Type: OTHER

Identifier Source: secondary_id

DAIT RTB-002

Identifier Type: -

Identifier Source: org_study_id

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