MedPath

Immune System Suppression With Alemtuzumab and Tacrolimus in Liver Transplantation Patients

Phase 2
Completed
Conditions
Liver Disease
Liver Transplantation
Interventions
Procedure: Liver transplant
Procedure: Immunosuppression withdrawal
Registration Number
NCT00105235
Lead Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Brief Summary

Alemtuzumab is a man-made antibody used to treat certain blood disorders. Tacrolimus is a drug used to decrease immune system activity in people who have received organ transplants so that the new organ will not be rejected. This study will determine whether treatment with alemtuzumab and tacrolimus is effective in preventing organ rejection and maintaining the recipient's health after liver transplantation in patients with end-stage liver disease, and whether gradual tapering of tacrolimus treatment is safe for these patients.

Detailed Description

Organ transplantation is a common procedure in hospitals, but organ rejection and serious side effects are potential problems for the patient. Alemtuzumab is a monoclonal antibody that binds to and depletes excess T cells in the bone marrow of leukemia patients. In this study, alemtuzumab will destroy the recipient's white blood cells (WBCs) at the time of transplantation. It is hoped that WBCs produced after alemtuzumab administration will recognize the transplanted liver as "self" and not reject the new liver.

Drugs that suppress the immune system, such as tacrolimus, have contributed to increased success of transplantation. However, to prevent organ rejection, transplant recipients need to take immunosuppressive drugs for the rest of their lives, and these drugs make patients more susceptible to infection, endangering their health and survival. Regimens that are less toxic to or can eventually be withdrawn from transplant recipients are needed. This study will evaluate the effects of two in-patient doses of alemtuzumab followed by maintenance antirejection medication given to liver transplant patients post-transplant. This study will also determine if post-transplant tacrolimus therapy can be slowly and safely tapered off and withdrawn a year after transplant. Participants in this study will be patients with end-stage liver disease who will undergo liver transplantation at the start of the study.

This study will last at least 2 years. Patients will undergo liver transplantation at the start of the study on Day 0. Patients will receive in-patient infusions of alemtuzumab on Days 0 and 4. Starting on Day 1, patients will receive oral cyclosporine, mycophenolate mofetil, and/or tacrolimus daily. Patients will be hospitalized for at least 1 week after transplantation. Because of suppression of patients' immune systems by alemtuzumab and these other immunosuppressants, they will also receive prophylactic medications for a minimum of 3 months after transplantation to prevent opportunistic infections.

There will be at least eight study visits; they will occur at Days 4, 7, and 14 and at Months 1, 3, 6, 9, and 12. Patients will have liver biopsies at Day 0 and Months 6 and 12. At Month 12, participants will have assessments and blood tests to determine if they meet certain criteria and are eligible to undergo tacrolimus tapering. Patients eligible for tapering will undergo a 12-month gradual withdrawal of tacrolimus; they will be followed for an additional 2 years, with study visits at Months 18, 24, 30, and 36. Patients ineligible for tacrolimus tapering will continue taking their antirejection medication for the duration of the study; they will be followed for an additional year, with study visits at Months 18 and 24.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
27
Inclusion Criteria
  • Diagnosis of nonimmune, nonviral, end-stage liver disease
  • Need liver transplant
  • Willing to use acceptable means of contraception for the duration of the study
Exclusion Criteria
  • Previous transplant
  • Multiorgan transplant or living donor transplant
  • Donor liver from a donor positive for antibody against hepatitis B core antigen or hepatitis C virus
  • Donor liver from a non-heart-beating donor
  • Liver failure due to autoimmune disease, such as autoimmune hepatitis, primary sclerosing cholangitis, or primary biliary cirrhosis
  • Hepatitis B or C virus infection
  • HIV infection
  • Stage III or higher hepatocellular cancer based on pre-transplant imaging
  • History of cancer. Patients with hepatocellular cancer, adequately treated in situ cervical carcinoma, or adequately treated basal or squamous cell carcinoma of skin are not excluded.
  • Active systemic infection at the time of transplantation
  • Clinically significant chronic renal, cardiovascular, or cerebrovascular disease
  • Any investigational drug within 6 weeks of study entry
  • Hypersensitivity to alemtuzumab or tacrolimus

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
AlemtuzumabLiver transplantLiver transplant, with two in-patient infused doses of alemtuzumab; followed by maintenance immunotherapy with cyclosporine, mycophenolate mofetil, and/or tacrolimus; with possible immunosuppression withdrawal
AlemtuzumabImmunosuppression withdrawalLiver transplant, with two in-patient infused doses of alemtuzumab; followed by maintenance immunotherapy with cyclosporine, mycophenolate mofetil, and/or tacrolimus; with possible immunosuppression withdrawal
AlemtuzumabAlemtuzumabLiver transplant, with two in-patient infused doses of alemtuzumab; followed by maintenance immunotherapy with cyclosporine, mycophenolate mofetil, and/or tacrolimus; with possible immunosuppression withdrawal
AlemtuzumabCyclosporineLiver transplant, with two in-patient infused doses of alemtuzumab; followed by maintenance immunotherapy with cyclosporine, mycophenolate mofetil, and/or tacrolimus; with possible immunosuppression withdrawal
AlemtuzumabTacrolimusLiver transplant, with two in-patient infused doses of alemtuzumab; followed by maintenance immunotherapy with cyclosporine, mycophenolate mofetil, and/or tacrolimus; with possible immunosuppression withdrawal
AlemtuzumabMycophenolate mofetilLiver transplant, with two in-patient infused doses of alemtuzumab; followed by maintenance immunotherapy with cyclosporine, mycophenolate mofetil, and/or tacrolimus; with possible immunosuppression withdrawal
Primary Outcome Measures
NameTimeMethod
Proportion of Participants Who Have Graft Loss or DeathWithin 1 year of post-transplantation

Proportion of participants who had liver graft loss or who died within 1 year of undergoing transplantation. Note: Participants who discontinued treatment or terminated the study prior to 1 year post transplantation are considered treatment failures and are included in this measure.

Secondary Outcome Measures
NameTimeMethod
Proportion of Participants Successfully Withdrawn and Remain Off ImmunosuppressantsFrom 1 year post- transplantation until study completion or participant termination (participants followed up to 48 months post-transplant)

This measure of tolerance induction includes the proportion of participants who qualify for immunosuppression withdrawal as determined by a review of individual clinical results by a protocol withdrawal committee, were successfully withdrawn from immunosuppressants, and remained off immunosuppressants at the time the trial ended. Successful withdrawal definition: participants who remain off immunosuppression for at least 8 weeks and do not restart immunosuppressant drugs after successful withdrawal.

Proportion of Participants Who Had Graft Loss or DeathWithin 2 years after initiation of immunosuppression withdrawal

Proportion of participants who had liver graft loss or who died or terminated from the study within 2 years of initiating immunosuppression withdrawal

Number of Events: Immunosuppression-related ComplicationsFrom transplantation until study completion or participant termination (participants followed up to 60 months)

Certain events are associated with immunosuppression. This measure looks at post-transplant infection, post-transplant malignancies, post-transplant diabetes, and post-transplant renal failure. Immunosuppression withdrawal is intended to reduce these type of events. However, reduction in immunosuppression can lead to complications in liver and renal function, as measured by acute rejection, chronic rejection, and post-transplant renal failure. Lower numbers for any of these events indicates greater success with transplantation and immunosuppression withdrawal (where applicable)

Proportion of Participants Successfully Withdrawn From ImmunosuppressantsFrom 1 year post- transplantation until study completion or participant termination (participants followed up to 48 months post-transplant)

This measure of tolerance induction includes the proportion of participants who qualify for immunosuppression withdrawal as determined by a review of individual clinical results by a protocol withdrawal committee. Successful withdrawal definition: participants who remain off immunosuppression for at least 8 weeks.

Trial Locations

Locations (9)

University of Miami School of Medicine

🇺🇸

Miami, Florida, United States

University of California, San Francisco

🇺🇸

San Francisco, California, United States

University of Michigan

🇺🇸

Ann Arbor, Michigan, United States

Cleveland Clinic

🇺🇸

Cleveland, Ohio, United States

University of Colorado

🇺🇸

Denver, Colorado, United States

University of Pennsylvania

🇺🇸

Philadelphia, Pennsylvania, United States

University of Wisconsin

🇺🇸

Madison, Wisconsin, United States

Baylor University

🇺🇸

Dallas, Texas, United States

University of Alberta

🇨🇦

Edmonton, Alberta, Canada

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