A Study of SIPLIZUMAB in AILD and LT Patients

Description

There is a significant unmet need for safe and effective therapeutic approaches to prevent immune-mediated graft injury and its complications in liver transplant (LT) recipients with autoimmune liver disease (AILD) including autoimmune hepatitis and primary sclerosing cholangitis. Siplizumab is an anti-cluster of differentiation 2 (CD2) monoclonal antibody that has demonstrated a favorable safety profile of siplizumab in over 779 human subjects and has been shown to target memory T cells-a key driver in the immune processes surrounding rejection and autoimmunity post LT in AILD. The purpose of this pilot, open-label phase 1 study is to determine the safety of siplizumab for induction in patients with AILD undergoing LT. Up to eight (8) subjects will receive siplizumab 0.6 mg/kg/dose on the day of transplant (Day 0) and Day 4 post-transplant, for a total of two doses. All subjects will be followed in the study for 12 months post-LT.

Conditions

Autoimmune Liver Disease, Liver Transplant Disorder, Autoimmune Hepatitis, Primary Sclerosing Cholangitis, End Stage Liver DIsease, Cirrhosis, Liver

Talk to us

Study Overview

On this page

Study Details

Study overview

There is a significant unmet need for safe and effective therapeutic approaches to prevent immune-mediated graft injury and its complications in liver transplant (LT) recipients with autoimmune liver disease (AILD) including autoimmune hepatitis and primary sclerosing cholangitis. Siplizumab is an anti-cluster of differentiation 2 (CD2) monoclonal antibody that has demonstrated a favorable safety profile of siplizumab in over 779 human subjects and has been shown to target memory T cells-a key driver in the immune processes surrounding rejection and autoimmunity post LT in AILD. The purpose of this pilot, open-label phase 1 study is to determine the safety of siplizumab for induction in patients with AILD undergoing LT. Up to eight (8) subjects will receive siplizumab 0.6 mg/kg/dose on the day of transplant (Day 0) and Day 4 post-transplant, for a total of two doses. All subjects will be followed in the study for 12 months post-LT.

A 12-Month, Open-Label Study Evaluating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Siplizumab As Induction Therapy in Patients with Autoimmune Liver Diseases Undergoing Liver Transplantation (SET-SAIL)

A Study of SIPLIZUMAB in AILD and LT Patients

Condition
Autoimmune Liver Disease
Intervention / Treatment

-

Contacts and Locations

New York

Columbia University Irving Medical Center/NewYork-Presbyterian Hospital, New York, New York, United States, 10032

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 1. Able to provide informed consent
  • 2. Age ≥ 18 years old
  • 3. Clinical diagnosis of AIH and/or PSC
  • 4. Listed for liver transplantation
  • 5. Epstein-Barr virus (EBV) seropositive within 12 months of screening
  • 1. Presence or history of significant liver disease other than AIH or PSC, including viral hepatitis, alcohol-related liver disease and biopsy-proven non-alcoholic steatohepatitis
  • 2. Prior transplant
  • 3. Listed for multiorgan transplant
  • 4. Acute liver failure
  • 5. Known malignancy, including cholangiocarcinoma and hepatocellular carcinoma
  • 6. Other investigational products in the last 30 days or 5 half lives
  • 7. Pregnant/lactating or unwilling to use contraception
  • 8. Leukopenia (WBC less than 2,000/mm3
  • 9. Absolute lymphocyte count \< 200/mm3
  • 10. Sero-positive for HIV-1
  • 11. Hepatitis C Virus (HCV) antibody or RNA positive (within 6 months of screening)
  • 12. HBsAg, hepatitis B virus (HBV) DNA or HBcAb positive (within 6 months of screening)
  • 13. Alcohol use exceeding 30g/day for men or 20g/day for women, and/or known phosphatidylethanol (PETH) level \>80 in the 3 months prior to LT
  • 14. Untreated latent TB infection as detected by QuantiFERON Gold Plus Interferon Gamma Release Assay (IGRA) (or current standard interferon gamma release assay for TB)
  • 15. Receipt of any live-attenuated vaccine within 2 months of transplant.
  • 1. Renal failure with dialysis or with estimated glomerular filtration rate (eGFR) \< 30 at the time of LT
  • 2. Model for end-stage liver disease (MELD)-Na score \>30
  • 3. Donor features of Donation after Cardiac Death (DCD), HCV Ab or nucleic acid testing (NAT+), HBcAb or HBsAg+, or blood types A, B, and O incompatible organ

Ages Eligible for Study

18 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Elizabeth C. Verna,

Elizabeth Verna, MD, PRINCIPAL_INVESTIGATOR, Columbia University Irving Medical Center/ New York Presbyterian Hospital

Study Record Dates

2026-03-31