Using Siplizumab to prevent immune issues in liver transplant patients with autoimmune liver disease
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)
This study is testing if a new medication called siplizumab can help prevent immune problems in adults with autoimmune liver disease who are getting a liver transplant.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 8 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Columbia University Academic / other |
| Drugs / interventions | Siplizumab |
| Locations | 1 site (New York, New York) |
| Trial ID | NCT06455280 on ClinicalTrials.gov |
What this trial studies
This pilot, open-label phase 1 study evaluates the safety of siplizumab, an anti-CD2 monoclonal antibody, as induction immunosuppression in patients with autoimmune liver disease undergoing liver transplantation. The study will involve up to eight adult participants who will receive two doses of siplizumab: one on the day of transplant and another on Day 4 post-transplant. Participants will be monitored for 12 months following the transplant to assess the safety and effectiveness of the treatment in preventing immune-mediated graft injury. This approach aims to address the unmet need for effective therapies in this patient population.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older with a clinical diagnosis of autoimmune hepatitis or primary sclerosing cholangitis who are listed for liver transplantation.
Not a fit: Patients with significant liver disease other than autoimmune hepatitis or primary sclerosing cholangitis, or those with a history of malignancy, will not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve outcomes for liver transplant patients with autoimmune liver disease by reducing the risk of graft rejection.
How similar studies have performed: While this approach is novel for this specific patient population, similar immunosuppressive strategies have shown promise in other transplant settings.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion 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 Exclusion Criteria: 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. ADDITIONAL exclusion criteria to be reviewed at the time 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
Where this trial is running
New York, New York
- Columbia University Irving Medical Center/NewYork-Presbyterian Hospital — New York, New York, United States (Recruiting)
Study contacts
- Principal investigator: Elizabeth Verna, MD — Columbia University Irving Medical Center/ New York Presbyterian Hospital
- Study coordinator: Theresa Lukose, PharmD
- Email: tt2103@cumc.columbia.edu
- Phone: 212-305-3839
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.