Autoimmune Liver DiseasePhase 1SET-SAILNCT06455280
A Study of SIPLIZUMAB in AILD and LT Patients
A Phase 1 study testing Siplizumab in people with Autoimmune Liver Disease and Liver Transplant Disorder, run by Elizabeth C. Verna at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autoimmune Liver Disease, Liver Transplant Disorder, Autoimmune Hepatitis
- What is being tested?
- Siplizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Sep 2024to 31 Mar 2028
- Phase
- Phase 1
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Able to provide informed consent
- Age ≥ 18 years old
- Clinical diagnosis of AIH and/or PSC
- Listed for liver transplantation
- Epstein-Barr virus (EBV) seropositive within 12 months of screening
Who cannot join
- 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
- Prior transplant
- Listed for multiorgan transplant
- Acute liver failure
- Known malignancy, including cholangiocarcinoma and hepatocellular carcinoma
- Other investigational products in the last 30 days or 5 half lives
Show all 19Show fewer
- Pregnant/lactating or unwilling to use contraception
- Leukopenia (WBC less than 2,000/mm3
- Absolute lymphocyte count < 200/mm3
- Sero-positive for HIV-1
- Hepatitis C Virus (HCV) antibody or RNA positive (within 6 months of screening)
- HBsAg, hepatitis B virus (HBV) DNA or HBcAb positive (within 6 months of screening)
- 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
- Untreated latent TB infection as detected by QuantiFERON Gold Plus Interferon Gamma Release Assay (IGRA) (or current standard interferon gamma release assay for TB)
- Receipt of any live-attenuated vaccine within 2 months of transplant.
- ADDITIONAL exclusion criteria to be reviewed at the time of transplant
- Renal failure with dialysis or with estimated glomerular filtration rate (eGFR) < 30 at the time of LT
- Model for end-stage liver disease (MELD)-Na score >30
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Columbia University Irving Medical Center/NewYork-Presbyterian Hospital
New York, New York, 10032
Theresa Lukose, PharmD, 212-305-3839, tt2103@cumc.columbia.edu
Elizabeth Verna, MD
About this study
Official title: 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)
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.
Read the detailed description
The purpose of this study is to evaluate the safety of siplizumab when used as induction immunosuppression in patients with primary sclerosing cholangitis (PSC) or autoimmune hepatitis (AIH) undergoing liver transplantation. Induction immunosuppression drugs are very potent anti-rejection drugs that are given immediately after transplantation to prevent rejection. Siplizumab is investigational, meaning it has not yet been approved for market use for this disease condition by the United States Food and Drug Administration (FDA).
Adult patients (18 years of age and older) listed for LT with the specific AILD diagnoses of PSC or AIH
All subjects will receive 0.6 mg/kg/dose intravenously on the day of transplant (Day 0) intraoperatively and on post-transplant Day 4.
Participation in this study will last approximately 15 months (~ 3 months on the LT waitlist, up to 12 months participation post-LT)
- Conditions
- Autoimmune Liver Disease, Liver Transplant Disorder, Autoimmune Hepatitis, Primary Sclerosing Cholangitis, End Stage Liver DIsease, Cirrhosis, Liver
- Being tested
- Siplizumab
- Keywords
- Autoimmune Liver Disease, Liver Transplant Disorder, Autoimmune Hepatitis, Primary Sclerosing Cholangitis, End Stage Liver DIsease, Cirrhosis, Liver
- Medical subject headings
- Hepatitis, Autoimmune, Cholangitis, Sclerosing, End Stage Liver Disease, Liver Cirrhosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SiplizumabDrug
Siplizumab is an anti-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.
| Study group | Role | What they receive |
|---|---|---|
| Open Label | Experimental | subjects will receive 0.6 mg/kg/dose intravenously on the day of transplant (Day 0) intraoperatively and on post-transplant Day 4. Receives: Drug: Siplizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Serious infection in the first month post-transplant,
viral, bacterial or fungal infection that leads to readmission, prolonged hospitalization, reoperation, intensive care unit admission, graft loss or death.
Measured: 1 Month post-transplant
Secondary outcomes (7)
Incidence of immune-mediated liver injury
biopsy proven acute rejection (BPAR), or recurrent AILD
Measured: 12 month Post-transplant
Incidence of graft loss or death
Loss of liver allograft or incidence of mortality
Measured: 12 month Post-transplant
Incidence of BPAR
biopsy proven acute rejection within 12 Month post-transplant
Measured: 12 month Post-transplant
Incidence of treated BPAR
biopsy proven acute rejection that requires treatment within 12 Month post-transplant
Measured: 12 month Post-transplant
Incidence of refractory BPAR
biopsy proven acute rejection within 12 Month post-transplant that is not responsive to treatment
Measured: 12 month Post-transplant
Incidence of development of donor specific antibodies (DSA)
Donor specific antibodies within 12 Month Post-transplant
Measured: 12 month Post-transplant
Incidence of recurrent AILD
based upon histology for autoimmune hepatitis [AIH] and histology and/or imaging for primary sclerosing cholangitis [PSC]
Measured: 12 month Post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2024
- Study start11 Sep 2024
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autoimmune 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open-Label Study
- Masking
- None (open label)
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Elizabeth C. Verna
- Sponsor type
- University, hospital or other
- Collaborators
- ITB-Med LLC
- Responsible party
- Sponsor-investigator, Elizabeth C. Verna, Columbia University
- Investigators
- Elizabeth Verna, MD, Principal investigator, Columbia University Irving Medical Center/ New York Presbyterian Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06455280
- Sponsor's protocol ID
- AAAU7303
- Acronym
- SET-SAIL
- Status last verified
- Nov 2025
- First submitted
- 7 Jun 2024
- Last updated
- 24 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No