Siplizumab for Autoimmune Liver Disease

(SET-SAIL Trial)

Recruiting in Palo Alto (17 mi)

Overseen by Elizabeth Verna, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Recruiting

Sponsor: Elizabeth C. Verna

Disqualifiers: Viral hepatitis, Malignancy, HIV, others

No Placebo Group

Trial Summary

What is the purpose of this trial?

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.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

How does the drug Siplizumab differ from other treatments for autoimmune liver disease?

Siplizumab is unique because it targets specific immune cells, potentially offering a more targeted approach compared to traditional immunosuppressive therapies, which broadly suppress the immune system. ^{1 2 3 4 5}

Research Team

Elizabeth Verna, MD

Principal Investigator

Columbia University Irving Medical Center/ New York Presbyterian Hospital

Eligibility Criteria

This trial is for liver transplant recipients with autoimmune liver diseases like autoimmune hepatitis and primary sclerosing cholangitis. Participants should not have other conditions that could interfere with the study or pose a risk.

Inclusion Criteria

I have been diagnosed with autoimmune hepatitis or primary sclerosing cholangitis.

I am on the waiting list for a liver transplant.

Able to provide informed consent

Exclusion Criteria

I am on the waiting list for a multiorgan transplant.

Pregnant/lactating or unwilling to use contraception

Leukopenia (WBC less than 2,000/mm3)

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

3 months

Treatment

Participants receive siplizumab 0.6 mg/kg/dose intravenously on the day of transplant (Day 0) and on post-transplant Day 4

1 week

2 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including measurement of CD2 receptor occupancy and incidence of immune-mediated liver injury

12 months

Regular visits (in-person and virtual)

Open-label extension (optional)

Participants may opt into continuation of treatment long-term

Long-term

Treatment Details

Interventions

• Siplizumab (Monoclonal Antibodies)

Trial Overview The trial tests Siplizumab, an anti-CD2 monoclonal antibody, to see if it's safe and can prevent immune-mediated graft injury post-transplant in patients with AILD. It involves two doses of the drug given around the time of transplant.

Participant Groups

Group I: Open Label Experimental Treatment 1 Intervention

subjects will receive 0.6 mg/kg/dose intravenously on the day of transplant (Day 0) intraoperatively and on post-transplant Day 4.

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Columbia University Irving Medical Center/NewYork-Presbyterian Hospital New York, NY

Who Is Running the Clinical Trial?

Elizabeth C. Verna

Lead Sponsor

Trials

1

Patients Recruited

8+

ITB-Med LLC

Industry Sponsor

Trials

12

Patients Recruited

390+

Findings from Research

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Chronic Cholestatic Liver Injury Attributable to Vedolizumab. Stine, JG., Wang, J., Behm, BW.[2020]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment of autoimmune liver disease: current and future therapeutic options. Trivedi, PJ., Hirschfield, GM.[2022]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Autoimmune liver disease. Mieli-Vergani, G., Vergani, D.[2023]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A case series analysis of serious exacerbations of viral hepatitis and non-viral hepatic injuries in tocilizumab-treated patients. Biehl, A., Harinstein, L., Brinker, A., et al.[2021]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Research advances in autoimmune liver diseases in 2016]. Li, B., Wang, QX., Ma, X.[2018]

References

1. United States pubmed.ncbi.nlm.nih.gov

Chronic Cholestatic Liver Injury Attributable to Vedolizumab. [2020]

2. United States pubmed.ncbi.nlm.nih.gov

Treatment of autoimmune liver disease: current and future therapeutic options. [2022]

3. India pubmed.ncbi.nlm.nih.gov

Autoimmune liver disease. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

A case series analysis of serious exacerbations of viral hepatitis and non-viral hepatic injuries in tocilizumab-treated patients. [2021]

5. China pubmed.ncbi.nlm.nih.gov

[Research advances in autoimmune liver diseases in 2016]. [2018]