Clinical Trial: Sequential, Related Donor Partial Liver Transplantation Followed by Bone Marrow Transplantation for Hepatocellular Carcinoma (HCC)

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Sequential, Related Donor Partial Liver Transplantation Followed by Bone Marrow Transplantation for Fibrolamellar or Non-fibrolamellar Hepatocellular Carcinoma (HCC) Including Fibrolamellar HCC

Brief Summary:

This trial is a phase II, single arm, open-label, single center study to assess a reduced-intensity conditioning regimen, bone marrow transplantation and high dose PTCy in recipients of a partial liver allograft from a Human Leukocyte Antigen (HLA)-matched or -haploidentical living related donor in patients with HCC.

The primary objective of this trial is to characterize recurrence-free survival at 1 year following bone marrow transplantation among recipients of prior partial liver transplantation from the same donor.


Detailed Summary:

The purpose of this study is to characterize the safety and anti-tumor efficacy of sequential partial liver transplantation followed by bone marrow transplantation from the same living related donor. This treatment applies to patients whose cancer remains confined to the liver but is too widespread to be removed by surgery or treated by a liver transplant from a deceased donor. The purpose of this combined treatment is to reduce the risk of the cancer coming back after the liver transplant The bone marrow transplant may reduce the risk of cancer relapse in two ways. First, patients who have combined bone marrow and solid organ transplants may be able to get off all anti-rejection drugs, which inhibit the immune system from destroying cancer cells. Second, the donor's bone marrow contains cells of the immune system, which can attack any cancer cells that remain after the liver transplant.

This trial is a phase II, single arm, open-label, single center pilot study to assess a reduced-intensity conditioning regimen, bone marrow transplantation and high dose post-transplantation cyclophosphamide (PTCy) in recipients of a partial liver allograft from a Human Leukocyte Antigen (HLA)-matched or -haploidentical living related donor in patients with HCC. The trial includes analyses of tumor characteristics and the number and phenotype of tumor infiltrating lymphocytes in the explanted tumor. The trial also includes periodic monitoring of circulating hepatocytes to correlate with tumor response.

The study is expected to take two years to complete accrual of six patients, and the primary objective of this trial is to characterize recurrence-free survival at 1 year following bone marrow transplantation among recipients of prior partial liver transplantation from the same donor. Secondary objectives include documenting percentage of pa
Sponsor: Sidney Kimmel Comprehensive Cancer Center

Current Primary Outcome: 1 year disease-free survival (at 1 year after BMT) [ Time Frame: 1 year ]

Disease-free is defined as the lack of radiographic evidence of recurrence by computed tomography or MRI.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Occurrence of Graft versus Host Disease [ Time Frame: 1 year ]
    Determine the cumulative incidences of acute grades II-IV, III-IV and chronic graft-versus-host disease
  • Death [ Time Frame: 1 year ]
    Proportion of transplanted participants who die
  • Liver allograft failure [ Time Frame: 1 year ]
    Determine the proportion of transplanted participants with liver allograft rejection demonstrated by a biopsy or clinically if a biopsy cannot be performed.


Original Secondary Outcome: Same as current

Information By: Sidney Kimmel Comprehensive Cancer Center

Dates:
Date Received: February 26, 2016
Date Started: March 2016
Date Completion: January 2020
Last Updated: May 17, 2016
Last Verified: May 2016