Clinical Trial: Stem Cell Transplantation in Patients With Primary Biliary Cirrhosis

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional

Official Title: Non-myeloablative Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Primary Biliary Cirrhosis: A Phase I Study

Brief Summary: This disease is believed to be due to immune cells, cells which normally protect the body, but are now destroying the bile ducts in the liver. When the ducts are damaged, bile builds up in the liver and damages liver tissue. Over time, the disease can cause cirrhosis and may make the liver stop working. This study is designed to examine whether treating patients with high dose Cyclophosphamide and Fludarabine (drugs which reduce the function of your immune system) and CAMPATH-1H (a protein that kills the immune cells that are thought to be causing PBC), followed by return of blood stem cells that have been previously collected from patient brother or sister will stop or reverse the disease. The purpose of the Cyclophosphamide, Fludarabine and CAMPATH-1H is to decrease immune system. The purpose of the stem cell infusion is to restore blood production, which will be severely impaired by the Cyclophosphamide, Fludarabine and CAMPATH-1H, and to produce a normal immune system that will no longer attack the body.

Detailed Summary:

There will be no randomization in this study. All subjects who are determined to be eligible for the study treatment will receive high dose cyclophosphamide, fludarabine and CAMPATH-1H followed by infusion of allogeneic peripheral blood stem cells. The procedures the subject will undergo are as follows:

  1. Physician visit to determine potential eligibility for the study. Subjects will be evaluated by a transplant physician. They will have a complete physical examination and will provide a full medical history at these visits. In addition, subjects will be asked to complete a quality of life Questionnaire . Finally, the study will be described in detail by the transplant physician and nurse and the consent form will be provided to be taken home to read.
  2. HLA Testing. In order to proceed on this study, subjects will need to have a HLA identical matched sibling donor. The subject and his or her siblings will need to undergo HLA typing in order to determine whether there is a match or Cord blood stem cells will be obtained from the New York Blood Center Cord Blood Registry which is an internationally recognized registry or, if a match is not available, from Stemcyte, a commercial registry that specializes in minority donors. One unit of HLA matched cord blood unit will be infused on day zero.
  3. Insurance verification. Subjects who remain interested in pursuing the study treatment and who have severe scleroderma that is following a progressive course will proceed to the insurance verification phase. Third party payment or self-pay must be verified before subjects can proceed.
  4. Consent form. Prior to proceeding, the appropriate signatures will be obtained on the consent form. Subjects will be given an opportunity to ask further questions of the atten
    Sponsor: Richard Burt, MD

    Current Primary Outcome: No liver-related death or LTx over the 2-year (extended to 5 years) follow-up; Normalization of serum alkaline phosphatase over 6 months;Amelioration of PBC histological stage with reduction of both inflammation and fibrosis scores 42 [ Time Frame: 5 years after transplant ]

    Original Primary Outcome:

    • No liver-related death or LTx over the 2-year (extended to 5 years) follow-up
    • Normalization of serum alkaline phosphatase over 6 months
    • Amelioration of PBC histological stage with reduction of both inflammation and fibrosis scores 42


    Current Secondary Outcome:

    Original Secondary Outcome:

    • Histological scoring based on the criteria for inflammation and fibrosis 42
    • Mayo score calculation
    • Normalization or 50% improvement of LFT
    • Quality of life changes 38
    • Changes in serum lipid profile


    Information By: Northwestern University

    Dates:
    Date Received: October 24, 2006
    Date Started: January 2006
    Date Completion:
    Last Updated: October 4, 2012
    Last Verified: October 2012