Clinical Trial: Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors (SAA MUD)

Brief Summary:

Patients have been diagnosed with severe Aplastic Anemia that have not responded to treatment with immunosuppressive therapy (drugs that suppress the immune system, for example Steroids). The immune system is the system in the body that helps protect the body and fights bacterial, viral and fungal infections.

Research studies have shown that patients with Aplastic Anemia have improved survival (may live longer) after receiving a HLA (Human Leukocyte Antigen) identical sibling (brother and sister) stem cell transplants. Patients who do not have matched siblings can undergo immunosuppressive therapy, which has also shown to improve outcome. Unfortunately patients who do not respond to immunosuppressive therapy usually die. The best chance of survival for these patients is an HLA matched unrelated or mismatched related stem cell transplant as described below.

Stem cells are created in the bone marrow. They mature into different types of blood cells that people need including red blood cells which carry oxygen around the body, white blood cells which help fight infections, and platelets which help the blood to clot and prevent bleeding. For a matched unrelated stem cell transplant, stem cells are collected from a person (donor) who is not related to the patient but who has the same type of stem cells. For a mismatched related stem cell transplant, stem cells are collected from a donor who is related to the patient and whose stem cells are almost the same as those of the patient but not exactly. The patient then receives high dose chemotherapy. This chemotherapy kills the stem cells in the patient's bone marrow. Stem cells that have been collected from the donor are then given to the patient to replace the stem cells that have been killed.

The major problems associated with thes

Detailed Summary:

Before patients receive any treatments, they will have the following:

  • Complete history and physical exam
  • Dental consultation
  • Bone marrow aspirate
  • Blood tests
  • Urinanalysis
  • Tests to look at your lungs (Chest X-ray and PFT's and DLCO)
  • Tests to look at your heart (EKG, MUGA, or echocardiogram)
  • Pregnancy test for females of childbearing age.

Once the decision has been made by the patients to have a mismatched related or matched unrelated stem cell transplant, the patient will receive treatment called a "conditioning regimen" to prepare their body for the transplant. The purpose of this conditioning regimen is to kill the stem cells in the bone marrow, so that the patients immune system is suppressed (lowered or stopped from working), so their body will not reject the donors stem cells (transplant).

The medicines which will be given as part of the conditioning regimen include Cyclophosphamide (Cytoxan) and Campath 1H. Both of these medications are approved by the Food and Drug Administration. Cyclophosphamide will be given for four days in a row. Cyclophosphamide is broken down into different chemicals and removed from the body in the urine. These different chemicals can cause bleeding in the bladder. Mesna is a drug that will be given with Cytoxan to prevent the build up of these chemicals that cause bleeding from the bladder. Campath 1H will also be given for four days. After the medicines, the patient will receive total body irradiation (TBI). Total body irradiation is strong doses of rad
Sponsor: Baylor College of Medicine

Current Primary Outcome: Number of Subjects Alive at 100 Days Post Transplant [ Time Frame: 100 days ]

Original Primary Outcome: To assess the safety of the conditioning regimen using Cytoxan, TBI and Campath 1H in patients with severe aplastic anemia (SAA). [ Time Frame: 100 days, 1 year and 2 years post transplant ]

Current Secondary Outcome:

  • Number of Patients With Engraftment Rate at 100 Days Post Transplant [ Time Frame: 100 days post transplant ]
    Absolute neutrophil count greater than 0.5 X 10^9/ml for at least 3 days
  • Number of Patients With Acute GVHD at 100 Days Post Transplant [ Time Frame: 100 days ]
  • Number of Patients With Chronic GVHD at 2 Years Post Transplant [ Time Frame: 2 years ]
  • Number of Subjects Alive at 1 Year Post Transplant [ Time Frame: 1 year ]
  • Number of Subjects Alive at 2 Years Post Transplant [ Time Frame: 2 years ]


Original Secondary Outcome:

  • To assess engraftment and the risk of graft failure in this patient population. [ Time Frame: 28 and 100 days post transplant ]
  • To assess the risk of GVHD both acute and chronic. [ Time Frame: 100 days, 1 year and 2 years post transplant ]
  • To estimate survival at 100 days, 1 year and 2 years. [ Time Frame: 100 days, 1 year, 2 years post transplant ]


Information By: Baylor College of Medicine

Dates:
Date Received: December 19, 2007
Date Started: February 2002
Date Completion:
Last Updated: March 24, 2016
Last Verified: March 2016