Last updated on December 2007

A Monoclonal Antibody Conditioning Regimen for Allogeneic Stem Cell Transplant in Patients With Fanconi Anemia.


Brief description of study

Patients eligible for this study have an aggressive blood disease, Fanconi Anemia (FA) that requires an allogeneic (meaning the cells come from a donor) stem cell transplant using a family member or nearly identical matched donor. Stem cells are cells in the bone marrow and blood that can form a whole new blood system. Usually, these patients are given high doses of chemotherapy before receiving a stem cell transplant in order to keep the immune system from rejecting the donor stem cells, and to kill any diseased cells that remain in the body. However, some patients, due to complications with their condition, may have a high risk of acquiring possibly life-threatening treatment-related side effects such as graft versus host disease (GVHD). GVHD occurs when the new stem cells (graft) recognize that the body tissues of the patient (host) are different from those of the donor. When this happens, cells in the graft may attack the host organs, primarily the skin, the liver and the intestines. Even with the strongest available treatments, graft rejection can occur or some diseased cells may survive and cause relapse. Instead of the high dose chemotherapy usually given before a transplant, this research study uses a new pre-treatment combination of two drugs, Anti-CD45 and CAMPATH-1H. Anti-CD45 and CAMPATH-1H are antibodies against certain types of blood cells, including FA cells. CAMPATH-1H is particularly important because it stays active in the body for a long time after infusion, which means it may work longer at preventing GVHD symptoms.

Detailed Study Description

Before treatment begins, patients will be evaluated to confirm that they meet the requirements of this study. Participants will need to have a central line. This is a thin plastic catheter or tube that is placed during surgery into one of the large veins in the chest or neck. Central lines are used to give intravenous (IV) medications (medicines that go directly into the vein) or to take blood samples without you having to endure frequent needle sticks. CAMPATH-1H will be given as a daily 4-hour IV (intravenous, by vein) infusion for three days. Anti-CD45 will be given as a daily 6-hour IV infusion over the next 4 days. There will then be a one-day rest period before receiving the stem cell transplant. After the transplant, participants will be followed closely. While in hospital, patients will have a physical examination done daily. They will also have a daily urinalysis (where some of your urine is tested to see what it contains) and blood tests done, which are standard for anyone receiving a transplant. To see how the Anti-CD45 is working in the body, blood will be drawn 2 hours before each infusion of the Anti-CD45, at the end of each infusion of the CD45 and then 24 hours (1 day) and 48 hours (2 days) after the end of the last infusion of Anti-CD45. No more than 42 mls (8-9 teaspoonfuls) of total blood will be drawn over the course of the four Anti-CD45 infusions. When able to leave the hospital, the patient will be followed in the outpatient clinic and will have visits and tests which are standard (the same) for anyone who receives a transplant.

Clinical Study Identifier: NCT00058565

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Malcolm K Brenner, MB, PhD,

Baylor College of Medicine
Houston, TX United States
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Malcolm K Brenner, MB, PhD

Texas Children's Hospital
Houston, TX United States
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Malcolm M Brenner, MB, PhD

The Methodist Hospital
Houston, TX United States
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