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A Monoclonal Antibody Conditioning Regimen for Allogeneic Stem Cell Transplant in Patients With Fanconi Anemia. - NCT00058565-77030A(Clinical Trial 116293)



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City:  Houston
State:  
TX
Zip Code: 77030
Conditions: Fanconi's Anemia
Purpose: 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.
Study summary: 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.
Criteria: Eligibility Criteria: - Patients with Fanconi anemia of all ages are eligible - Diagnosis of Fanconi anemia must be confirmed by studies of peripheral blood or bone marrow sensitivity to mitomycin-C or DEB - Severe aplasia as evidenced by a hypocellular bone marrow and at least 2 of the 3 criteria below: - ANC < 500/mm3 - Hemoglobin < 10 gm/dl with reticulocyte count < 1% - Platelet count < 50,000/mm3 - Availability of an HLA matched or mismatched (up to one haplotype) family member who has been documented not to have Fanconi anemia or of an unrelated HLA matched stem cell donor. Fully matched is defined at 6/6 match by high resolution DR based DNA typing. - Female patients of childbearing age must have a negative pregnancy test and be willing to use an effective means of birth control. Exclusion Criteria: - Patients with a life expectancy (<6 weeks) limited by diseases other than FA. - Patients with leukemic transformation - Patients with symptomatic cardiac disease, or evidence of significant cardiac disease by echocardiogram (i.e., shortening fraction <25%). - Patients with severe renal disease(Creatinine >2 x normal for age) - Patients with known allergy to rat serum products. - Patients with a Karnofsky score <70%. - Patients with a severe infection that on evaluation by the Principal Investigator precludes ablative chemotherapy or successful transplantation. - Patients with severe personality disorder or mental illness. - Patients with documented HIV positivity.
Study is available at: Baylor College of Medicine
Houston, TX 77030
United States

Primary Contact:
Malcolm K Brenner, MB, PhD,
Email: mbrenner@bcm.tmc.edu
Phone: 832-824-4663
If you are interested in this clinical trial please use the contact information above. If you would like to get additional information about this clinical trial please visit ClinicalTrials.gov.
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Data Source: ClinicalTrials.gov
Date Processed: March 21, 2011
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