Stem Cell Transplantation to Treat Leukocyte Adhesion Deficiency
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|ClinicalTrials.gov Identifier: NCT00031005|
Recruitment Status : Completed
First Posted : February 21, 2002
Last Update Posted : March 4, 2008
This study will investigate the safety and effectiveness of a modified stem cell transplant procedure for treating leukocyte adhesion deficiency (LAD). LAD is an inherited blood disorder of leukocytes (infection-fighting white blood cells) that leaves patients vulnerable to life-threatening infections. Transplantation of donated stem cells (cells produced by the bone marrow that mature into blood cells) can improve the immune system of patients with LAD. However, this procedure carries a significant risk of death, particularly in patients with active infection because it requires completely suppressing the immune system with high-dose chemotherapy and radiation. In addition, T-cells (a type of white blood cell) from the donor may cause what is called graft vs. host disease (GvHD), in which the donor cells recognize the patient's cells as foreign and mount an immune response to destroy them. To try to reduce these risks, the donor's T-cells will be removed from the rest of the stem cells to be transplanted.
Patients with LAD who weigh at least 12 kg (26.4 LB), who do not have an active infection, and who have a family member that is a well-matched donor may be eligible for this study. Pregnant or breast feeding women may not participate. Candidates will have a medical history, physical examination and blood tests, lung and heart function tests, X-rays or computed tomography (CT) scans of the body, and dental and eye examinations. They will fill out questionnaires that measure emotional well being, quality of life and intelligence (the ability to learn and understand).
Stem cells will be collected from both the patient and donor. To do this, the hormone G-CSF will be injected under the skin for several days to move stem cells from the bone marrow to the bloodstream. The stem cells will be collected by apheresis, where blood is drawn through a needle placed in one arm and pumped into a machine separating and removing the required cells. The rest of the blood is then returned through a needle in the other arm.
Before the transplant, a central venous line (large plastic tube) is placed into a major vein. This tube can stay in the body and be used during the entire treatment period to deliver the donated stem cells, give medications, transfuse blood, if needed, and withdraw blood samples. Several days before the transplant procedure, patients will begin a conditioning regimen of low-dose chemotherapy with cyclophosphamide, fludarabine, and Campath 1H. When the conditioning therapy is completed, the stem cells will be infused. To help prevent rejection of donor cells, cyclosporine will be given by mouth or by vein starting 1 month after the transplant procedure.
The average hospital stay for stem cell transplantation is 21 days. After discharge, patients will return for follow-up clinic visits weekly or twice weekly for 2 to 3 months. These visits will include a symptom check, physical examination, and blood tests. Subsequent visits will be scheduled at 4, 6, 9, and 12 months after the transplant, or more often if required, and then yearly
|Condition or disease||Intervention/treatment||Phase|
|Leukocyte-Adhesion Deficiency Syndrome||Procedure: modified stem cell transplant||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Enrollment :||10 participants|
|Official Title:||Treatment of Leukocyte Adhesion Deficiency With Allogeneic Stem Cell Transplantation|
|Study Start Date :||February 2002|
|Study Completion Date :||February 2003|
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00031005
|United States, Maryland|
|National Institute of Allergy and Infectious Diseases (NIAID)|
|Bethesda, Maryland, United States, 20892|