Stem Cell Transplantation to Treat Systemic Mastocytosis

Completed · Phase 2

Conditions studied: Mastocytosis

In brief

This study will investigate the safety and effectiveness of an experimental stem cell transplant procedure for treating mastocytosis-a disease of abnormal mast cell growth. Patients often feel faint, have skin problems, joint and bone pain, low blood counts and enlarged liver, spleen or lymph nodes. As yet, there is no cure for mastocytosis, and treatment is aimed at controlling symptoms. Stem cells are cells produced by the bone marrow that mature into the different blood components-white cells, red cells and platelets. Transplantation of allogeneic (donated) stem cells is a mainstay of therapy for some forms of leukemia. Patients first receive intensive chemotherapy and radiation to rid the body of cancer cells. This "conditioning" is followed by transplantation of donated stem cells to generate new, healthy bone marrow. In addition to producing the new bone marrow, the donated cells also fight any residual tumor cells that might have remained in the body. This is called a "graft-versus-tumor" effect. This study will examine whether a stem cell transplant from a healthy donor can similarly target and destroy mast cells in a "graft-versus-mast cell" effect. Also, to try to reduce the harmful side effects of chemotherapy and radiation, this study will use lower dose chemotherapy and no radiation. Patients with advanced mastocytosis between 10 and 80 years old may be eligible for this study. They will be tested for HLA type matching with a sibling and will undergo a medical history, physical examination and several tests to determine eligibility for the study. Participants will undergo apheresis to collect lymphocytes (a type of white blood cell) for immune function tests. In this procedure, blood is drawn through a needle in the arm, similar to donating a unit of blood. The lymphocytes are then separated and collected by a cell separator machine, and the rest of the blood is returned through a needle in the other arm. Patients will also have a central venous line (flexible plastic tube) placed in their upper chest leading to a vein. This line will remain in place throughout the transplant and recovery period and will be used to transfuse blood components, administer medicines, infuse the donated stem cells, and draw blood for tests. Patients will begin conditioning with cyclophosphamide, starting 7 days before the transplant, and fludarabine, starting 5 days before the transplant, to prevent rejection of the donated cells. From 1 to 3 days after the chemotherapy is completed, the stem cells will be transfused through the central venous line. Also, from 4 days before the transplantation until about 3 months after the procedure, patients will receive cyclosporine and mycophenolate mofetil-drugs that help prevent both rejection of the donated cells and attack by the donor cells on the patient's cells (called graft-versus-host disease). Patients will stay in the hospital about 20 to 30 days after the transplant. After discharge, they will continue to take antibiotics, cyclosporine and mycophenolate mofetil at home. If the mastocytosis progresses, cyclosporine and mycophenolate mofetil will be tapered over 4 weeks. If the mastocytosis persists, patients may receive additional transfusions of donor lymphocytes to help kill the mast cells. Patients' progress will be followed weekly or twice weekly for 3 months, then at 6, 12, 18, 24, 30, 36, 48 and 60 months after transplant, and then twice a year for various tests, treatments and examinations.

Key facts

Study ID
NCT00006413
Run by
National Heart, Lung, and Blood Institute (NHLBI)
People needed
25
Starts
2000-10-13
Expected to finish
2006-12-14
Last updated by the study team
2017-07-02

Who can join

Age: 2 and older, up to 80. Sex: any. Healthy volunteers: not accepted.

Where it is running

Full record on ClinicalTrials.gov

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