View clinical trials related to Fanconi Anemia.
Filter by:OBJECTIVES: I. Determine the probability of engraftment with total body irradiation, cyclophosphamide, fludarabine, and anti-thymocyte globulin followed by HLA nongenotypically identical donor, T-cell depleted hematopoietic cell transplantation in patients with Fanconi's anemia. II. Determine the incidence of acute and chronic graft-versus-host disease in these patients after undergoing this treatment regimen. III. Determine the one-year survival rate in these patients after undergoing this treatment regimen. IV. Determine the toxicity of this treatment regimen in these patients. V. Determine the incidence of relapse in patients with myelodysplastic syndrome or acute myeloid leukemia after undergoing this treatment regimen.
OBJECTIVES: I. Determine the safety of transferring the Fanconi anemia complementation group C (FACC) gene to hematopoietic progenitors by retroviral mediated gene transfer in patients with Fanconi's anemia, complementation group C. II. Determine the extent of engraftment following this treatment regimen without prior ablation of recipient marrow in these patients. III. Determine the ability of this treatment regimen to correct the cell phenotype and improve hematopoietic function in these patients.
OBJECTIVES: I. Determine the effectiveness of moderate dose cyclophosphamide and radiotherapy in terms of improving survival and reducing the morbidity following allogeneic bone marrow transplantation in patients with myelodysplastic syndrome and acute leukemia related to Fanconi's anemia.
OBJECTIVES: I. Determine the effectiveness of moderate dose cyclophosphamide and total lymphoid radiotherapy in terms of improving the survival and reducing the morbidity following allogeneic bone marrow transplantation in patients with Fanconi's aplastic anemia.
OBJECTIVES: I. Assess the efficacy of recombinant human granulocyte colony-stimulating factor (G-CSF) in raising the absolute neutrophil count, platelet count, and hemoglobin level in patients with inherited bone marrow failure syndromes. II. Assess the efficacy of a reduced maintenance dose in patients who respond to daily G-CSF. III. Assess the toxic effects of G-CSF in these patients. IV. Measure bone marrow progenitor colonies before and after G-CSF. V. Measure CD34-positive cells in marrow and blood before and after G-CSF using flow cytometry and immunohistochemistry.
OBJECTIVES: I. Ascertain whether stem cell transplantation (SCT) is an effective method by which missing or dysfunctional enzymes can be replaced in patients with various inborn errors of metabolism. II. Determine whether clinical manifestations of the specific disease may be arrested or reversed by this treatment.
OBJECTIVES: I. Classify renal tubular defects using clinical and biochemical findings in patients with Fanconi syndrome and cystinosis.
Diamond Blackfan anemia (DBA) is a condition in which the bone marrow is underdeveloped. DBA is considered a congenital disease, meaning patients are born with it. In DBA there is a lack of cells that give rise to red blood cells. The other elements produced in the bone marrow, such as white blood cells and platelets, are normal. Standard treatments used for this disorder such as steroids and bone marrow transplants are associated with failure, relapse, side-effects, increased morbidity, and even death. Two drugs, antithymocyte globulin (ATG) and cyclosporin have been used to treat DBA, but have only provided occasional responses. No study has ever combined these two drugs for the treatment of DBA. This study is designed to explore the combined use of ATG and cyclosporine as a rational approach to the treatment of DBA.
Fanconi's Anemia is an inherited disorder that can produce bone marrow failure. In addition, some patients with Fanconi's anemia have physical defects usually involving the skeleton and kidneys. The major problem for most patients is aplastic anemia, the blood counts for red blood cells, white blood cells, and platelets are low because the bone marrow fails to produce these cells. Some patients with Fanconi's anemia can develop leukemia or cancers of other organs. Many laboratory studies have suggested that Fanconi's anemia is caused by an inherited defect in the ability of cells to repair DNA. Recently, the gene for one of the four types of Fanconi's anemia, type C, has been identified. It is known that this gene is defective in patients with Fanconi's anemia type C. Researchers have conducted laboratory studies that suggest Fanconi's anemia type C may be treatable with gene therapy. Gene therapy works by placing a normal gene into the cells of patients with abnormal genes responsible for Fanconi's anemia type C. After the normal gene is in place, new normal cells can develop and grow. Drugs can be given to these patients kill the remaining abnormal cells. The new cells containing normal genes and will not be harmed by these drugs. The purpose of this study is to test whether researchers can safely place the normal Fanconi's anemia type C gene into cells of patients with the disease. The gene will be placed into special cells in the bone marrow called stem cells. These stem cells are responsible for producing new red blood cells, white blood cells, and platelets.
To evaluate if HLA-mismatched, unrelated-donor umbilical cord blood stem and progenitor cell units (UCBU) offered a clinically acceptable alternative to matched unrelated-donor allogeneic bone marrow for transplantation with 180-day disease free survival as the endpoint. HLA typing was performed using DNA-base high resolution methods to determine HLA alleles. Patients with "true" HLA 3/6 and 4/6 matches were evaluated. In addition, a separate study in adults addressed the problem of limited cell dose and engraftment failure. The study was not planned as a randomized comparative clinical trial. Instead, it is a phase II/III efficacy study.