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Aplastic Anemia Clinical Trials

A listing of Aplastic Anemia medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

RESULTS

Found (73) clinical trials

hATG+CsA vs hATG+CsA+Eltrombopag for SAA

This is a superiority trial aiming to increase the 3 month complete response rate. The sample size is calculated on the hypothesis that the experimental treatment will increase the 3 months response rate up to 21% (by 3 folds, based on the 7% reported in Scheinberg et al [17]). Under ...

Phase

Cancer in Inherited Bone Marrow Failure Syndromes

Background A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders. Mutations in IBMFS genes are relevant to carcinogenesis in sporadic cancers. Patients with IBMFS who develop cancer differ in their genetic and/or environmental features from patients with ...

Phase N/A

Stem Cell Transplant Using Peripheral and Cord Blood Stem Cells to Treat Severe Aplastic Anemia and Myelopdysplastic Syndrome

Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are life-threatening bone marrow disorders. For SAA patients, long term survival can be achieved with immunosuppressive treatment. However, of those patients treated with immunosuppressive therapy, one quarter to one third will not respond, and about 50 percent of responders will relapse. Allogeneic ...

Phase

Cytoxan Fludara and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia

OBJECTIVES Primary To determine the probability of engraftment in patients with Fanconi anemia treated with cyclophosphamide, fludarabine phosphate, and antithymocyte globulin followed by HLA-genotypically identical sibling donor hematopoietic stem cell transplantation that is T-cell depleted. Secondary To evaluate the incidence of acute graft-versus-host disease (GVHD) and chronic GVHD in patients ...

Phase

TBI Dose De-escalation for Fanconi Anemia

Study Treatment: Patients will receive voriconazole (antifungal therapy) by mouth beginning 1 month prior to conditioning therapy, if possible. 1) The subject is to receive total body irradiation (300 cGy) with thymic shielding; it will be given six days before the stem cells are given (day -6). 2) Day -5 ...

Phase

Fludarabine Phosphate Cyclophosphamide and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide Mycophenolate Mofetil Tacrolimus and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders

PRIMARY OBJECTIVES: I. Determine safety of nonmyeloablative conditioning and hematopoietic cell transplantation (HCT) from human leukocyte antigen (HLA)-haploidentical related donors for patients with nonmalignant inherited disorders who do not have an HLA-matched related or unrelated donor. SECONDARY OBJECTIVES: I. Determine whether nonmyeloablative conditioning and HCT from an HLA-haploidentical related donor ...

Phase

FANCA Gene Transfer for Fanconi Anemia Using a High-safety High-efficiency Self-inactivating Lentiviral Vector

Fanconi anemia is a rare, inherited disease that is caused by a gene defect and that primarily affects an individual's bone marrow, resulting in decreased production of blood cells. The major problem for most patients is aplastic anemia, the blood counts for red blood cells, white blood cells, and platelets ...

Phase N/A

Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia

Fludarabine-based preparative regimen followed by an allogeneic hematopoietic stem cell transplant using related or unrelated donor in persons 0-70 years of age diagnosed with dyskeratosis congenita or severe aplastic anemia who have bone marrow failure characterized by a requirement for red blood cell and platelet transfusions. Three different preparative regimens ...

Phase N/A

Safety Study of Cord Blood Units for Stem Cell Transplants

Background The requirement for licensure of human cell and blood products became part of the FDA final rules for Human Cells, Tissues, and Tissue-Based Products (HCT/Ps) (March 29, 2004 and May 25, 2005) in an effort to prevent transmission of communicable disease, minimize contamination and preserved integrity and function during ...

Phase

Unrelated And Partially Matched Related Donor PSCT w/ TCR Depletion for Patients With BMF

This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell transplant (HSCT) in patients with acquired and inherited bone marrow failure (BMF) syndromes. Previously established, disease-specific transplant preparative regimens ...

Phase N/A