Nonmyeloablative Allogeneic Transplant
Aplastic AnemiaParoxysmal Nocturnal Hemoglobinuria16 moreAllogeneic transplant from a matched sibling for the treatment of a variety of illnesses including bone marrow failure states, leukemias, myelodysplastic or myeloproliferative syndromes, lymphoma, or myeloma using a nonmyeloablative preparative regimen.
Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With...
Severe Aplastic AnemiaBone Marrow Failure SyndromesOur primary objective is to determine if it is feasible for SAA patients to be transplanted using non-myeloablative conditioning and post transplantation cyclophosphamide with partially HLA-mismatched donors.
Efficacy of Romiplostim in Treatment of SAA in Adults Previously Untreated With or Refractory to...
Severe Aplastic Anemia (SAA)Romiplostim has been used in clinical trials for the treatment of severe and very severe aplastic anemia (SAA/vSAA) in Asian participants who are either previously untreated with immunosuppressive therapy (IST) or refractory to IST. This study will evaluate the efficacy of romiplostim in the treatment of participants with SAA/vSAA. The primary objectives of this study are to: Arm 1: Evaluate the efficacy of romiplostim and IST in adult SAA/vSAA participants who are previously untreated with IST (1L) Arm 2: Evaluate the efficacy of romiplostim treatment in adult SAA/vSAA participants who are refractory to IST (2L+)
Post Transplant Cyclophosphamide (Cytoxan) for GvHD Prophylaxis
LeukemiaLymphoma4 moreThe main purpose of this study is to assess the effects of cyclophosphamide (cytoxan) in the post transplant setting to prevent onset of acute graft-versus-host disease (GVHD). The primary objective is to determine the incidence of grade II-IV acute GVHD following Allogeneic (allo) Hematopoeitic Cell Transplant (HCT) using post-transplant cyclophosphamide (cytoxan) for patients with human leukocyte antigen (HLA) matched unrelated (MUD) and mismatched unrelated (MMUD) donors. Other objectives for this study will be the determination of disease-free survival (DFS) and overall survival (OS) following allo HCT and assess the safety of post-transplant cyclophosphamide (cytoxan) for MUD and MMUD transplantation. Disease recurrence and time to recurrence in patients receiving post-transplant cyclophosphamide compared to historical control without post-transplant cyclophosphamide (cytoxan) will also be evaluated. Other objectives will be to determine the time of onset, severity, responsiveness to treatment, organs involved of acute and chronic GVHD as well as observation of Immune Reconstitution over time.
Alemtuzumab and Rituximab in Aplastic Anemia
Aplastic AnemiaThe purpose of the study is to evaluate the efficacy of combination of alemtuzumab and rituximab as first line therapy in aplastic anemia.
A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin)...
Recurrent Severe Aplastic AnemiaRefractory Severe Aplastic AnemiaThis phase I trial evaluates the safety and feasibility of using a reduced-intensity regimen of cyclophosphamide, pentostatin, and anti-thymocyte globulin prior to a CD4+ T-cell depleted haploidentical hematopoietic cell transplant (haploHCT) for the treatment of patients with severe aplastic anemia that does not respond to treatment (refractory) or that has come back (recurrent). Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's deoxyribonucleic acid. It may also lower the body's immune response. Pentostatin blocks a protein needed for cell growth. Anti-thymocyte globulin is an immunosuppressive drug can destroy immune cells known as T-cells. HaploHCT transfers blood-forming stem cells from a healthy partially-matched donor to a patient. Administering a regimen of cyclophosphamide, pentostatin, and anti-thymocyte globulin before haploHCT may help make room for the new, healthy cells and may reduce the risk of graft versus host disease.
Reduced-Intensity Preparative Regimen for Allogeneic Stem Cell Transplantation in Patients With...
Severe Aplastic AnemiaThe purpose of this study is to evaluate the effectiveness of allogeneic transplant after a reduced-intensity preparative regimen for patient, to evaluate survival, and to evaluate the side effects of this treatment. The patient will be in the study for two years for treatment and active monitoring. After treatment and active monitoring are over, the patient's medical condition will be followed indefinitely.
Trial of Two Central Venous Catheter (CVC) Flushing Schemes in Pediatric Hematology and Oncology...
Childhood CancerAplastic Anemia1 moreThe purpose of this study is to determine whether flushing Central Venous Catheters (CVCs) with Normal saline once per week is not inferior to flushing with 10U/ml heparin 3 times per week, in preventing CVC occlusions.
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
Hurler SyndromeFanconi Anemia13 moreThis is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem cell transplant for serious non-malignant diseases as well as to assess the immunological effects of abatacept. Participants will be followed for 2 years.
Traditional Chinese Medicine in the Supportive Management of Anaemic and Cytopenic (Leukopenia,...
Myelodysplastic Syndrome (MDS)Aplastic Anaemia (AA)2 moreThe purpose of this study is to study the efficacy of Traditional Chinese Medicine (TCM) on anaemic and cytopenic haematological disorders including myelodysplastic syndrome (MDS), aplastic anaemia (AA), myelofibrosis (MF) and thalassemia intermedia who do not have or did not respond to available treatment options.