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Sponsored by: |
National Heart, Lung, and Blood Institute (NHLBI) |
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Information provided by: | National Cancer Institute (NCI) |
ClinicalTrials.gov Identifier: | NCT00608413 |
RATIONALE: Giving chemotherapy, antithymocyte globulin, and total-body irradiation before a donor umbilical cord blood transplant and a donor peripheral stem cell transplant helps stop the growth of abnormal cells. It also stops the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving tacrolimus, mycophenolate mofetil, and prednisone or methylprednisolone before and after transplant may stop this from happening.
PURPOSE: This phase II trial is studying how well giving cyclophosphamide together with fludarabine, antithymocyte globulin, and total-body irradiation followed by donor umbilical cord blood transplant and donor peripheral stem cell transplant works in treating patients with severe aplastic anemia or myelodysplastic syndromes associated with severe neutropenia that has not responded to immunosuppressive therapy.
Condition | Intervention | Phase |
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Leukemia Myelodysplastic Syndromes Precancerous/Nonmalignant Condition |
Drug: anti-thymocyte globulin Drug: cyclophosphamide Drug: fludarabine phosphate Drug: methylprednisolone Drug: mycophenolate mofetil Drug: prednisone Drug: tacrolimus Procedure: in vitro-treated peripheral blood stem cell transplantation Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation Procedure: total-body irradiation Procedure: umbilical cord blood transplantation |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Open Label |
Official Title: | Co-Infusion of Umbilical Cord Blood and Haploidentical CD34+ Cells Following Nonmyeloablative Conditioning as Treatment for Severe Aplastic Anemia and MDS Associated With Severe Neutropenia Refractory to Immunosuppressive Therapy |
Estimated Enrollment: | 40 |
Study Start Date: | January 2008 |
Estimated Primary Completion Date: | December 2013 (Final data collection date for primary outcome measure) |
OBJECTIVES:
Primary
Secondary
OUTLINE:
After completion of study treatment, patients are followed every 3 months for 3 years, every 6 months for 2 years, and then annually thereafter.
Ages Eligible for Study: | 8 Years to 55 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
Diagnosed of 1 of the following:
Severe aplastic anemia characterized by all of the following:
Myelodysplastic syndromes characterized by all of the following:
At least one HLA-haploidentical (≥ 3/6 HLA matched) related donor (2-75 years old) available
At least one 4/6 HLA-matched (HLA-A, B, and DR loci) umbilical cord blood (UCB) unit from the National Marrow Donor Program available
UCB unit must contain a minimum total nucleated cells (TNC) (prior to thawing) of ≥ 1.5 x 10^7 cells/kg of recipient body weight
PATIENT CHARACTERISTICS:
PRIOR CONCURRENT THERAPY:
United States, Maryland | |
NIH - Warren Grant Magnuson Clinical Center | Recruiting |
Bethesda, Maryland, United States, 20892-1182 | |
Contact: Clinical Trials Office - NIH - Warren Grant Magnuson Clinical 800-411-1222 |
Study Chair: | Richard W. Childs, MD | National Heart, Lung, and Blood Institute (NHLBI) |
Responsible Party: | National Heart, Lung, and Blood Institute ( Richard W. Childs ) |
Study ID Numbers: | CDR0000585757, NHLBI-08-H-0046 |
Study First Received: | February 5, 2008 |
Last Updated: | December 25, 2008 |
ClinicalTrials.gov Identifier: | NCT00608413 |
Health Authority: | Unspecified |
refractory anemia with ringed sideroblasts previously treated myelodysplastic syndromes secondary myelodysplastic syndromes refractory anemia |
aplastic anemia de novo myelodysplastic syndromes childhood myelodysplastic syndromes |
Prednisone Precancerous Conditions Refractory anemia Methylprednisolone Prednisolone acetate Cyclophosphamide Tacrolimus Leukemia Preleukemia Anemia, Refractory Anemia, Aplastic Neoplasm Metastasis Mycophenolate mofetil Methylprednisolone Hemisuccinate |
Myelodysplastic syndromes Hematologic Diseases Myelodysplasia Myelodysplastic Syndromes Anemia Methylprednisolone acetate Fludarabine monophosphate Antilymphocyte Serum Neutropenia Prednisolone Fludarabine Bone Marrow Diseases Aplastic anemia |
Antimetabolites Anti-Inflammatory Agents Antimetabolites, Antineoplastic Immunologic Factors Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Physiological Effects of Drugs Hormones, Hormone Substitutes, and Hormone Antagonists Antiemetics Hormones Neuroprotective Agents Pathologic Processes Therapeutic Uses Syndrome Alkylating Agents |
Disease Neoplasms by Histologic Type Antineoplastic Agents, Hormonal Gastrointestinal Agents Immunosuppressive Agents Protective Agents Glucocorticoids Pharmacologic Actions Neoplasms Autonomic Agents Myeloablative Agonists Antineoplastic Agents, Alkylating Peripheral Nervous System Agents Antirheumatic Agents Central Nervous System Agents |