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A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Adults and Children With Bone Marrow Failure Syndromes or Inherited Metabolic or Hematologic Diseases (NCT00003662)

National Cancer Institute (NCI)
RATIONALE: Umbilical cord blood transplantation may be able to replace cells destroyed by chemotherapy or radiation therapy. PURPOSE: Phase II trial to study the effectiveness of umbilical cord blood transplantation in treating patients who have hematologic cancer or other hematologic or metabolic diseases.
  • Biological: anti-thymocyte globulin
    • Drug: busulfan
      • Drug: cyclophosphamide
        • Drug: cyclosporine
          • Drug: melphalan
            • Drug: methylprednisolone
              • Procedure: umbilical cord blood transplantation
                • Radiation: radiation therapy
                  Ages eligible for Study
                  Genders eligible for Study
                  Accepts Healthy Volunteers
                  OBJECTIVES: I. Determine the rates of durable engraftment in patients with severe aplastic anemia, myelodysplastic syndrome, inborn errors of metabolism, or inherited hematopoietic disorders refractory to medical management, who are undergoing high dose chemoradiotherapy followed by unrelated cord blood (UCB) transplantation. II. Determine the incidence and severity of acute and chronic graft-versus-host disease in these patients. III. Monitor overall and event-free survival of these patients. IV. Evaluate rate and quality of immunologic reconstitution of these patients. V. Determine whether nucleated cell or progenitor cell content of the graft is predictive of engraftment.

                  OUTLINE: This is a multicenter study. Patients are stratified according to low vs high weight. Patients with severe aplastic anemia, myelodysplastic syndrome, or bone marrow failure receive cyclophosphamide IV over 1 hour on days -6 to -3 or melphalan IV over 20 minutes on days -4 to -2, antithymocyte globulin (ATG) IV over 4 hours or methylprednisolone IV over 1 hour twice a day on days -3 to -1, and total lymphoid irradiation on day -1. On day 0, patients receive umbilical cord blood (UCB) infusion. Patients with inborn errors of metabolism or inherited hematopoietic disorders receive oral busulfan every 6 hours on days -9 to -6, cyclophosphamide IV over 1 hour on days -5 to -2 or melphalan IV over 20 minutes on days -4 to -2, and ATG IV over 4 hours or methylprednisolone IV over 1 hour on days -3 to -1. On day 0, patients receive UCB infusion. Patients with Fanconi's anemia receive ATG IV over 4 hours or methylprednisolone IV over 1 hour on days -6 to -1, cyclophosphamide IV over 1 hour on days -5 to -2, thoracoabdominal irradiation on day -1, and then the UCB infusion on day 0. Patients also receive cyclosporine and methylprednisolone beginning on day -2 and continuing as necessary as graft-versus-host disease prophylaxis. Patients are followed indefinitely for survival and late toxicity.

                  PROJECTED ACCRUAL: A total of 4-90 patients will be accrued for this study within 5 years.

                  15 locations

                  United States (15)
                  • University of Florida Health Science Center
                    Not specified
                    Gainesville, Florida, United States, 32610-0296
                  • Division of Pediatric Surgery
                    Not specified
                    Jacksonville, Florida, United States, 32207
                  • H. Lee Moffitt Cancer Center and Research Institute
                    Not specified
                    Tampa, Florida, United States, 33612
                  • Rush-Presbyterian-St. Luke's Medical Center
                    Not specified
                    Chicago, Illinois, United States, 60612
                  • University of Chicago Cancer Research Center
                    Not specified
                    Chicago, Illinois, United States, 60637
                  • Children's Hospital of New Orleans
                    Not specified
                    New Orleans, Louisiana, United States, 70118
                  • Cardinal Glennon Children's Hospital
                    Not specified
                    Saint Louis, Missouri, United States, 63104
                  • Hackensack University Medical Center
                    Not specified
                    Hackensack, New Jersey, United States, 07601
                  • Roswell Park Cancer Institute
                    Not specified
                    Buffalo, New York, United States, 14263-0001
                  • New York Blood Center
                    Not specified
                    New York, New York, United States, 10021
                  • Lineberger Comprehensive Cancer Center, UNC
                    Not specified
                    Chapel Hill, North Carolina, United States, 27599-7295
                  • Duke Comprehensive Cancer Center
                    Not specified
                    Durham, North Carolina, United States, 27710
                  • St. Christopher's Hospital for Children
                    Not specified
                    Philadelphia, Pennsylvania, United States, 19134-1095
                  • Medical University of South Carolina
                    Not specified
                    Charleston, South Carolina, United States, 29425-0721
                  • University of South Carolina School of Medicine
                    Not specified
                    Columbia, South Carolina, United States, 29203
                  31 July, 1998
                  02 March, 2011
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