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Sponsored by: |
Fairview University Medical Center |
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Information provided by: | Office of Rare Diseases (ORD) |
ClinicalTrials.gov Identifier: | NCT00006054 |
OBJECTIVES: I. Provide curative immunoreconstituting allogeneic bone marrow transplantation for patients with primary immunodeficiencies.
II. Determine relevant outcomes of this treatment in these patients including quality of survival, extent of morbidity and mortality from complications of the treatment (e.g., graft versus host disease, regimen related toxicities, B- cell lymphoproliferative disease), and completeness of functional immunoreconstitution.
Condition | Intervention |
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Immunologic Deficiency Syndromes Chediak-Higashi Syndrome Common Variable Immunodeficiency Graft Versus Host Disease X-Linked Lymphoproliferative Syndrome Familial Erythrophagocytic Lymphohistiocytosis Hemophagocytic Lymphohistiocytosis X-Linked Agammaglobulinemia Wiskott-Aldrich Syndrome Chronic Granulomatous Disease X-Linked Hyper IgM Syndrome Severe Combined Immunodeficiency Leukocyte Adhesion Deficiency Syndrome Virus-Associated Hemophagocytic Syndrome |
Drug: anti-thymocyte globulin Drug: busulfan Drug: cyclophosphamide Drug: cyclosporine Drug: etoposide Drug: methotrexate Drug: methylprednisolone Drug: prednisone Procedure: Allogeneic Bone Marrow Transplantation |
Study Type: | Interventional |
Study Design: | Treatment |
Study Start Date: | March 2000 |
PROTOCOL OUTLINE: Patients with severe combined immunodeficiency (SCID) using a matched sibling donor receive allogeneic bone marrow or umbilical cord blood transplantation on day 0. Patients receive graft versus host disease (GVHD) prophylaxis with methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV on days -3 to 50. Patients with SCID using donors other than histocompatible siblings, Wiskott Aldrich syndrome using a histocompatible sibling donor, Wiskott Aldrich syndrome and under 5 years of age using donors other than histocompatible siblings, X-linked CD40 ligand deficiency using a histocompatible sibling donor, X-linked CD40 ligand deficiency and under 5 years of age using donors other than histocompatible siblings, other primary immunodeficiencies without manifestations of hemophagocytosis using a histocompatible sibling donor, or other primary immunodeficiencies without manifestations of hemophagocytosis and under 5 years of age using donors other than histocompatible siblings receive busulfan IV over 2 hours every 6 hours on days -9 to -6, cyclophosphamide IV on days -5 to -2, and antithymocyte globulin (ATG) twice daily on days -4 to -1. Allogeneic bone marrow or umbilical cord blood transplantation takes place on day 0. Patients receive graft versus host disease (GVHD) prophylaxis with methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV on days -3 to 50. Patients with hemophagocytic lymphohistiocytosis, Chediak Higashi syndrome, X-linked lymphoproliferative syndrome, severe progressive Langerhans cell histiocytosis, or other primary immunodeficiencies with complications of hemophagocytosis receive busulfan IV over 2 hours every 6 hours on days -9 to -6, cyclophosphamide IV over 2 hours on days -5 to -2, etoposide IV over 22 hours on days -5 to -3, and ATG IV twice daily on days -2, -1, 1, and 2.
Allogeneic bone marrow or umbilical cord blood transplantation takes place on day 0. Patients receive graft versus host disease (GVHD) prophylaxis with methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV on days -3 to 50. Patients with Wiskott Aldrich syndrome or other primary immunodeficiencies without manifestations of hemophagocytosis, who are over 5 years of age and using donors other than histocompatible siblings, receive busulfan IV over 2 hours every 6 hours on days -6 and -5, cyclophosphamide IV over 2 hours on days -4 and -3, total body irradiation on day -2, and ATG IV over 2 hours twice daily on days -2, -1, 2, and 3. Allogeneic bone marrow or umbilical cord blood transplantation takes place on days 0 and 1. Patients receive GVHD prophylaxis with methylprednisolone IV every 12 hours on days 2-21, oral prednisone every 12 hours on days 22-100 and then tapered off over days 101 to 128, and cyclosporine IV over 2 hours every 8-12 hours on days
Ages Eligible for Study: | up to 35 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
United States, Minnesota | |
Fairview University Medical Center | |
Minneapolis, Minnesota, United States, 55455 |
Study Chair: | K. Scott Baker | Fairview University Medical Center |
Study ID Numbers: | 199/15104, UMN-MT-1995-26, UMN-MT-9526 |
Study First Received: | July 5, 2000 |
Last Updated: | June 23, 2005 |
ClinicalTrials.gov Identifier: | NCT00006054 History of Changes |
Health Authority: | Unspecified |
Chediak-Higashi syndrome Langerhans cell histiocytosis Wiskott-Aldrich syndrome X-linked agammaglobulinemia X-linked hyper IgM syndrome X-linked lymphoproliferative syndrome chronic granulomatous disease common variable immunodeficiency complement deficiency disease-related problem/condition familial erythrophagocytic lymphohistiocytosis |
genetic diseases and dysmorphic syndromes graft versus host disease hematologic disorders hemophagocytic lymphohistiocytosis histiocytosis immunologic disorders and infectious disorders leukocyte adhesion deficiency syndrome primary immunodeficiency disease rare disease severe combined immunodeficiency virus-associated hemophagocytic syndrome |
Anti-Inflammatory Agents Prednisone Cyclosporine Agammaglobulinemia Methylprednisolone Hormone Antagonists Miconazole Hormones, Hormone Substitutes, and Hormone Antagonists Antiemetics Leukocyte Disorders Hormones Cyclosporins Graft Versus Host Disease Wiskott-Aldrich Syndrome Thrombocytopenia |
Hemorrhagic Disorders Methotrexate Hyperkinesis Infant, Newborn, Diseases Etoposide Metabolic Disorder Virus Associated Hemophagocytic Syndrome Methylprednisolone Hemisuccinate Purpura Letterer-Siwe Disease Lymphohistiocytosis, Hemophagocytic Metabolic Diseases Immunoproliferative Disorders Antineoplastic Agents, Hormonal Hematologic Diseases |
Anti-Inflammatory Agents Anti-Infective Agents Prednisone Antimetabolites, Antineoplastic Agammaglobulinemia Cyclosporine Molecular Mechanisms of Pharmacological Action Methylprednisolone Physiological Effects of Drugs DNA Repair-Deficiency Disorders Hormones, Hormone Substitutes, and Hormone Antagonists Antiemetics Leukocyte Disorders Hormones Cyclosporins |
Wiskott-Aldrich Syndrome Pathologic Processes Thrombocytopenia Hemorrhagic Disorders Therapeutic Uses Abortifacient Agents Infant, Newborn, Diseases Methotrexate Dermatologic Agents Nucleic Acid Synthesis Inhibitors Methylprednisolone Hemisuccinate Phagocyte Bactericidal Dysfunction Lymphohistiocytosis, Hemophagocytic Reticuloendotheliosis Metabolic Diseases |