Treatment Trials

3 Clinical Trials for Various Conditions

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      RECRUITING
      Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
      Description

      This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.

      Conditions
      SCIDOmenn's SyndromeReticular DysgenesisWiskott-Aldrich SyndromeBare Lymphocyte SyndromeCommon Variable ImmunodeficiencyChronic Granulomatous DiseaseCD40 Ligand DeficiencyHyper IgM SyndromeX-linked Lymphoproliferative DiseaseHemophagocytic LymphohistiocytosisGriscelli SyndromeChediak-Higashi SyndromeLangerhan's Cell Histiocytosis
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      RECRUITING
      Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
      Description

      This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft failure and to aid in immune reconstitution following hematopoietic stem cell transplantation.

      Conditions
      Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)Hereditary AnemiasInflammatory Conditions
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      RECRUITING
      Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
      Description

      The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.

      Conditions
      Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)Hereditary AnemiasInflammatory ConditionsSystemic Juvenile Idiopathic Arthritis (sJIA)
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