Stem Cell Transplant for Immunologic or Histiocytic Disorders (NCT00176865) | Clinical Trial Compass
CompletedPhase 2
Stem Cell Transplant for Immunologic or Histiocytic Disorders
United States19 participantsStarted 2002-08
Plain-language summary
This study tests the clinical outcomes of a preparative regimen of fludarabine (FLU), anti-thymocyte globulin (ATG)/or Campath, and melphalan; followed by hematopoietic stem cell transplant, and a post transplant regimen of Cyclosporin A (CsA) in patients with immunologic or histiocytic disorders. The researchers hypothesize that this regimen will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease (GVHD).
Patients will be randomized biologically into one of 3 arms based upon donor availability: (a) human leukocyte antigen (HLA) genotypic matched sibling donor, (b) HLA phenotypic matched unrelated peripheral blood stem cell (PBSC) donor, (c) two HLA 0-2 antigen mismatched unrelated cord blood donors (double cord).
Who can participate
Age range35 Years
SexALL
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Inclusion Criteria:
Patients with immunodeficiencies or histiocytic disorders 0-35 years of age with an acceptable stem cell donor and disease characteristic defined by the following:
* Patients with histocytic disorders (hemophagocytic lymphohistiocytosis of any etiology and refractory Langerhans cell histiocytosis) who do not meet eligibility criteria for a myeloablative transplant procedure
* Patients with immunodeficiency disorders in whom residual immune function may not require a fully myeloablative preparative regimen or patient is ineligible for standard myeloablative preparative regimen (any form of severe combined immunodeficiency \[SCID\], or other immunodeficiency with T cell defect)
* Patients with immunodeficiency disorders that have had poor outcome with myeloablative stem cell transplants (including, but not limited to, common variable immunodeficiency \[CVID\], Wiskott Aldrich Syndrome \[WAS\] if \> 5 years of age, ataxia telangiectasia)
* Patients with immunodeficiencies or histocytic disorders that require a second stem cell transplant (SCT) for any reason
Exclusion Criteria:
* Karnofsky or Lansky performance score \<70
* Glomerular filtration rate (GFR)\<30% predicted
* Cardiac function \<50% normal by echocardiogram
* Serum creatinine \> 2x normal for age/weight
* Pregnant or lactating females
* Active serious infection that has not had an adequate course of therapy pre-SCT. Any patient with acquired immune deficiency syndrome (AIDS) or AIDS-related c…
What they're measuring
1
Number of Subjects With Mixed Chimerism
Timeframe: Day 100
Trial details
NCT IDNCT00176865
SponsorMasonic Cancer Center, University of Minnesota