Stem Cell Transplant for Immunologic or Histiocytic Disorders

Brief Title

Stem Cell Transplant for Immunologic or Histiocytic Disorders

Official Title

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Immunologic or Histiocytic Disorders Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism

Brief 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).

Detailed Description

      Prior to transplantation, subjects will receive Melphalan, Fludarabine and Anti-Thymocyte
      Globulin (ATG) or Campath. These three drugs are being given to subjects to help the new stem
      cells take and grow. On the day of transplantation, subjects will receive stem cells
      transfused via intravenous (IV) catheter.

      After stem cell transplantation, subjects will be given Cyclosporin A (CsA) and mycophenolate
      mofetil (MMF) to reduce the risk of graft-versus-host disease, the complication that occurs
      when the donor's stem cells react against the patient.

Study Phase

Phase 2

Study Type


Primary Outcome

Number of Subjects With Mixed Chimerism

Secondary Outcome

 Percentage of Donor Chimerism at 100 Days


Hemophagocytic Lymphohistiocytosis


Stem Cell Transplant

Study Arms / Comparison Groups

 Arm 1 - Matched sibling donor
Description:  Stem Cell Transplant: human leukocyte antigen (HLA) genotypic matched sibling donor and pre-treatment with fludarabine, melphalan, anti-thymocyte globulin or Campath 1H and post-treatment with Cyclosporin A, mycophenolate mofetil and Intravenous immunoglobulin (IVIG)


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

August 2002

Completion Date

August 2014

Primary Completion Date

August 2012

Eligibility Criteria

        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 complex (ARC)
             or human immunodeficiency virus (HIV) seropositivity




N/A - 35 Years

Accepts Healthy Volunteers



Angela Smith, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Secondary IDs


Responsible Party


Study Sponsor

Masonic Cancer Center, University of Minnesota

Study Sponsor

Angela Smith, MD, Principal Investigator, Masonic Cancer Center, University of Minnesota

Verification Date

December 2017