Stem Cell Transplant for Immunologic or Histiocytic Disorders

Completed · Phase 2

Conditions studied: Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome, Immunologic Deficiency Syndromes, Langerhans-Cell Histiocytosis

In brief

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).

Key facts

Study ID

NCT00176865

Run by

Masonic Cancer Center, University of Minnesota

People needed

19

Starts

2002-08-01

Expected to finish

2014-08-01

Last updated by the study team

2017-12-28

Who can join

Age: any, up to 35. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Masonic Cancer Center University of Minnesota — Minneapolis, Minnesota, United States

Full record on ClinicalTrials.gov

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