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

Phase 2
35 Years
Open (Enrolling)
Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome, Immunologic Deficiency Syndromes, Langerhans-Cell Histiocytosis

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Trial Information

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

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.

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

- 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

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To demonstrate the safety and the ability to establish stable mixed chimerism (>10% donor cells at day 100) using a nonmyeloablative preparative regimen in a phase 2 pilot trial

Outcome Time Frame:

Day 100

Safety Issue:


Principal Investigator

Angela Smith, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Masonic Cancer Center, University of Minnesota


United States: Institutional Review Board

Study ID:




Start Date:

August 2002

Completion Date:

August 2017

Related Keywords:

  • Hemophagocytic Lymphohistiocytosis
  • X-Linked Lymphoproliferative Disorders
  • Chediak-Higashi Syndrome
  • Griscelli Syndrome
  • Immunologic Deficiency Syndromes
  • Langerhans-Cell Histiocytosis
  • Stem cell transplant
  • immunodeficiency
  • donor lymphocyte infusion
  • Chediak-Higashi Syndrome
  • Histiocytosis
  • Histiocytosis, Langerhans-Cell
  • Immunologic Deficiency Syndromes
  • Lymphohistiocytosis, Hemophagocytic
  • Lymphoproliferative Disorders



Masonic Cancer Center University of Minnesota Minneapolis, Minnesota  55455