Allogeneic Hematopoietic Stem Cell Transplant for Patients With Immunologic or Histiocytic Disorders Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism
N/A
35 Years
Both
Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome, Immunologic Deficiency Syndromes, Langerhans-Cell Histiocytosis
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
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
Type of Study:
Interventional
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:
Yes
Principal Investigator
Angela Smith, MD
Investigator Role:
Principal Investigator
Investigator Affiliation:
Masonic Cancer Center, University of Minnesota
Authority:
United States: Institutional Review Board
Study ID:
MT2002-12
NCT ID:
NCT00176865
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
Name | Location |
|---|---|
| Masonic Cancer Center University of Minnesota | Minneapolis, Minnesota 55455 |
