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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Last refreshed on: 16 December 2017
Main ID:  NCT00176865
Date of registration: 12/09/2005
Prospective Registration: No
Primary sponsor: Masonic Cancer Center, University of Minnesota
Public title: Stem Cell Transplant for Immunologic or Histiocytic Disorders
Scientific title: Allogeneic Hematopoietic Stem Cell Transplant for Patients With Immunologic or Histiocytic Disorders Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism
Date of first enrolment: August 2002
Target sample size: 19
Recruitment status: Completed
Study type:  Interventional
Study design:  Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 2
Countries of recruitment
United States
Name:     Angela Smith, MD
Affiliation:  Masonic Cancer Center, University of Minnesota
Key inclusion & exclusion 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

Age minimum: N/A
Age maximum: 35 Years
Gender: All
Health Condition(s) or Problem(s) studied
Chediak-Higashi Syndrome
Griscelli Syndrome
Hemophagocytic Lymphohistiocytosis
Immunologic Deficiency Syndromes
Langerhans-Cell Histiocytosis
X-Linked Lymphoproliferative Disorders
Drug: Anti-thymocyte globulin (ATG)
Drug: Campath 1H
Drug: Cyclosporin A
Drug: Fludarabine
Drug: Intravenous immunoglobulin (IVIG)
Drug: Melphalan
Drug: Mycophenolate mofetil
Procedure: Stem Cell Transplant
Primary Outcome(s)
Number of Subjects With Mixed Chimerism [Time Frame: Day 100]
Secondary Outcome(s)
Compare Quality of Life (QOL) [Time Frame: Pretransplant, 1 year, 2 years and 5 years]
Incidence of Chronic Graft Versus Host Disease (cGVHD) [Time Frame: 6 months and 1 year]
Incidence of Grade 2-4 Acute Graft Versus Host Disease (aGVHD) [Time Frame: Day 100]
Incidence of Grade 3-4 Acute Graft Versus Host Disease (aGVHD) [Time Frame: Day 100]
Number of Subjects Alive at 100 Days [Time Frame: Day 100]
Number of Subjects Alive at One Year [Time Frame: Day 365]
Percentage of Donor Chimerism at 100 Days [Time Frame: Day 100]
Percentage of Donor Chimerism at 180 Days [Time Frame: Day 180]
Percentage of Donor Chimerism at 365 Days [Time Frame: Day 365]
Secondary ID(s)
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results available: Yes
Date Posted: 14/04/2017
Date Completed:
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