Stem Cell Transplant for Immunologic or Histiocytic Disorders

• Register: ClinicalTrials.gov
• 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
• URL: https://clinicaltrials.gov/show/NCT00176865
• 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

Contacts

• 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

Hemophagocytic Lymphohistiocytosis

Griscelli Syndrome

X-Linked Lymphoproliferative Disorders

Immunologic Deficiency Syndromes

Langerhans-Cell Histiocytosis

Chediak-Higashi Syndrome

Intervention(s)

Drug: Cyclosporin A

Drug: Melphalan

Drug: Campath 1H

Drug: Mycophenolate mofetil

Procedure: Stem Cell Transplant

Drug: Anti-thymocyte globulin (ATG)

Drug: Intravenous immunoglobulin (IVIG)

Drug: Fludarabine

Primary Outcome(s)

Number of Subjects With Mixed Chimerism [Time Frame: Day 100]

Secondary Outcome(s)

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]

Percentage of Donor Chimerism at 100 Days [Time Frame: Day 100]

Incidence of Chronic Graft Versus Host Disease (cGVHD) [Time Frame: 6 months and 1 year]

Percentage of Donor Chimerism at 180 Days [Time Frame: Day 180]

Compare Quality of Life (QOL) [Time Frame: Pretransplant, 1 year, 2 years and 5 years]

Number of Subjects Alive at 100 Days [Time Frame: Day 100]

Percentage of Donor Chimerism at 365 Days [Time Frame: Day 365]

Number of Subjects Alive at One Year [Time Frame: Day 365]

Secondary ID(s)

MT2002-12

0207M29448

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 14/04/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00176865