NCT00176826

T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders

In-vivo T-cell Depletion and Hematopoietic Stem Cell Transplantation for Life-Threatening Immune Deficiencies and Histiocytic Disorders

• Status: Terminated
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 22 (actual)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 55 Years
• Healthy volunteers: Not accepted

Summary

The hypothesis is to determine if a preparative regimen of busulfan, cyclophosphamide, and antithymocyte globulin (ATG) plus allogeneic stem cell transplantation will be effective in the treatment of immune deficiencies and histiocytic disorders.

Detailed description

Subjects will begin chemotherapy as a preparative regimen, which is intended to completely eliminate their defective immune system and bone marrow. The preparative regimen consists of the chemotherapy drugs (busulfan, cyclophosphamide, and antithymocyte globulin (ATG)). Transplantation: subjects will then have a source of blood stem cells (bone marrow) from their donor administered into their catheter. Medication will be given to help prevent Graft-Versus Host Disease (GVHD). The ATG will help to deplete the donor stem cells of the type of cells that can cause GVHD and will also help to promote engraftment of the new stem cells. Recovery Phase: The second phase of treatment consists of a period after transplantation during which we wait for the return of bone marrow function. This usually takes two to four weeks. Subjects will be given a blood cell growth factor, G-CSF, to help speed recovery of the white blood cells and potentially decrease the risk of infection and decrease the time until the bone marrow recovers.

Conditions

• Hemophagocytic Lymphohistiocytosis
• X-Linked Lymphoproliferative Disorders
• Chediak-Higashi Syndrome
• Griscelli Syndrome
• Immunologic Diseases
• Langerhans-Cell Histiocytosis
• Hematologic Diseases

Interventions

Type	Name	Description
PROCEDURE	Stem Cell Transplant	Infusion of hematopoietic stem cells (bone marrow, cord blood, peripheral blood stem cells) following myeloablative conditioning regimen.
DRUG	Myeloablative conditioning regimen	Busulfan intravenously for 4 days followed by cyclophosphamide intravenously for 4 days. Rabbit ATG is given intravenously for 4 doses pre-transplant.

Timeline

• Start date: 2000-09-01
• Primary completion: 2012-08-01
• Completion: 2015-08-01
• First posted: 2005-09-15
• Last updated: 2018-01-23
• Results posted: 2014-04-28

Locations

1 site across 1 country: United States