NCT00176878

Stem Cell Transplant for Bone Marrow Failure Syndromes

Bone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure Disorders

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 35 Years
• Healthy volunteers: Not accepted

Summary

The researchers hypothesize that it will be possible to perform unrelated bone marrow or cord blood transplants in a safer manner by using less intensive therapy yet still achieve an acceptable level of donor cell engraftment for non-malignant congenital bone marrow failure disorders.

Detailed description

Prior to transplantation, subjects will receive the drugs busulfan (orally or through the catheter), as well as fludarabine and anti-thymocyte globulin (ATG) via the catheter. Busulfan, fludarabine and ATG will be given with Total Lymphoid Irradiation (TLI) to help the new donor bone marrow take and grow after transplantation. Those patients receiving donor marrow will have the T cells (a type of white blood cell in the donor marrow) removed to lower the risk that the new marrow will react to their body, a condition called Graft-Versus-Host-Disease (GVHD). After bone marrow transplantation, subjects will receive drugs to help prevent GVHD, including cyclosporin and mycophenolate mofetil (MMF). Blood samples are taken at day 28, day 60, day 100, 1 year and as required by medical status yearly for five years after transplant to evaluate how well the new marrow is growing. A bone marrow biopsy is required at day 21, at day 100 and 1 year.

Conditions

• Diamond-Blackfan Anemia
• Kostmann's Neutropenia
• Shwachman-Diamond Syndrome

Interventions

Type	Name	Description
PROCEDURE	Stem cell transplant	Stem cell transplant on Day 0 - healthy marrow from an unrelated individual. A minimum of 1.0 x 10\^9/kg nucleated cells/kg ideal body weight will be collected with a goal of 2.0 x 10\^9/kg.
DRUG	Fludarabine monophosphate	fludarabine 175 mg/m\^2 (total) on Days -6 through -3.
PROCEDURE	Total lymphoid irradiation	Dose 500 cGy radiation therapy to specific areas of the body
DRUG	Busulfan	Busulfan 8 mg/kg (total) on Days - 8 and -7 (orally or through the catheter),
BIOLOGICAL	anti-thymocyte globulin	anti-thymocyte globulin (ATG) 15 mg/kg on days -2 and -1 via catheter

Timeline

• Start date: 2000-06-01
• Primary completion: 2009-03-01
• Completion: 2009-03-01
• First posted: 2005-09-15
• Last updated: 2017-12-28
• Results posted: 2009-08-06

Locations

1 site across 1 country: United States