Stem Cell Transplant for Bone Marrow Failure Syndromes
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|ClinicalTrials.gov Identifier: NCT00176878|
Recruitment Status : Completed
First Posted : September 15, 2005
Results First Posted : August 6, 2009
Last Update Posted : December 28, 2017
|Condition or disease||Intervention/treatment||Phase|
|Diamond-Blackfan Anemia Kostmann's Neutropenia Shwachman-Diamond Syndrome||Procedure: Stem cell transplant Drug: Fludarabine monophosphate Procedure: Total lymphoid irradiation Drug: Busulfan Biological: anti-thymocyte globulin||Phase 2 Phase 3|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||10 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Bone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure Disorders|
|Study Start Date :||June 2000|
|Actual Primary Completion Date :||March 2009|
|Actual Study Completion Date :||March 2009|
Experimental: Bone Marrow Failure Disorders
Patients with Diamond-Blackfan Anemia, Kostmann's Neutropenia, Shwachman-Diamond Syndrome
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.
Other Name: BMTDrug: Fludarabine monophosphate
fludarabine 175 mg/m^2 (total) on Days -6 through -3.
Other Name: FludaraProcedure: Total lymphoid irradiation
Dose 500 cGy radiation therapy to specific areas of the body
Other Name: TLIDrug: Busulfan
Busulfan 8 mg/kg (total) on Days - 8 and -7 (orally or through the catheter),
Other Name: BusulfexBiological: anti-thymocyte globulin
anti-thymocyte globulin (ATG) 15 mg/kg on days -2 and -1 via catheter
Other Name: ATG
- Number of Patients Alive (Survival) at 2 Years [ Time Frame: 2 years ]Calculated from day 1 of transplant to last contact.
- Number of Patients Alive at Three Years (Survival) [ Time Frame: 3 years ]Number of subjects who survived 3 years post-transplant.
- Number of Patients With Succcessful Engraftment After Transplantation [ Time Frame: 42 Days ]Number of patients who received non-genotypic identical marrow or cord blood cells using a "non-myeloablative" preparative regimen and exhibited engraftment at Day 42.
- Number of Patients With Grade 2-4 Acute Graft Versus Host Disease [ Time Frame: 100 Days ]Number of patients with Grade 2, 3 and 4 Acute (normally observed within the first 100 days) Graft Versus Host Disease. Acute GVHD is staged as follows: overall grade (skin-liver-gut) with each organ staged individually from a low of 1 to a high of 4. Patients with grade IV GVHD usually have a poor prognosis. Grade 2 = moderate, Grade 3 = severe, Grade 4 = life threatening.
- Number of Patients With Chronic Graft Versus Host Disease [ Time Frame: 2 years ]Number of patients who exhibited chronic (normally occurs after 100 days) Graft Versus Host Disease at 2 years post transplant. Chronic graft-versus-host-disease, over its long-term course, can also cause damage to the connective tissue and exocrine glands.
- Number of Patients With Disease Recurrence [ Time Frame: 2 years ]Number of patients who exhibited disease recurrence at 2 years.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00176878
|United States, Minnesota|
|University of Minnesota Medical Center|
|Minneapolis, Minnesota, United States, 55455|
|Principal Investigator:||Paul Orchard, MD||University of Minnesota Medical Center|