Status:
COMPLETED
Stem Cell Transplant for Bone Marrow Failure Syndromes
Lead Sponsor:
Masonic Cancer Center, University of Minnesota
Conditions:
Diamond-Blackfan Anemia
Kostmann's Neutropenia
Eligibility:
All Genders
Up to 35 years
Phase:
PHASE2
PHASE3
Brief 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 ...
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 an...
Eligibility Criteria
Inclusion
- Patients eligible for transplantation under this protocol will be \<35 years of age, and will be diagnosed with:
- a bone marrow failure syndrome unresponsive to available therapy, including but not limited to Diamond-Blackfan anemia, Shwachman Diamond syndrome or Kostmann's neutropenia but exclusive of aplastic anemia.
- Diamond Blackfan Anemia:
- Patients must show evidence of steroid resistance requiring equivalent of \>6 transfusions yearly despite steroid therapy.
- Evidence of developing aplasia or myelodysplasia will also be criteria for transplantation.
- Kostmann's Neutropenia, Shwachman-Diamond syndrome:
- Patients must have been previously diagnosed as having a clinical picture characteristic of Shwachman-Diamond syndrome (exocrine pancreatic insufficiency, growth retardation, metaphyseal dysostosis, neutropenia), or must have a bone marrow aspirate consistent with Kostmann's neutropenia, with no evidence of acute leukemia.
- Patients must have failed therapy with granulocyte-colony stimulating factor (G-CSF), as determined by an inability to maintain an absolute neutrophil count (ANC) \>750 cells/ml(3), or manifesting recurrent infections despite G-CSF administration resulting in life threatening infections or repeated hospitalizations (\<4 /year).
Exclusion
- Patients \>35 years of age
- Karnofsky score \<70%
- Hepatic dysfunction as determined by bilirubin \>3.0, ALT \>150, or active hepatitis
- Pulmonary function tests with forced volume vital capacity (FVC) and forced expiratory volume (FEV) \<70%; O2 saturation \<94%
- Renal dysfunction with glomerular filtration rate (GFR) \<30% of predicted.
- Cardiac compromise, with left ejection fraction \<45%.
- Severe, stable neurologic impairment.
- Human immunodeficiency virus (HIV) positivity.
- Pregnant or lactating females
Key Trial Info
Start Date :
June 1 2000
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2009
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT00176878
Start Date
June 1 2000
End Date
March 1 2009
Last Update
December 28 2017
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
University of Minnesota Medical Center
Minneapolis, Minnesota, United States, 55455