Stem Cell Transplant for Bone Marrow Failure Syndromes

Completed · Phase 2/Phase 3

Conditions studied: Diamond-Blackfan Anemia, Kostmann's Neutropenia, Shwachman-Diamond Syndrome

In brief

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.

Key facts

Study ID

NCT00176878

Run by

Masonic Cancer Center, University of Minnesota

People needed

10

Starts

2000-06-01

Expected to finish

2009-03-01

Last updated by the study team

2017-12-28

Who can join

Age: any, up to 35. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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).

You may not qualify if…

• 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

Where it is running

• University of Minnesota Medical Center — Minneapolis, Minnesota, United States

Full record on ClinicalTrials.gov

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