Stem Cell Transplant for Bone Marrow Failure Syndromes
Bone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure Disorders
2 other identifiers
interventional
10
1 country
1
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 of donor cell engraftment for non-malignant congenital bone marrow failure disorders.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jun 2000
Longer than P75 for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 1, 2000
CompletedFirst Submitted
Initial submission to the registry
September 12, 2005
CompletedFirst Posted
Study publicly available on registry
September 15, 2005
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2009
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2009
CompletedResults Posted
Study results publicly available
August 6, 2009
CompletedDecember 28, 2017
December 1, 2017
8.8 years
September 12, 2005
June 18, 2009
December 3, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of Patients Alive (Survival) at 2 Years
Calculated from day 1 of transplant to last contact.
2 years
Secondary Outcomes (5)
Number of Patients Alive at Three Years (Survival)
3 years
Number of Patients With Succcessful Engraftment After Transplantation
42 Days
Number of Patients With Grade 2-4 Acute Graft Versus Host Disease
100 Days
Number of Patients With Chronic Graft Versus Host Disease
2 years
Number of Patients With Disease Recurrence
2 years
Study Arms (1)
Bone Marrow Failure Disorders
EXPERIMENTALPatients with Diamond-Blackfan Anemia, Kostmann's Neutropenia, Shwachman-Diamond Syndrome
Interventions
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.
fludarabine 175 mg/m\^2 (total) on Days -6 through -3.
Dose 500 cGy radiation therapy to specific areas of the body
Busulfan 8 mg/kg (total) on Days - 8 and -7 (orally or through the catheter),
anti-thymocyte globulin (ATG) 15 mg/kg on days -2 and -1 via catheter
Eligibility Criteria
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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
University of Minnesota Medical Center
Minneapolis, Minnesota, 55455, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Limitations and Caveats
Pharmacokinetic parameters in patients receiving 2 mg/kg/dose of busulfan twice daily was not performed. No data is available.
Results Point of Contact
- Title
- Paul Orchard, M.D.
- Organization
- Masonic Cancer Center, University of Minnesota
Study Officials
- PRINCIPAL INVESTIGATOR
Paul Orchard, MD
University of Minnesota Medical Center
Publication Agreements
- PI is Sponsor Employee
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 12, 2005
First Posted
September 15, 2005
Study Start
June 1, 2000
Primary Completion
March 1, 2009
Study Completion
March 1, 2009
Last Updated
December 28, 2017
Results First Posted
August 6, 2009
Record last verified: 2017-12