Hematopoietic Stem Cell Transplant for Fanconi Anemia
| Status: | Completed |
|---|---|
| Conditions: | Anemia |
| Therapuetic Areas: | Hematology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 4/17/2018 |
| Start Date: | April 2009 |
| End Date: | August 30, 2016 |
A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
The trial proposed is a single arm phase II treatment protocol designed to examine
engraftment, toxicity, graft-versus-host disease, and ultimate disease-free survival
following a novel cytoreductive regimen including busulfan, cyclophosphamide and fludarabine
and anti-thymocyte globulin (ATG- a non-chemotherapy drug whose role is to kill your immune
system) for the treatment of patients with Fanconi anemia who have severe aplastic anemia
(SAA), or myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML), lacking
HLA-genotypically identical donors using stem cell transplants derived from (1)
HLA-compatible unrelated donors or (2) HLA haplotype-mismatched related donors.
engraftment, toxicity, graft-versus-host disease, and ultimate disease-free survival
following a novel cytoreductive regimen including busulfan, cyclophosphamide and fludarabine
and anti-thymocyte globulin (ATG- a non-chemotherapy drug whose role is to kill your immune
system) for the treatment of patients with Fanconi anemia who have severe aplastic anemia
(SAA), or myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML), lacking
HLA-genotypically identical donors using stem cell transplants derived from (1)
HLA-compatible unrelated donors or (2) HLA haplotype-mismatched related donors.
We are currently recruiting patients.
Inclusion Criteria:
- Fanconi Anemia (confirmed by mitomycin C or DEB chromosomal breakage testing and one
of the following hematological diagnoses: Severe Aplastic Anemia, Myelodysplastic
Syndrome, Acute Myelogenous Leukemia
- Karnofsky or Lansy performance scale > or = to 70%.
- Must have adequate cardiac, hepatic, renal and pulmonary function.
- Must have 7/8 or 8/8 available unrelated donor.
Exclusion Criteria:
- Pregnant or breastfeeding.
- Active CNS leukemic involvement
- Active uncontrolled viral, bacterial or fungal infection
- Positive for HIV.
We found this trial at
1
site
Medical College of Wisconsin The Medical College (MCW) of Wisconsin is a major national research...
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