Hematopoietic Stem Cell Transplant for Fanconi Anemia
This trial is active, not recruiting.
|Sponsor||Medical College of Wisconsin|
|Collaborator||Memorial Sloan Kettering Cancer Center|
|Start date||April 2009|
|End date||May 2018|
|Trial size||25 participants|
|Trial identifier||NCT01071239, FA 08/89|
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.
|Endpoint classification||safety/efficacy study|
|Intervention model||single group assignment|
Bone Marrow processing using the CliniMACs device
To measure the incidence and quality of engraftment and hematopoietic reconstitution.
time frame: 1, 3, 6 and 12 months post transplant date
The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD
time frame: weekly for the first 30 days and then 3, 6, and 12 months post transplant date
Male or female participants of any age.
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.
|Official title||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|
|Principal investigator||David A Margolis, MD|
|Description||We are currently recruiting patients.|
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