Multicenter Transplant Study for Fanconi Anemia



Status:Completed
Conditions:Anemia
Therapuetic Areas:Hematology
Healthy:No
Age Range:Any
Updated:6/30/2016
Start Date:October 2009
End Date:July 2015

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A Multicenter 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 multicenter treatment protocol designed to examine transplant
related events in patients with Fanconi anemia who lack matched sib donors have severe
aplastic anemia (SAA), or myelodysplastic syndrome(MDS) or acute myelogenous leukemia (AML).

The trial proposed is a single arm phase II multicenter 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 ATG (Anti-thymocyte globulin)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 (HLA = human leukocyte antigen) identical donors using stem
cell transplants derived from HLA-compatible unrelated donors or HLA haplotype-mismatched
related donors using Miltenyi's CliniMACS.

Inclusion Criteria:

- Confirmed Diagnosis of Fanconi Anemia

- One of the following:Severe Aplastic Anemia or Severe Single Lineage Cytopenia;
Myelodysplastic Syndrome; Acute Myelogenous Leukemia

- Unrelated and Related Donors

- Adequate Physical Function (Cardiac, Hepatic, Renal, Pulmonary)

- Available for Long-Term Follow Up

- Performance status >= 70%

Exclusion Criteria:

- Co-existing medical problems that increase the risk of transplant

- Active CNS (central nervous system) leukemic involvement

- Pregnant or Breastfeeding (Females)

- Active, Uncontrolled Infection

- HIV/HTLV (Human T-lymphotropic virus)Positive
We found this trial at
1
site
3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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Cincinnati, OH
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