Fanconi Anemia Transplant Lacking Genotypically Identical Donor



Status:Not yet recruiting
Conditions:Anemia
Therapuetic Areas:Hematology
Healthy:No
Age Range:Any
Updated:4/2/2016
Start Date:April 2009

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Multicenter Phase II Trial-Hematopoietic Stem Cell Transplantation for Treatment of Patients With FA Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

A research study for patients with Fanconi Anemia whose bone marrow has changed and now
failed, giving rise to a pre-leukemia or leukemia. This study is a Phase II clinical trial
in which patients will undergo allogenic transplant of stem cells, meaning they will receive
bone marrow cells from a healthy donor. The purpose of this study is to see if transplant
course of treatment will lower the risk of graft vs. host disease.


Inclusion Criteria:

- Diagnosis: Patients must have a diagnosis of Fanconi anemia (confirmed by mitomycin C
or diepoxybutane [DEB] chromosomal breakage testing at an approved laboratory)

- Hematologic Diagnosis and Status: Patients must have one of the following hematologic
diagnoses:

1. Severe Aplastic Anemia (SAA), or Severe Isolated Single lineage

Cytopenia with bone marrow cellularity of < 25% AND at least one of the
following features:

- Platelet count < 20 x 109/L or platelet transfusion dependence

- ANC < 1000 x 109/L

- Hgb < 8 gm/dl or red cell transfusion dependence

2. Myelodysplatic Syndrome (MDS) (Appendix 1: MDS Classification). MDS at any
stage, based on either one of the following classifications:

- WHO Classification

- Refractory anemia and transfusion dependence

- Any of other stages

- IPSS Classification

- Low risk (score 0) and transfusion dependence

- Any other risk groups Score > 0.5

3. Acute Myelogenous Leukemia: Patients with acute leukemia are included in this
trial in remission, refractory or relapsed disease

Exclusion Criteria:

- Active CNS leukemic involvement

- Female patients who are pregnant or breast-feeding

- Active uncontrolled viral, bacterial or fungal infection

- Patient seropositive for HIV-I/II; HTLV -I/II
We found this trial at
1
site
450 Brookline Ave
Boston, Massachusetts 2215
617-632-3000
Dana-Farber Cancer Institute Since it’s founding in 1947, Dana-Farber has been committed to providing adults...
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Boston, MA
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