Fanconi's Anemia Clinical Trial
OBJECTIVES: I. Determine the safety of transferring the Fanconi anemia complementation group
C (FACC) gene to hematopoietic progenitors by retroviral mediated gene transfer in patients
with Fanconi's anemia, complementation group C.
II. Determine the extent of engraftment following this treatment regimen without prior
ablation of recipient marrow in these patients.
III. Determine the ability of this treatment regimen to correct the cell phenotype and
improve hematopoietic function in these patients.
PROTOCOL OUTLINE: Patients receive filgrastim (G-CSF) subcutaneously daily on days 0-6
followed by apheresis to collect peripheral blood stem cells (PBSC) on days 5-7. PBSCs are
processed in vitro for enrichment of CD34 cells and transduced with a Fanconi's anemia
complementation C (FACC) retroviral vector on days 5-10. Patients receive transduced PBSCs
IV over no more than 2 hours on days 8-10. PBSC infusions may be repeated no more than every
2 months for up to 4 courses total.
Patients are followed monthly for 3 months, every 3 months for 9 months, every 6 months for
the next year, and then yearly thereafter.
;
Primary Purpose: Treatment
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