Gene Transfer for Patients With Fanconi Anemia Complementation Group A (FANCA)
I. To determine the safety of lentiviral gene transfer for patients with Fanconi anemia
complementation group A (FANCA).
I. To determine the feasibility of collection of the number of hematopoietic progenitor
cells from Fanconi anemia complementation group A patients that would be expected to have
potential for therapeutic benefit after transduction and infusion. The mobilization will be
performed with G-CSF (filgrastim) or with a combination of G-CSF and plerixafor for patients
aged 18 and older. Additional bone marrow may be collected if insufficient cells are
collected after mobilization and apheresis.
II. To determine the transduction efficiency for human FA patient hematopoietic progenitor
cells transduced with a clinical grade lentiviral vector encoding the gene for Fanconi
anemia complementation group A.
III. To determine if the clinical grade transduction will result in phenotypic correction of
gene modified cells by in vitro assays.
IV. To determine if infusion of FANCA gene-modified cells will result in engraftment and
improvement in blood counts in FA patients.
STEM CELL MOBILIZATION: Patients receive filgrastim subcutaneously (SC) twice daily on days
-5 to -1. Patients 18 years of age or older also receive plerixafor SC on days -2 and -1.
CELL COLLECTION: Patients undergo apheresis for collection of stem/progenitor cells on days
-2 and -1. Patients with insufficient cell mobilization (< 1 x 10^6 CD34+ cells/kg) undergo
bone marrow harvest.
REINFUSION: Patients undergo reinfusion of genetically modified hematopoietic progenitor
cells on day 0.
After completion of study treatment, patients are followed up periodically for 15 years.
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Toxicity and safety of lentiviral gene transfer
Adverse events will be graded by Common Terminology Criteria for Adverse Events (CTCAE), Version 4.
Up to 15 years
Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
United States: Food and Drug Administration
|Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium||Seattle, Washington 98109|