Phase I Pilot Study of CD34 Enriched, Fanconi's Anemia Complementation Group C Gene Transduced Autologous Peripheral Blood Stem Cell Transplantation in Patients With Fanconi's Anemia
Recruitment status was Active, not recruiting
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.
Genetic: Autologous stem cells transduced with FACC retroviral vector
Procedure: Autologous Stem Cell Transplantation
|Study Design:||Primary Purpose: Treatment|
|Study Start Date:||March 2000|
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.