Gene Therapy for Fanconi Anemia

Fanconi Anemia Clinical Trial

Official title: Gene Transfer for Patients With Fanconi Anemia Complementation Group A (FANCA)

Status Terminated

Clinical Trial Summary

This clinical trial will access the toxicity and efficacy of infusion of gene modified cells for patients with Fanconi anemia (FA). Infusion of autologous patient blood stem cells that have been corrected in the laboratory by introduction of the normal gene may improve blood counts in patients with FA.

Clinical Trial Description

OUTLINE:

STEM CELL MOBILIZATION FOR CELL COLLECTION: Patients receive filgrastim subcutaneously (SC) twice daily (BID) for up to 6 days (on days 1-6 of mobilization). Patients receive plerixafor SC once daily (QD) on days 4-6 of mobilization. Peripheral blood stem cell (PBSC) count will be checked daily starting on day 4 of mobilization. Patients who have a PBSC count of >= 5 CD34+ cells/mcL will undergo up to 2 apheresis collections on consecutive days.

BONE MARROW HARVEST FOR CELL COLLECTION: Patients with inadequate PBSC counts undergo bone marrow harvest for collection of stem/progenitor cells.

REINFUSION: Patients receive methylprednisolone intravenously (IV) or prednisone orally (PO) on days -1 to 7 followed by a rapid taper over approximately 1 week and undergo reinfusion of genetically modified hematopoietic stem/progenitor cells on day 0.

After completion of study treatment, patients are followed up periodically for 15 years. ;

Related Conditions & MeSH terms

• Anemia
• Fanconi Anemia
• Fanconi Syndrome

• NCT number: NCT01331018
• Study type: Interventional
• Source: Fred Hutchinson Cancer Center
• Status: Terminated
• Phase: Phase 1
• Start date: February 22, 2012
• Completion date: February 15, 2024