NCT01331018

Gene Therapy for Fanconi Anemia

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

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Fred Hutchinson Cancer Center · Academic / Other
• Sex: All
• Age: 4 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Fanconi Anemia

Interventions

Type	Name	Description
PROCEDURE	Bone Marrow Aspiration	Undergo bone marrow harvest
BIOLOGICAL	Filgrastim	Given SC
BIOLOGICAL	Genetically Engineered Hematopoietic Stem Progenitor Cells	Undergo infusion of genetically modified hematopoietic progenitor cell therapy
OTHER	Laboratory Biomarker Analysis	Correlative studies
PROCEDURE	Leukapheresis	Undergo leukapheresis
DRUG	Methylprednisolone	Given IV
DRUG	Plerixafor	Given SC
DRUG	Prednisone	Given PO

Timeline

• Start date: 2012-02-22
• Primary completion: 2024-02-15
• Completion: 2024-02-15
• First posted: 2011-04-07
• Last updated: 2024-05-17

Locations

1 site across 1 country: United States