Bone Marrow Cell Gene Transfer in Individuals With Fanconi Anemia

NCTID	NCT00272857 (View at clinicaltrials.gov)
Description	Fanconi anemia (FA) is a disease that affects an individual's bone marrow. It is caused by a defective gene in the bone marrow cells that produce various types of blood cells. Individuals with FA may experience fatigue, bleeding, and increased infections. The purpose of this study is to evaluate the safety and effectiveness of a gene transfer procedure in generating new, healthy cells in individuals with FA. (Show More)
Development Status	Inactive
Indication	Fanconi Anemia
Disease Ontology Term	DOID:0111095
Compound Name	MSCV-FANCA
Sponsor	Boston Children's Hospital
Funder Type	Other
Recruitment Status	Completed
Enrollment Count	3
Results Posted	Not Available

Target Gene/Variant	FANCA
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	MSCV
Editor Type
Dose 1	3.5-4.5E5 CD34+ cells/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Clinical study did not demonstrate long-term engraftment or selective advantage