Gene Therapy for Fanconi Anemia | Aplastic Anemia and MDS International Foundation

Clinical Trial: NCT01331018

Gene Therapy for Fanconi Anemia
For more details on this clinical trial, including contact information, please see this trial’s listing on


I. To determine the safety of lentiviral gene transfer for patients with Fanconi anemia complementation group A (FANCA).


I. 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.

II. To determine if the clinical grade transduction will result in phenotypic correction of gene modified cells by in vitro assays.

III. To determine if infusion of FANCA gene-modified cells will result in engraftment and improvement in blood counts in FA patients.


BONE MARROW HARVEST FOR CELL COLLECTION: Patients undergo bone marrow harvest for collection of stem/progenitor cells on day -1.

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.

Study Date: 
Wed, 03/16/2011 to Fri, 12/01/2017
Biological: Genetically Engineered Hematopoietic Stem Progenitor Cells Undergo infusion of genetically modified hematopoietic progenitor cell therapy Other Name: Genetically Engineered HSPCs Procedure: Harvest Procedure Undergo bone marrow harvest Other Name: Harvest Other: Laboratory Biomarker Analysis Correlative studies