Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A
Status:
Active, not recruiting
Trial end date:
2022-04-01
Target enrollment:
Participant gender:
Summary
This is an open, Phase I / II clinical trial to evaluate the safety and efficacy of a
hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector
carrying the FANCA gene for patients with Fanconi Anemia of Subtype A .
CD34 + cells derived from bone marrow and / or mobilized peripheral blood (fresh and / or
cryopreserved) from patients with Fanconi subtype A (FA-A), will be transduced ex vivo with a
lentiviral vector carrying the gene FANCA (orphan drug) . After transduction the cells will
be inoculated in patients in order to restore their hematopoiesis with genetically corrected
stem cells.
Centro de Investigación en Red de Enfermedades Raras (CIBERER) Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) Hospital Vall d'Hebron Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz Universitat Autonoma de Barcelona