Gene Therapy Using a Self-inactivating Lentiviral Vector to Treat Fanconi Anemia in Children and Young Adults

What this Trial Is About

Fanconi anemia is a rare inherited disease that affects bone marrow, leading to low counts of red blood cells, white blood cells, and platelets. It often presents with physical defects and is diagnosed mainly in childhood with a high fatality rate. This trial evaluates a gene therapy using a self-inactivating lentiviral vector to correct the defective gene in patients with Fanconi anemia, aiming to assess both safety and efficacy. The study involves the infusion of gene-modified autologous hematopoietic and mesenchymal stem cells carrying the FANCA gene. Dosing includes 5x106 to 1x107 modified cells per kilogram of body weight, with the possibility of additional infusions depending on individual circumstances. This gene transfer protocol is conducted ex vivo, meaning cells are modified outside the body before being infused back into the patient. Participants will be closely monitored for safety using standard criteria over six months, with additional assessments of treatment response and quality of life up to one year. The study includes comprehensive evaluations of immune reconstitution, gene correction efficiency, and long-term disease symptom improvement. The total participation may extend through the study completion in 2030, with regular assessments to track outcomes and adverse events.

FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector