Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A

Summary

This is an open-label Phase II clinical trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for pediatric patients with Fanconi Anemia, subtype A (FA-A). Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the FANCA gene. After transduction, the corrected stem cells will be infused intravenously back to the patient with the goal of preventing bone marrow failure.

Timeline

Start

2019-11-28

Primary completion

2025-02

Completion

2025-02

Drugs

Indications