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

Fanconi Anemia Complementation Group A Clinical Trial

Official title: A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients With Fanconi Anemia Subtype A

Status Active, not recruiting

Clinical Trial 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.

Clinical Trial Description

This is a pediatric open-label Phase II clinical trial to assess the efficacy of a hematopoietic gene therapy consisting of autologous CD34+ enriched cells transduced with a lentiviral vector carrying the FANCA gene in pediatric subjects with FA-A.

Enriched CD34+ hematopoietic stem cells will be transduced ex vivo with the therapeutic lentiviral vector and infused via intravenous infusion following transduction without any prior conditioning. ;

Related Conditions & MeSH terms

• Anemia
• Fanconi Anemia
• Fanconi Anemia Complementation Group A
• Fanconi Syndrome

• NCT number: NCT04069533
• Study type: Interventional
• Source: Rocket Pharmaceuticals Inc.
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: November 28, 2019
• Completion date: February 2025