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

PHASE2Active, not recruitingINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

November 28, 2019

Primary Completion Date

February 28, 2025

Study Completion Date

February 28, 2025

Conditions

Fanconi Anemia Complementation Group A

Interventions

BIOLOGICAL

RP-L102

CD34+ enriched cells from subjects with Fanconi anemia subtype A transduced ex vivo with lentiviral vector carrying the FANCA gene

Trial Locations (2)

Hospital Infantil Universitario Niño Jesús (HIUNJ), Madrid

University College London Great Ormond Street Institute of Child Health (GOSH), London

All Listed Sponsors

lead

Rocket Pharmaceuticals Inc.

INDUSTRY

NCT04069533 - Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A | Biotech Hunter | Biotech Hunter