Safety and Efficacy of Gene Therapy of the Sickle Cell Disease by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains CD34+ Cells Transduced ex Vivo With the GLOBE1 Lentiviral Vector Expressing the βAS3 Globin Gene in Patients With

Clinical Trial
Study Details
Enrollment
6
Interventions
Description

Each patient will receive a single IV infusion of DREPAGLOBE drug product

Name
DREPAGLOBE drug product
Type
Genetic
Phase
Phase 1
Phase 2
Status
COMPLETED
Study ID #
NCT03964792
Study Type
Interventional
Summary

The purpose of this study is to evaluate the Safety and Efficacy of Gene Therapy of the Sickle Cell disease by Transplantation of an Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced ex vivo with the GLOBE1 lentiviral vector expressing the βAS3 globin gene (GLOBE1 βAS3 Modified Autologous CD34+ Cells) in Patients with Sickle Cell Disease (SCD)

Study Overview
Conditions
Sickle Cell Disease
Contacts and Locations
Locations
Facility

Department of Biotherapy, Necker-Enfants Malades Hospital
75015 Paris
France

GeoPoint
48.85341, 2.3488