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 Sickle Cell Disease (DREPAGLOBE)

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

6

Participants

Timeline

Start Date

November 12, 2019

Primary Completion Date

July 28, 2022

Study Completion Date

January 31, 2024

Conditions
Sickle Cell Disease
Interventions
GENETIC

DREPAGLOBE drug product

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

Trial Locations (1)

75015

Department of Biotherapy, Necker-Enfants Malades Hospital, Paris

All Listed Sponsors
lead

Assistance Publique - Hôpitaux de Paris

OTHER

NCT03964792 - 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 Sickle Cell Disease (DREPAGLOBE) | Biotech Hunter | Biotech Hunter