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)

NCTID	NCT03964792 (View at clinicaltrials.gov)
Description	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) (Show More)
Indication	Sickle Cell Disease
Compound Name	DREPAGLOBE
Sponsor	Assistance Publique - Hôpitaux de Paris
Funder Type	Other
Status	Active not recruiting
Enrollment Count	6

Target Gene/Variant	HBB
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	LV
Editor Type
Dose 1	Transduced CD34+ cells (range: 6 - 8.1 x 10^6 cells/kg)
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	France

Has US IND	False
Recent Updates	Uses the same βAS3-globin as NCT02247843; vector performed poorly