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)
Development Status	Active
Indication	Sickle Cell Disease
Disease Ontology Term	DOID:0081445
Compound Name	DREPAGLOBE
Sponsor	Assistance Publique - Hôpitaux de Paris
Funder Type	Other
Recruitment Status	Active not recruiting
Enrollment Count	6
Results Posted	Not Available

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	none
Dose 1	Transduced CD34+ cells (range: 6 - 8.1E6 cells/kg)
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	France

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