Gene Transfer for Patients With Sickle Cell Disease

NCTID	NCT02186418 (View at clinicaltrials.gov)
Description	The purpose of this Phase 1/2 study is to determine the feasibility and safety of stem cell collection and gamma-globin gene transfer, and success of gene correction in subjects with sickle cell disease (Show More)
Development Status	Inactive
Indication	Sickle Cell Disease
Disease Ontology Term	DOID:0081445
Compound Name	ARU-1801
Sponsor	Children's Hospital Medical Center, Cincinnati
Funder Type	Other
Recruitment Status	Terminated
Enrollment Count	7
Results Posted	Not Available

Target Gene/Variant	HBG(G16D)
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 (dose range: 1.4-7.1E6 cells/kg)
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Clinical development was discontinued in June 2022