CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease

NCTID	NCT04091737 (View at clinicaltrials.gov)
Description	This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor cells by apheresis after mobilization with plerixafor, reduced intensity conditioning with melphalan, and administration of CSL200. (Show More)
Development Status	Inactive
Indication	Sickle Cell Disease
Disease Ontology Term	DOID:0081445
Compound Name	CSL200
Compound Description	human gamma-globinG16D/short-hairpin RNA734-transduced CD34-positive cells
Sponsor	CSL Behring
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	1
Results Posted	Not Available

Target Gene/Variant	HBG1/HBG2
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 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations	Orphan Drug Designation
Recent Updates	Trial was terminated due to unanticipated delays, only enrolled 1 patient

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