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Clinical Trial Report

Gene Therapy Trial Report

Summary

CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease


NCTID NCT04091737 (View at clinicaltrials.gov)
Description
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

Therapy Information


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

Study Record Dates


Current Stage Phase1
Submit Date 2019-09-13
Completion Date 2021-05-05
Last Update 2021-06-18

Participation Criteria


Eligible Age 18 Years - 45 Years
Standard Ages Adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


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

Resources/Links


Resources/Links

No External Links Available.