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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
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.
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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.