Ignite Creation Date:
2024-05-06 @ 1:42 PM
Last Modification Date:
2024-10-26 @ 1:18 PM
Study NCT ID:
NCT04091737
Status:
TERMINATED
Last Update Posted:
2021-06-18
First Post:
2019-09-13
Brief Title:
CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease
Sponsor:
CSL Behring
Organization:
CSL Behring
Study Overview
Official Title:
A Phase 1 Pilot Study to Evaluate the Safety and Feasibility of Gene Therapy With CSL200 Autologous Enriched CD34 Cell Fraction That Contains CD34 Cells Transduced With Lentiviral Vector Encoding Human γ-GlobinG16D and Short-Hairpin RNA734 in Adult Subjects With Severe Sickle Cell Disease
Status:
TERMINATED
Status Verified Date:
2021-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Unanticipated delays not for safety reasons
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None