Ignite Creation Date:
2024-10-26 @ 3:38 PM
Last Modification Date:
2024-10-26 @ 3:38 PM
Study NCT ID:
NCT06565026
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-08-13
Brief Title:
CS-101 in Patients With Sickle Cell Disease
Sponsor:
None
Organization:
None
Study Overview
Official Title:
An Open-Label Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CD34 Human Hematopoietic Stem Cells Modified Using Transformer Base Editor in Participants With Severe Sickle Cell Disease
Status:
RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The goal of this open label single-arm clinical study is to learn about the safety and efficacy of CS-101 injection in treating sickle cell disease
Detailed Description:
CS-101 is an autologous CD34 cell suspension edited by in vitro base editing technology which modifies the BCL11A binding site in HBG promoter so that it loses the ability to bind to BCL11A which can re-induce the production of γ-globin chain and increase the concentration of fetal hemoglobinHbF in the blood compensating for the function of missing adult hemoglobin HbA to achieve clinical cure The therapy addresses two major challenges in the current treatment of the disease lack of matching donors and graft-versus-host diseases in allogeneic hematopoietic stem cell transplantation
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None