Ignite Creation Date:
2025-12-24 @ 11:51 AM
Ignite Modification Date:
2025-12-28 @ 3:27 PM
Study NCT ID:
NCT06291961
Status:
RECRUITING
Last Update Posted:
2024-07-01
First Post:
2024-02-23
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major
Sponsor:
CorrectSequence Therapeutics Co., Ltd
Organization:
Study Overview
Official Title:
A Single-arm, Open-label Phase I Clinical Trial to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamic Profile of a Single Dose of CS-101 Injection in Subjects With β-thalassemia Major
Status:
RECRUITING
Status Verified Date:
2024-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
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 in treating patients with β-thalassemia major anemia.
Detailed Description:
CS-101 is an autologous CD34+(Cluster of differentiation 34) cell suspension, edited by in vitro base editing technology, which modifies the BCL11A(B-cell lymphoma/leukemia 11A) binding site in HBG(Hemoglobin Subunit Gamma) 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 HbF(fetal hemoglobin) in the blood, compensating for the function of missing HbA(adult hemoglobin) 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:
False
Is a FDA Regulated Drug?:
False
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?: