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Clinical Trial 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.


Clinical Trial 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 Design


Related Conditions & MeSH terms


NCT number NCT06291961
Study type Interventional
Source CorrectSequence Therapeutics Co., Ltd
Contact Yaliang Li
Phone +8618621046122
Email yaliang.li@correctsequence.com
Status Recruiting
Phase Phase 1
Start date April 18, 2024
Completion date July 2025

See also
  Status Clinical Trial Phase
Active, not recruiting NCT00999349 - Therapeutic Effects of Silymarin in Patients With B-thalassemia Major Phase 2/Phase 3
Completed NCT02151526 - A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease Phase 1/Phase 2
Recruiting NCT02342145 - Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major Phase 4
Completed NCT00171301 - Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration) Phase 4
Completed NCT02198508 - Clinical Trial of Deferasirox Combination Treatment With Deferiprone In Thalassaemia Patients N/A
Not yet recruiting NCT01511848 - Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and Desferal In Conditions of Iron Overload Phase 2/Phase 3
Recruiting NCT03653338 - T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias Phase 1/Phase 2