NCT06291961

A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major

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: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: CorrectSequence Therapeutics Co., Ltd · Industry
• Sex: All
• Age: 12 Years – 35 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Beta-Thalassemia Major

Interventions

Type	Name	Description
GENETIC	CS-101 injection	Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique

Timeline

• Start date: 2024-04-18
• Primary completion: 2025-11-17
• Completion: 2025-11-17
• First posted: 2024-03-04
• Last updated: 2026-02-10

Locations

3 sites across 1 country: China