NCT06291961

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

Study 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.

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Interventions

CS-101 injectionGENETIC

Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique

Study Locations

Facility	City	State	Country
The First Affiliated Hospital of Guangxi Medical University	Nanning	Guangxi	China
Children's Hospital of Fudan University	Shanghai	Shanghai Municipality	China
Ruijin Hospital Shanghai JiaoTong University School of Medicine	Shanghai	Shanghai Municipality	China

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026