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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AEs(Adverse Events) and SAEs(Serious Adverse Events) after CS-101 infusion
Timeframe: From signing informed consent to 12 months post-CS-101 infusion
Overall survival rate
Timeframe: Up to 12 months post-CS-101 infusion
Proportion of subjects with engraftment
Timeframe: Within 42 days post-CS-101 infusion
Time to neutrophil engraftment
Timeframe: Up to 12 months post-CS-101 infusion
Time to platelet engraftment
Timeframe: Up to 12 months post-CS-101 infusion
Incidence of transplant-related mortality
Timeframe: From baseline to 100 days post-CS-101 infusion
Change in fetal hemoglobin(HbF) concentration over time
Timeframe: Up to 12 months post-CS-101 infusion
Change in total hemoglobin(Hb) concentration over time
Timeframe: Up to 12 months post-CS-101 infusion