The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of base-edited autologous hematopoietic stem cell transplantation(CS-101) in treating patients with β-thalassemia major.
Age range
3 Years – 17 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Frequency and severity of adverse events(AEs) as assessed by CTCAE v5.0
Timeframe: From signing informed consent to 180 days post-CS-101 infusion
Occurrence of engraftment
Timeframe: within 42 days post-CS-101 infusion
Time to neutrophil and platelet engraftment
Timeframe: Days post-CS-101 infusion
Occurrence of transplant-related death
Timeframe: baseline to 100 days post-CS-101 infusion
Occurrence of all-cause death
Timeframe: From signing informed consent to 180 days post-CS-101 infusion
Occurrence of achieving transfusion reduction for at least 3 consecutive months
Timeframe: From 3 months post -CS-101 infusion to 3 months post -CS-101 infusion