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
Voluntarily signed informed consent. Male or female participants aged 12 to 35 years (inclusive). The participant or their legally authorized representative must sign the informed consent. If the participant is under 18 years of age, their legally authorized representative must also sign the informed consent.
Diagnosed with β-thalassemia major (transfusion-dependent). Received at least 8 units of red blood cell transfusions within 12 months prior to screening, and documented hemoglobin level ≤ 70 g/L pre-transfusion.
Good general condition: Karnofsky score (≥16 years of age) ≥ 60, or Lansky Play-Performance score (<16 years of age) ≥ 60.
For females of childbearing potential: From the start of the screening, highly effective contraception or complete abstinence (if this is their usual lifestyle), and agree to maintain such contraception throughout the study.
Received other investigational products or other experimental interventions within 30 days prior to signing informed consent or within 6 elimination half-lives of the drug (whichever is longer).
Received or is receiving thalidomide, hydroxyurea, and/or luspatercept within 3 months prior to screening.
Previous received allogeneic hematopoietic stem cell transplantation, gene therapy, or gene-editing therapy; or participants who can be maintained with standard therapy.
Participants with a matched sibling donor, or with a matched unrelated / haploidentical related donor and judged by the investigator to have no high-risk factors for allogeneic hematopoietic stem cell transplantation.
Single group
CS-101 injection
GeneticAutologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique
AEs(Adverse Events) and SAEs(Serious Adverse Events) after CS-101 infusion
Frequency and severity of adverse events(AEs）as assessed by CTCAE(Common Terminology Criteria for Adverse Events)v5.0
Overall survival
Proportion of Subjects with engraftment
Subjects with engraftment is defined as neutrophil engrafted
Time to neutrophil engraftment
Proportion of subjects achieving transfusion independence for at least 6 consecutive months
Maintaining transfusion independence for at least 6 consecutive months while maintaining a weighted mean hemoglobin≥90g/L
Changes in targeted editing efficiency in peripheral blood nucleated cells over time
Changes in targeted editing efficiency in bone marrow nucleated cells over time
Change in fetal hemoglobin(HbF) concentration over time
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This trial is not recruiting at the moment. You can still explore other options:
