CS-101 in Patients With β-thalassemia

Ki vehet részt

• 6 to 35 years old(inclusive) male or female subjects at the time of informed consenting Diagnosis of β-thalassemia, genotypes include but are not limited to β+β0，βEβ0，β0β0, etc History of at least≥8 units/year of packed RBC transfusions in the prior 12 months prior to the screening period Generally in good condition, Karnofsky performance score≥60 points for subjects≥16 years old at the time of autologous hematopoietic stem cell collection, or Lansky Play-Performance score≥60 points for subjects under 16 years old, or equivalent clinical evaluation as the investigator site's common practice

Ki nem vehet részt

• Treatment with other investigational medications or other experimental interventions 30 days prior to signing informed consent or within 6 half-lives of the drug, whichever is longer; Subjects who have received or are receiving thalidomide and/or Luspatercept, when their drug-drug interaction on the efficacy and safety of CS-101 cannot be ruled out, unless at least there are 3 test results showing the total hemoglobin level before transfusion is below 9g/dL in the past 6 months before screening; Previously received allogeneic hematopoietic stem cell transplantation or gene(edited) therapy; Subjects have available related fully matching donors and are eligible and prepared for allogeneic hematopoietic stem cell transplantation; Those with active infections, including but not limited to: HIV, hepatitis B, hepatitis C, cytomegalovirus, Epstein-Barr virus and treponema pallidum test positive, or known tuberculosis, parasitic infection, etc. who are judged by the investigator to be unsuitable to participate in this study; Echocardiography results with ejection fraction below 45%. Advanced liver disease, defined as：; Aspartate aminotransferase (AST), alanine aminotransferase (ALT) >3 × upper limit of normal (ULN) or:; Baseline International Normalized Ratio (INR) >1.5 × ULN; MRI during the screening period showed heavy iron overload and is judged by the investigator to be unable to participate in the study