An interventional study of CS-101 in Beta-Thalassemia, sponsored by Children's Hospital of Fudan University. Recruiting at 1 site in China. Open to participants aged 3 Years to 17 Years. Per ClinicalTrials.gov, last updated 2025-09-10.
Sponsored by Children's Hospital of Fudan University · Not applicable, Interventional, and Other
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-03 (NCT06065189)
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-03 (NCT06065189) .
Subjects in the CS-101-03 study will be entered into long-term follow-up of this study up to 2 years post-infusion at the completion of the last (6-month) follow-up visit after treatment with CS-101 Injection.
305 studies on the registry are indexed under beta-Thalassemia; 70 are open to participants now.
This study's planned enrollment of 5 is below the median of 38 across 210 interventional studies indexed under beta-Thalassemia.
Browse beta-Thalassemia studies →Children's Hospital of Fudan University is the lead sponsor of 262 studies on the registry; 92 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
All participants who complete CS-101-03(NCT0606518) studie after CS-101 infusion will be asked to participate in this long-term follow-up study.
Genetic: CS-101
Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique
Frequency and severity of SAEs and CS-101 related AES as assessed by CTCAE v5.0
CommonTerminology Criteria for Adverse Events（CTCAE）has 5 levels of AE determination, increasing in severity as the level increases
Time frame: Signing of informed consent up to 2 years post CS-101 infusion
Occurrence of all-cause death
Time frame: Signing of informed consent up to 2 years post CS-101 infusion
New malignancies and hematologic disorders
Based on ICD-11
Time frame: Signing of informed consent up to 2 years post CS-101 infusion
Occurrence of achieving transfusion independence for at least 12 consecutive months
Time frame: From 3 months after last RBC transfusion up to 2 years post CS-101 infusion
Change in fetal hemoglobin(HbF) concentration over time
Time frame: up to 2 years post-CS-101 infusion
Change in total hemoglobin(Hb) concentration over time
Time frame: up to 2 years post-CS-101 infusion
Chimerism level in Peripheral blood and bone marrow Proportion of alleles with intended genetic modification in peripheral blood leukocytes and bone marrow over time
Proportion of alleles with intended genetic modification in peripheral blood leukocytes and bone marrow over time
Time frame: up to 2 years post-CS-101 infusion
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Children's Hospital of Fudan University