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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
OTHER
Masking
NONE
Enrollment
5
Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, China
RECRUITINGFrequency 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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