A Long-term Follow-up Study in Participants Who Received CS-101
- Conditions
- Beta-Thalassemia
- Interventions
- Genetic: CS-101
- Registration Number
- NCT06479616
- Lead Sponsor
- Children's Hospital of Fudan University
- Brief Summary
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)
- Detailed Description
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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 5
- Participants (or his or her legally appointed and authorized representative or guardian) must sign and date informed consent form (ICF) and, where applicable, an assent form
- Participants must have received CS-101 infusion in last IIT study
- There are no exclusion criteria
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description long term follow up CS-101 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.
- Primary Outcome Measures
Name Time Method Frequency and severity of SAEs and CS-101 related AES as assessed by CTCAE v5.0 Signing of informed consent up to 2 years post CS-101 infusion CommonTerminology Criteria for Adverse Events（CTCAE）has 5 levels of AE determination, increasing in severity as the level increases
Occurrence of all-cause death Signing of informed consent up to 2 years post CS-101 infusion Occurrence of achieving transfusion independence for at least 12 consecutive months From 3 months after last RBC transfusion up to 2 years post CS-101 infusion New malignancies and hematologic disorders Signing of informed consent up to 2 years post CS-101 infusion Based on ICD-11
- Secondary Outcome Measures
Name Time Method Change in fetal hemoglobin(HbF) concentration over time up to 2 years post-CS-101 infusion Change in total hemoglobin(Hb) concentration over time 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 up to 2 years post-CS-101 infusion Proportion of alleles with intended genetic modification in peripheral blood leukocytes and bone marrow over time
Trial Locations
- Locations (1)
Children's Hospital of Fudan University
🇨🇳Shanghai, Shanghai, China