Beta-Thalassemia
Conditions
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.
Interventions
GENETICCS-101
Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique
Sponsors
Children's Hospital of Fudan University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* There are no
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 | — |
| New malignancies and hematologic disorders | Signing of informed consent up to 2 years post CS-101 infusion | Based on ICD-11 |
| 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 | — |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
| Change in fetal hemoglobin(HbF) concentration over time | up to 2 years post-CS-101 infusion | — |
Countries
China
Contacts
Primary ContactXiaowen Zhai, M.D.
Backup ContactZifeng Li, M.S.
Outcome results
None listed