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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| long term follow up | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CS-101 | Genetic | Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 | Signing of informed consent up to 2 years post CS-101 infusion |
| Occurrence of all-cause death | Signing of informed consent up to 2 years post CS-101 infusion | |
| New malignancies and hematologic disorders | Based on ICD-11 | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Xiaowen Zhai, M.D. | Contact | +86-021-64931126 | zhaixiaowendy@163.com | |
| Zifeng Li, M.S. | Contact | +86-13920704768 | zfli18@fudan.edu.cn |
| Name | Affiliation | Role |
|---|---|---|
| Xiaowen Zhai, M.D. | Children's Hospital of Fudan University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital of Fudan University | Recruiting | Shanghai | Shanghai Municipality | 201102 | China |
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| ID | Term |
|---|---|
| D017086 | beta-Thalassemia |
| ID | Term |
|---|---|
| D013789 | Thalassemia |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
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| 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 |
| up to 2 years post-CS-101 infusion |
| D006402 |
| Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |