A Long-term Follow-up Study in Participants Who Received CS-101
A Long-term Follow-up Study Evaluating the Safety and Efficacy of Subjects With β-thalassemia Treated With Base-edited Autologous Hematopoietic Stem Cell (CS-101) Transplantation
1 other identifier
interventional
5
1 country
1
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)
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for not_applicable
Started Jun 2024
Typical duration for not_applicable
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 18, 2024
CompletedFirst Submitted
Initial submission to the registry
June 24, 2024
CompletedFirst Posted
Study publicly available on registry
June 28, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
June 1, 2027
September 10, 2025
September 1, 2025
2.5 years
June 24, 2024
September 4, 2025
Conditions
Outcome Measures
Primary Outcomes (4)
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
Secondary Outcomes (3)
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
Study Arms (1)
long term follow up
EXPERIMENTALAll participants who complete CS-101-03(NCT0606518) studie after CS-101 infusion will be asked to participate in this long-term follow-up study.
Interventions
Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique
Eligibility Criteria
You may qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, 201102, China
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Xiaowen Zhai, M.D.
Children's Hospital of Fudan University
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NA
- Masking
- NONE
- Purpose
- OTHER
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 24, 2024
First Posted
June 28, 2024
Study Start
June 18, 2024
Primary Completion (Estimated)
December 1, 2026
Study Completion (Estimated)
June 1, 2027
Last Updated
September 10, 2025
Record last verified: 2025-09