A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection
A Long-term Follow-up Study Evaluating the Safety and Efficacy of Subjects With β-thalassemia Treated With CS-101 Transplantation
1 other identifier
observational
8
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 -101-01 (NCT06291961),No investigational drug product will be administered in the study.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started May 2025
Longer than P75 for all trials
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
First Submitted
Initial submission to the registry
December 1, 2024
CompletedFirst Posted
Study publicly available on registry
December 5, 2024
CompletedStudy Start
First participant enrolled
May 15, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 1, 2039
ExpectedStudy Completion
Last participant's last visit for all outcomes
November 1, 2039
February 10, 2026
February 1, 2026
14.5 years
December 1, 2024
February 8, 2026
Conditions
Outcome Measures
Primary Outcomes (3)
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
Through 15 years post CS-101 infusion
Occurrence of all-cause death
Through 15 years post CS-101 infusion
Occurrence of achieving transfusion independence for at least 12 consecutive months
Through 15 years post CS-101 infusion
Secondary Outcomes (5)
Change in fetal hemoglobin(HbF) concentration over time
Through 15 years post CS-101 infusion
Change in total hemoglobin(Hb) concentration over time
Through 15 years post CS-101 infusion
Chimerism level in Peripheral blood Proportion of alleles with intended genetic modification in
Through 15 years post CS-101 infusion
Chimerism level in bone marrow Proportion of alleles with intended genetic modification in
Through 15 years post CS-101 infusion
Change in F-Cell concentration over time
Through 15 years post CS-101 infusion
Study Arms (1)
Safety and efficacy assessments
Safety evaluations,disease-specific assessments,and assessments to monitor for long-term complications of autologous transplant
Eligibility Criteria
Subject with β-thalassemia treated with CS-101 infusion in CS-101-01 study
You may qualify if:
- Participants must have received CS-101 infusion in CS-101-01 study
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
The First Affiliated Hospital of Guangxi Medical University
Nanning, China
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- observational
- Observational Model
- OTHER
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 1, 2024
First Posted
December 5, 2024
Study Start
May 15, 2025
Primary Completion (Estimated)
November 1, 2039
Study Completion (Estimated)
November 1, 2039
Last Updated
February 10, 2026
Record last verified: 2026-02
Data Sharing
- IPD Sharing
- Will not share