A Long-term Follow-up Study in Participants Who Received CS-101
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Safety and efficacy assessments.
- Who it may be relevant to
- Registry conditions: Beta-Thalassemia. Basic parameters: 6 years — 35 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
Overview
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-06 (NCT06024876),No investigational drug product will be administered in the study.
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 -101-06 (NCT06024876). Subjects in the CS-101-06 study will be entered into long-term follow-up of this study up to 15 years post-infusion at the completion of the last (12-month) followup visit after treatment with CS-101 Injection.
Interventions
- Other Safety and efficacy assessments
Safety evaluations,disease-specific assessments,and assessments to monitor for long-term complications of autologous transplant
Primary outcome measures
- Frequency and severity of SAEs and CS-101 related AES as assessed by CTCAE v5.0 [Time frame: Through 15 years post CS-101 infusion]
- New malignancies and hematologic disorders [Time frame: Through 15 years post CS-101 infusion]
- Occurrence of all-cause death [Time frame: Through 15 years post CS-101 infusion]
- Occurrence of achieving transfusion independence for at least 12 consecutive months [Time frame: Through 15 years post CS-101 infusion]
Secondary outcome measures (5)
- Change in fetal hemoglobin(HbF) concentration over time [Time frame: Through 15 years post CS-101 infusion]
- Change in total hemoglobin(Hb) concentration over time [Time frame: Through 15 years post CS-101 infusion]
- Chimerism level in Peripheral blood Proportion of alleles with intended genetic modification in [Time frame: Through 15 years post CS-101 infusion]
- Chimerism level in bone marrow Proportion of alleles with intended genetic modification in [Time frame: Through 15 years post CS-101 infusion]
- Change in F-Cell concentration over time [Time frame: Through 15 years post CS-101 infusion]
Eligibility criteria
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 study
Exclusion criteria
- There are no exclusion criteria
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Other
Study locations
China · 1 center
- The First Affiliated Hospital of Guangxi Medical University — Nanning
Identifiers
NCT: NCT06685536 · CS-101-13