A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection

• Conditions: Beta-Thalassemia

• Registration Number: NCT06717932
• Lead Sponsor: CorrectSequence Therapeutics Co., Ltd

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.

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 -101-01 (NCT06291961). Subjects in the CS-101-01 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) follow up visit after treatment with CS-101 Injection.

Study Timeline

• Status Verified: 2024-12
• Study First Submitted: 2024-12-01
• Study First Submitted QC: 2024-12-01
• Last Update Submitted: 2024-12-01
• Study First Posted: 2024-12-05
• Last Update Posted: 2024-12-05
• Study Start: 2025-05
• Primary Completion: 2039-11
• Study Completion: 2039-11

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 8

Inclusion Criteria

• 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

Exclusion Criteria

• There are no exclusion criteria

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Frequency and severity of SAEs and CS-101 related AES as assessed by CTCAE v5.0	Through 15 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	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 Outcome Measures

Name	Time	Method
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
Change in total hemoglobin(Hb) concentration over time	Through 15 years post CS-101 infusion
Change in fetal hemoglobin(HbF) concentration over time	Through 15 years post CS-101 infusion

Trial Locations

Locations (1)

The First Affiliated Hospital of Guangxi Medical University; 🇨🇳 Nanning, China