A Long-term Follow-up Study in Participants Who Received CS-101

• Conditions: Beta-Thalassemia

• Registration Number: NCT06685536
• 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 -101-06 (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.

Study Timeline

• Status Verified: 2024-11
• Study First Submitted: 2024-11-07
• Study First Submitted QC: 2024-11-10
• Study First Posted: 2024-11-12
• Study Start: 2025-01-18
• Last Update Submitted: 2025-02-07
• Last Update Posted: 2025-02-11
• Primary Completion: 2039-07
• Study Completion: 2039-07

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 5

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

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 achieving transfusion independence for at least 12 consecutive months	Through 15 years post CS-101 infusion
New malignancies and hematologic disorders	Through 15 years post CS-101 infusion	Based on ICD-11
Occurrence of all-cause death	Through 15 years post CS-101 infusion

Secondary Outcome Measures

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

Trial Locations

Locations (1)

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