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

Not Applicable

Recruiting

• Conditions: Beta-Thalassemia

• Registration Number: NCT06479616
• Lead Sponsor: Children's Hospital of Fudan University

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)

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 -101-03 (NCT06065189) .

Subjects in the CS-101-03 study will be entered into long-term follow-up of this study up to 2 years post-infusion at the completion of the last (6-month) follow-up visit after treatment with CS-101 Injection.

Study Timeline

• Status Verified: 2024-06
• Study Start: 2024-06-18
• Study First Submitted: 2024-06-24
• Study First Submitted QC: 2024-06-24
• Last Update Submitted: 2024-06-24
• Study First Posted: 2024-06-28
• Last Update Posted: 2024-06-28
• Primary Completion: 2026-12
• Study Completion: 2027-06

Recruitment & Eligibility

• Status: RECRUITING
• 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 IIT study

Exclusion Criteria

• There are no exclusion criteria

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Frequency and severity of SAEs and CS-101 related AES as assessed by CTCAE v5.0	Signing of informed consent up to 2 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	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
New malignancies and hematologic disorders	Signing of informed consent up to 2 years post CS-101 infusion	Based on ICD-11

Secondary Outcome Measures

Name	Time	Method
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	Proportion of alleles with intended genetic modification in peripheral blood leukocytes and bone marrow over time

Trial Locations

Locations (1)

Children's Hospital of Fudan University; 🇨🇳 Shanghai, Shanghai, China