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

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

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)

Study Overview

• Status: Recruiting
• Conditions: Beta-Thalassemia
• Intervention / Treatment: Genetic: CS-101

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 Type: Interventional
• Enrollment (Estimated): 5
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Xiaowen Zhai, M.D.; Phone Number: +86-021-64931126; Email: zhaixiaowendy@163.com
• Study Contact Backup: Name: Zifeng Li, M.S.; Phone Number: +86-13920704768; Email: zfli18@fudan.edu.cn

Study Locations

• China
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China, 201102
      • Recruiting
      • Children's Hospital of Fudan University
      • Contact: Zifeng Li, M.S.; Phone Number: +86-13920704768; Email: zfli18@fudan.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: long term follow up; All participants who complete CS-101-03(NCT0606518) studie after CS-101 infusion will be asked to participate in this long-term follow-up study.	Genetic: CS-101; Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	Signing of informed consent up to 2 years post CS-101 infusion
Occurrence of all-cause death		Signing of informed consent up to 2 years post CS-101 infusion
New malignancies and hematologic disorders	Based on ICD-11	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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	Proportion of alleles with intended genetic modification in peripheral blood leukocytes and bone marrow over time	up to 2 years post-CS-101 infusion

Collaborators and Investigators

• Sponsor: Children's Hospital of Fudan University
• Investigators: Principal Investigator: Xiaowen Zhai, M.D., Children's Hospital of Fudan University

Study record dates

Study Major Dates

• Study Start (Actual): June 18, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): June 1, 2027

Study Registration Dates

• First Submitted: June 24, 2024
• First Submitted That Met QC Criteria: June 24, 2024
• First Posted (Actual): June 28, 2024

Study Record Updates

• Last Update Posted (Estimated): September 10, 2025
• Last Update Submitted That Met QC Criteria: September 4, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Thalassemia; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; beta-Thalassemia
• Other Study ID Numbers: CS-101-04

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No