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

February 8, 2026 updated by: CorrectSequence Therapeutics Co., Ltd

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 -101-06 (NCT06024876),No investigational drug product will be administered in the study.

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

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 Type

Observational

Enrollment (Estimated)

5

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Guangxi
      • Nanning, Guangxi, China
        • The First Affiliated Hospital of Guangxi Medical University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Subject with β-thalassemia treated with CS-101 infusion in last study

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 study

Exclusion Criteria:

  • There are no exclusion criteria

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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
Time Frame: 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
Through 15 years post CS-101 infusion
New malignancies and hematologic disorders
Time Frame: Through 15 years post CS-101 infusion
Based on ICD-11
Through 15 years post CS-101 infusion
Occurrence of all-cause death
Time Frame: Through 15 years post CS-101 infusion
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
Through 15 years post CS-101 infusion

Secondary Outcome Measures

Outcome Measure
Time Frame
Change in fetal hemoglobin(HbF) concentration over time
Time Frame: Through 15 years post CS-101 infusion
Through 15 years post CS-101 infusion
Change in total hemoglobin(Hb) concentration over time
Time Frame: Through 15 years post CS-101 infusion
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
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
Through 15 years post CS-101 infusion
Change in F-Cell concentration over time
Time Frame: Through 15 years post CS-101 infusion
Through 15 years post CS-101 infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CorrectSequence Therapeutics Co., Ltd

Collaborators

First Affiliated Hospital of Guangxi Medical University

Investigators

  • Principal Investigator: Yongrong Lai, M.D., First Affiliated Hospital of Guangxi Medical University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 18, 2025

Primary Completion (Estimated)

July 1, 2039

Study Completion (Estimated)

July 1, 2039

Study Registration Dates

First Submitted

November 7, 2024

First Submitted That Met QC Criteria

November 10, 2024

First Posted (Actual)

November 12, 2024

Study Record Updates

Last Update Posted (Actual)

February 10, 2026

Last Update Submitted That Met QC Criteria

February 8, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CS-101-13

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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