A Phase 2 Safety and Efficacy Study Evaluating CS-101 in Participants With β-Thalassemia Major

March 19, 2026 updated by: CorrectSequence Therapeutics Co., Ltd

A Single-arm, Open-label Phase II Clinical Trial: Evaluating the Safety and Efficacy of a Single Dose of CS-101 Injection in Participants With β-thalassemia Major

The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating patients with β-Thalassemia Major

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Not yet recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

CS-101 is an autologous CD34+(Cluster of differentiation 34) cell suspension, edited by ex vivo base editing technology, which modifies the BCL11A binding site in HBG(Hemoglobin Subunit Gamma) promoter, so that it loses the ability to bind to BCL11A, which can re-induce the production of γ-globin chain and increase the concentration of HbF(fetal hemoglobin) in the blood, compensating for the function of missing HbA(adult hemoglobin) to achieve clinical cure. The therapy addresses two major challenges in the current treatment of the disease: lack of matching donors and graft-versus-host diseases in allogeneic hematopoietic stem cell transplantation.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
20

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

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

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • China
    • Guangxi
      • Nanning, Guangxi, China
        • The First Affiliated Hospital of Guangxi Medical University
        • Contact:
          • Yongrong Lai
        • Principal Investigator:
          • Yongrong Lai
    • Shanghai Municipality
      • Shanghai, Shanghai Municipality, China
        • Children's Hospital of Fudan University
        • Contact:
          • Xiaowen Zhai
        • Principal Investigator:
          • Xiaowen Zhai
      • Shanghai, Shanghai Municipality, China
        • Ruijin Hospital Shanghai Jiaotong University School of Medicine
        • Principal Investigator:
          • Weili Zhao
        • Principal Investigator:
          • Saijuan Chen

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Voluntarily signed informed consent. Male or female participants aged 12 to 35 years (inclusive). The participant or their legally authorized representative must sign the informed consent. If the participant is under 18 years of age, their legally authorized representative must also sign the informed consent.
  • Diagnosed with β-thalassemia major (transfusion-dependent). Received at least 8 units of red blood cell transfusions within 12 months prior to screening, and documented hemoglobin level ≤ 70 g/L pre-transfusion.
  • Good general condition: Karnofsky score (≥16 years of age) ≥ 60, or Lansky Play-Performance score (<16 years of age) ≥ 60.
  • For females of childbearing potential: From the start of the screening, highly effective contraception or complete abstinence (if this is their usual lifestyle), and agree to maintain such contraception throughout the study.
  • For males of childbearing potential: Use condoms or other methods to ensure effective contraception for sexual partners continuously from mobilization through the study period.

Exclusion Criteria:

  • Received other investigational products or other experimental interventions within 30 days prior to signing informed consent or within 6 elimination half-lives of the drug (whichever is longer).
  • Received or is receiving thalidomide, hydroxyurea, and/or luspatercept within 3 months prior to screening.
  • Previous received allogeneic hematopoietic stem cell transplantation, gene therapy, or gene-editing therapy; or participants who can be maintained with standard therapy.
  • Participants with a matched sibling donor, or with a matched unrelated / haploidentical related donor and judged by the investigator to have no high-risk factors for allogeneic hematopoietic stem cell transplantation.
  • Participants with coexisting α-thalassemia with more than 2 α-globin chain gene deletions or non-deletional mutations.
  • Known hypersensitivity to drugs used during autologous hematopoietic stem cell transplantation, excipients, or devices, judged by the investigator to be ineligible for this study.
  • Infection with HIV, cytomegalovirus, Epstein-Barr virus, or Treponema pallidum during screening; active HBV or HCV infection (participants with stable hepatitis B after treatment (HBV-DNA negative) and cured hepatitis C (HCV-RNA negative) may be included). Known active bacterial, viral, fungal, or parasitic infection.
  • Echocardiographic ejection fraction < 50%.
  • Laboratory abnormalities: AST or ALT > 3 × upper limit of normal (ULN); or International normalized ratio (INR) > 1.5 × ULN.
  • Cardiac severe iron overload detected by MRI during screening, judged by the investigator to be unsuitable for hematopoietic stem cell transplantation.
  • Current or history of malignancy.
  • Participants with known neurological consciousness disorders, psychological problems, or psychiatric diseases judged by the investigator to be unable to comply with study procedures.
  • Participants with known history of uncontrolled seizures judged by the investigator to be ineligible for this study.
  • Uncontrolled bleeding disorders.
  • Leukocyte count < 3 × 10⁹/L and/or platelet count < 100 × 10⁹/L not due to hypersplenism.
  • Participants with other severe cardiovascular, pulmonary, renal, gastrointestinal, hepatic diseases, and/or other organ disorders judged by the investigator to be ineligible for this study.
  • Pregnant or lactating females; females of childbearing potential with a positive serum pregnancy test.
  • Received live or live-attenuated vaccine within 90 days prior to myeloablation.
  • Participants with autoimmune diseases

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

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: CS-101 injection
Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique
Genetic: CS-101 injection
Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
AEs(Adverse Events) and SAEs(Serious Adverse Events) after CS-101 infusion
Time Frame: Up to 16 months post-CS-101 infusion
Frequency and severity of adverse events(AEs）as assessed by CTCAE(Common Terminology Criteria for Adverse Events)v5.0
Up to 16 months post-CS-101 infusion
Overall survival
Time Frame: Up to 16 months post-CS-101 infusion
Up to 16 months post-CS-101 infusion
Proportion of Subjects with engraftment
Time Frame: Within 42 days post-CS-101 infusion
Subjects with engraftment is defined as neutrophil engrafted
Within 42 days post-CS-101 infusion
Time to neutrophil engraftment
Time Frame: Up to 16 months post-CS-101 infusion
Up to 16 months post-CS-101 infusion
Time to platelet engraftment
Time Frame: Up to 16 months post-CS-101 infusion
Up to 16 months post-CS-101 infusion
Incidence of transplant-related mortality
Time Frame: Up to 100 days post-CS-101 infusion
Up to 100 days post-CS-101 infusion
Proportion of subjects achieving transfusion independence for at least 12 consecutive months
Time Frame: Up to 16 months post-CS-101 infusion
Maintaining transfusion independence for at least 12 consecutive months while maintaining a weighted mean hemoglobin≥90g/L
Up to 16 months post-CS-101 infusion

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Proportion of subjects achieving transfusion independence for at least 6 consecutive months
Time Frame: Up to 16 months post CS-101 Infusion
Maintaining transfusion independence for at least 6 consecutive months while maintaining a weighted mean hemoglobin≥90g/L
Up to 16 months post CS-101 Infusion
Changes in targeted editing efficiency in peripheral blood nucleated cells over time
Time Frame: Up to 16 months post CS-101 infusion
Up to 16 months post CS-101 infusion
Changes in targeted editing efficiency in bone marrow nucleated cells over time
Time Frame: Up to 16 months post CS-101 infusion
Up to 16 months post CS-101 infusion
Change in fetal hemoglobin(HbF) concentration over time
Time Frame: Up to 16 months post CS-101 infusion
Up to 16 months post CS-101 infusion
Change in total hemoglobin(Hb) concentration over time
Time Frame: Up to 16 months post CS-101 infusion
Up to 16 months post CS-101 infusion

Collaborators and Investigators

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

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
CorrectSequence Therapeutics Co., Ltd

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 (Estimated)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 5, 2026

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 31, 2028

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 31, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 19, 2026

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 19, 2026

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 24, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 24, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 19, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2026

More Information

Terms related to this study

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CS-101-09

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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