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

NCT07489196 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: CS-101-09
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 3

Brief Summary

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

Conditions

β-thalassemia Major

Outcome Measures

Primary Outcomes (7)

• AEs(Adverse Events) and SAEs(Serious Adverse Events) after 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

  Up to 16 months post-CS-101 infusion

• Proportion of Subjects with engraftment

  Subjects with engraftment is defined as neutrophil engrafted

  Within 42 days post-CS-101 infusion

• Time to neutrophil engraftment

  Up to 16 months post-CS-101 infusion

• Time to platelet engraftment

  Up to 16 months post-CS-101 infusion

• Incidence of transplant-related mortality

  Up to 100 days post-CS-101 infusion

• Proportion of subjects achieving transfusion independence for at least 12 consecutive months

  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 Outcomes (5)

• Proportion of subjects achieving transfusion independence for at least 6 consecutive months

  Up to 16 months post CS-101 Infusion

• Changes in targeted editing efficiency in peripheral blood nucleated cells over time

  Up to 16 months post CS-101 infusion

• Changes in targeted editing efficiency in bone marrow nucleated cells over time

  Up to 16 months post CS-101 infusion

• Change in fetal hemoglobin(HbF) concentration over time

  Up to 16 months post CS-101 infusion

• Change in total hemoglobin(Hb) concentration over time

  Up to 16 months post CS-101 infusion

Study Arms (1)

CS-101 injection

EXPERIMENTAL

Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique

Genetic: CS-101 injection

Interventions

CS-101 injection GENETIC

Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique

CS-101 injection

Eligibility Criteria

• Age: 12 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• CorrectSequence Therapeutics Co., Ltd: lead

Study Sites (3)

The First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, China

Location

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, China

Location

Ruijin Hospital Shanghai JiaoTong University School of Medicine

Shanghai, Shanghai Municipality, China

Location

Central Study Contacts

Xiaokai Li

CONTACT

+86 18686610731; xiaokai.li@correctsequence.com

Yaliang Li

CONTACT

+8618621046122; yaliang.li@correctsequence.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 19, 2026
• First Posted: March 24, 2026
• Study Start: April 5, 2026
• Primary Completion (Estimated): January 31, 2028
• Study Completion (Estimated): July 31, 2028
• Last Updated: March 24, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (3)