Testing CS-101, a gene-based treatment, for people with β-thalassemia major
Tests a single dose of CS-101 in people with β-thalassemia major.
Plain-language title generated from the registry record
Registry title: A Phase 2 Safety and Efficacy Study Evaluating CS-101 in Participants With β-Thalassemia Major
Key facts
- Where
- Nanning and Shanghai, ChinaSee the 3 sites
- Phase
- Phase 2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 12 and 35 years old?
Source: NCT07489196, who can take part
Where in China
- The First Affiliated Hospital of Guangxi Medical UniversityNanning, ChinaNo longer taking part
- Children's Hospital Of Fudan UniversityShanghai, ChinaNo longer taking part
- Ruijin Hospital Shanghai Jiaotong University School of MedicineShanghai, ChinaNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- CorrectSequence Therapeutics Co., Ltd
- Trial code
- NCT07489196
- Record updated
- 24 March 2026
- Original description
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 MajorRead full original descriptionShow less
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
Inclusion: 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: 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; Received live or live-attenuated vaccine within 90 days prior to myeloablation; 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; 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; 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; Participants with autoimmune diseases; Echocardiographic ejection fraction < 50%; Laboratory abnormalities: AST or ALT > 3 × upper limit of normal (ULN); or International normalized ratio (INR) > 1.5 × ULN; Leukocyte count < 3 × 10⁹/L and/or platelet count < 100 × 10⁹/L not due to hypersplenism; Pregnant or lactating females; females of childbearing potential with a positive serum pregnancy test; Participants with coexisting α-thalassemia with more than 2 α-globin chain gene deletions or non-deletional mutations.