β-thalassemia MajorPhase 2NCT07489196
A Phase 2 Safety and Efficacy Study Evaluating CS-101 in Participants With β-Thalassemia Major
A Phase 2 study testing CS-101 injection in people with β-thalassemia Major, run by CorrectSequence Therapeutics Co., Ltd at 3 sites in China.
Trial 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 Read more about this study
- Who is it for?
- Ages 12 to 35 years
- Conditions the study is for
- β-thalassemia Major
- What is being tested?
- CS-101 injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 3 sitesChina
- Study dates
- 5 Apr 2026to 31 Jul 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 19Show fewer
- 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
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China3 sites
The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi
Yongrong Lai
Yongrong Lai
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality
Xiaowen Zhai
Xiaowen Zhai
Ruijin Hospital Shanghai JiaoTong University School of Medicine
Shanghai, Shanghai Municipality
Saijuan Chen
Weili Zhao
About this study
Official title: 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
Read the 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.
- Conditions
- β-thalassemia Major
- Being tested
- CS-101 injection
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CS-101 injectionGenetic
Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique
| Study group | Role | What they receive |
|---|---|---|
| CS-101 injection | Experimental | Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique Receives: Genetic: CS-101 injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Up to 16 months post-CS-101 infusion
Overall survival
Measured: Up to 16 months post-CS-101 infusion
Proportion of Subjects with engraftment
Subjects with engraftment is defined as neutrophil engrafted
Measured: Within 42 days post-CS-101 infusion
Time to neutrophil engraftment
Measured: Up to 16 months post-CS-101 infusion
Time to platelet engraftment
Measured: Up to 16 months post-CS-101 infusion
Incidence of transplant-related mortality
Measured: 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
Measured: Up to 16 months post-CS-101 infusion
Secondary outcomes (5)
Proportion of subjects achieving transfusion independence for at least 6 consecutive months
Maintaining transfusion independence for at least 6 consecutive months while maintaining a weighted mean hemoglobin≥90g/L
Measured: Up to 16 months post CS-101 Infusion
Changes in targeted editing efficiency in peripheral blood nucleated cells over time
Measured: Up to 16 months post CS-101 infusion
Changes in targeted editing efficiency in bone marrow nucleated cells over time
Measured: Up to 16 months post CS-101 infusion
Change in fetal hemoglobin(HbF) concentration over time
Measured: Up to 16 months post CS-101 infusion
Change in total hemoglobin(Hb) concentration over time
Measured: Up to 16 months post CS-101 infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Mar 2026
- Study start5 Apr 2026 (estimated)
- Primary completion31 Jan 2028 (estimated)
- Study completion31 Jul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for β-thalassemia MajorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CorrectSequence Therapeutics Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07489196
- Sponsor's protocol ID
- CS-101-09
- Status last verified
- Mar 2026
- First submitted
- 19 Mar 2026
- Last updated
- 24 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07489196
- Acronym / sponsor ID
- CS-101-09
- Conditions
- β-thalassemia Major
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- CorrectSequence Therapeutics Co., LtdIndustry (pharma, biotech, devices)
- Study start
- 5 Apr 2026 (estimated)
- Primary completion
- 31 Jan 2028 (estimated)
- Study completion
- 31 Jul 2028 (estimated)
- First posted
- 24 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CS-101 injection | Experimental | Genetic: CS-101 injectionAutologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique |
Who may qualify
- Ages
- 12 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (19 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CS-101 injectionAutologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique | Genetic | CS-101 injection |
Primary outcome
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
Overall survival
Time frame: 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
Time to neutrophil engraftment
Time frame: Up to 16 months post-CS-101 infusion
Time to platelet engraftment
Time frame: Up to 16 months post-CS-101 infusion
Incidence of transplant-related mortality
Time frame: 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
5 secondary outcomes
Proportion of subjects achieving transfusion independence for at least 6 consecutive months
Time frame: 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
Changes in targeted editing efficiency in bone marrow nucleated cells over time
Time frame: 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
Change in total hemoglobin(Hb) concentration over time
Time frame: Up to 16 months post CS-101 infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Mar 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CS-101 injection in people with β-thalassemia Major, run by CorrectSequence Therapeutics Co., Ltd at 3 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 24 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Xiaokai Li (+86 18686610731, xiaokai.li@correctsequence.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
AEs(Adverse Events) and SAEs(Serious Adverse Events) after CS-101 infusion (time frame: Up to 16 months post-CS-101 infusion). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Mar 2026.