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Beta-Thalassemia MajorPhase 1NCT06291961

A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major

A Phase 1 study testing CS-101 injection in people with Beta-Thalassemia Major, run by CorrectSequence Therapeutics Co., Ltd at 3 sites in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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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 anemia. Read more about this study

Who is it for?
Ages 12 to 35 years
Conditions the study is for
Beta-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
9 participants
Where
3 sitesChina
Study dates
18 Apr 2024to 17 Nov 2025
Phase
Phase 1
Are there results?
None posted in the registry

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

  • 12 to 35 years old(inclusive) male or female subjects at the time of informed consenting. For minors, their legal representative is required to sign the informed consent form, besides, if the subjects aged 8 years or older, they should provide a signed and dated.
  • Diagnosis of β-thalassemia major.
  • Generally in good condition, Karnofsky performance score≥60 points for subjects≥16 years old, or Lansky Play-Performance score≥60 points for subjects under 16 years old.
  • For female subjects of childbearing potential: use effective contraceptive measures from the start of screening and agree to continue using such measures for contraception throughout the study
  • For male subjects who have a potential ability to father a child: use condoms or other methods continuously from the start of mobilization to ensure effective contraception for sexual partners during the study period

Who cannot join

  • Treatment with other investigational medications or other experimental interventions 30 days prior to signing informed consent or within 6 half-lives of the drug, whichever is longer
  • Subjects who have received or are receiving thalidomide and/or Luspatercept in the past 6 months before screening
  • Previously received allogeneic hematopoietic stem cell transplantation or gene(edited) therapy
  • Subjects have available related fully matching donors and are eligible and prepared for allogeneic hematopoietic stem cell transplantation
  • Patients with coexisting α-thalassemia and more than 2 deletions or non-deletional mutations in the α-globin chain coding genes
  • Known to be allergic to drugs used during autologous hematopoietic stem cell transplantation (including but not limited to granulocyte colony-stimulating factor, busulfan, dextran), excipients(such as dimethyl sulfoxide), or instruments(such as intravenous catheters) as determined by the investigator are deemed unsuitable to participate in this study
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  • Those with positive results in HIV, cytomegalovirus, Epstein-Barr virus and treponema pallidum test, active infection of hepatitis B, hepatitis C, or known tuberculosis, parasitic infection, etc. Hepatitis B stabilized on medication(HBV-DNA test negative) and cured hepatitis C(HCV-RNA test negative) can be considered acceptable.
  • Echocardiography shows ejection fraction below 45%
  • Laboratory indicators, defined as：Aspartate aminotransferase(AST), alanine aminotransferase(ALT) >3× upper limit of normal(ULN) or Baseline International Normalized Ratio(INR)>1.5×ULN.
  • MRI during the screening period shows severe cardiac iron overload and other conditions, and are judged by the investigator to be intolerable or inappropriate for autologous hematopoietic stem cell transplantation
  • Patients with past/present history of cancer
  • Known neurological disorders, psychological problems or mental illness, and is judged by the investigator to be unable to cooperate with the study procedures
  • Known history of uncontrolled epileptic seizures and is judged by the investigator to be unfit to participate in this study
  • The investigators determined that a non-hypersplenism-induced white blood cell count of<3×10^9/L, and/or a platelet count of<100×10^9/L.
  • Known history of other serious cardiovascular, pulmonary, renal diseases, digestive tract conditions, liver diseases and / or other conditions, etc., and are judged by the investigator to be intolerable or inappropriate for autologous hematopoietic stem cell mobilization, collection, and myeloablative conditioning and infusion
  • Pregnant or lactating women

Trial locations and contacts

3 sites in 1 country.

China3 sites

  • The First Affiliated Hospital of Guangxi Medical University

    Nanning, Guangxi

  • Children's Hospital of Fudan University

    Shanghai, Shanghai Municipality

  • Ruijin Hospital Shanghai JiaoTong University School of Medicine

    Shanghai, Shanghai Municipality

Other trials in China

About this study

Official title: A Single-arm, Open-label Phase I Clinical Trial to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamic Profile of a Single Dose of CS-101 Injection in Subjects 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 anemia.

Read the detailed description

CS-101 is an autologous CD34+(Cluster of differentiation 34) cell suspension, edited by in vitro base editing technology, which modifies the BCL11A(B-cell lymphoma/leukemia 11A) 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.

Being tested
CS-101 injection
Medical subject headings
beta-Thalassemia

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 in vitro base editing technique

Study groups in NCT06291961
Study groupRoleWhat they receive
CS-101 injectionExperimentalAutologous CD34+ hematopoietic stem cell suspension modified by in vitro 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.

  1. 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: From signing informed consent to 12 months post-CS-101 infusion

  2. Overall survival rate

    Measured: Up to 12 months post-CS-101 infusion

  3. Proportion of subjects with engraftment

    Subjects with engraftment is defined as neutrophil engrafted

    Measured: Within 42 days post-CS-101 infusion

  4. Time to neutrophil engraftment

    Time to neutrophil engraftment is defined as first day of 3 consecutive measurements of absolute neutrophil count≥0.5×10^9/L on three different days.

    Measured: Up to 12 months post-CS-101 infusion

  5. Time to platelet engraftment

    Time to platelet engraftment is defined as first day of 3 consecutive measurements of absolute platelet count≥20×10^9/L on three different days and without platelet transfusion.

    Measured: Up to 12 months post-CS-101 infusion

  6. Incidence of transplant-related mortality

    Incidence of transplant-related mortality(Transplant-related mortality events defined as deaths assessed by the investigator as potentially transplant-related)

    Measured: From baseline to 100 days post-CS-101 infusion

  7. Change in fetal hemoglobin(HbF) concentration over time

    Fetal hemoglobin(HbF) concentration from baseline to 9 months post-CS-101 infusion

    Measured: Up to 12 months post-CS-101 infusion

  8. Change in total hemoglobin(Hb) concentration over time

    Total hemoglobin concentration change from baseline to 9 months post-CS-101 infusion

    Measured: Up to 12 months post-CS-101 infusion

Secondary outcomes (4)

  1. Chimerism level in Peripheral blood and bone marrow

    Proportion of alleles with intended genetic modification in peripheral blood leukocytes and bone marrow over time

    Measured: Up to 12 months post-CS-101 infusion

  2. 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≥9 g/dL Proportion of subjects

    Measured: From CS-101 infusion up to 12 months post-CS-101 infusion

  3. Proportion of subjects achieving fetal hemoglobin(HbF) increase≥2.0 g/dL

    Fetal hemoglobin(HbF) concentration increase≥2.0 g/dL within 2 months post-CS-101 infusion

    Measured: Up to 2 months post-CS-101 infusion

  4. Proportion of subjects achieving fetal hemoglobin(HbF) increase≥7.0 g/dL

    Fetal hemoglobin(HbF) concentration increase≥7.0 g/dL within 6 months post-CS-101 infusion

    Measured: Up to 6 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.

  1. First posted4 Mar 2024
  2. Study start18 Apr 2024
  3. Primary completion17 Nov 2025
  4. Study completion17 Nov 2025

Resources

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 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
9 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06291961
Sponsor's protocol ID
CS-101-01
Status last verified
Feb 2026
First submitted
23 Feb 2024
Last updated
10 Feb 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CS-101 injection in people with Beta-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 16 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: finished, as of the sponsor's last update on 10 Feb 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. 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: From signing informed consent to 12 months post-CS-101 infusion). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06291961.

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 10 Feb 2026.

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