Beta-ThalassemiaEarly Phase 1NCT06024876
A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia
A Early Phase 1 study testing CS-101 in people with Beta-Thalassemia, run by CorrectSequence Therapeutics Co., Ltd at 1 site 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 β-thalassemia. Read more about this study
- Who is it for?
- Ages 6 to 35 years
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- CS-101
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteChina
- Study dates
- 26 Aug 2023to 1 Jul 2025
- Phase
- Early 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 6 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 6 to 35 years old(inclusive) male or female subjects at the time of informed consenting
- Diagnosis of β-thalassemia, genotypes include but are not limited to β+β0，βEβ0，β0β0, etc
- History of at least≥8 units/year of packed RBC transfusions in the prior 12 months prior to the screening period
- Generally in good condition, Karnofsky performance score≥60 points for subjects≥16 years old at the time of autologous hematopoietic stem cell collection, or Lansky Play-Performance score≥60 points for subjects under 16 years old, or equivalent clinical evaluation as the investigator site's common practice
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, when their drug-drug interaction on the efficacy and safety of CS-101 cannot be ruled out, unless at least there are 3 test results showing the total hemoglobin level before transfusion is below 9g/dL 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.
- Those with active infections, including but not limited to: HIV, hepatitis B, hepatitis C, cytomegalovirus, Epstein-Barr virus and treponema pallidum test positive, or known tuberculosis, parasitic infection, etc. who are judged by the investigator to be unsuitable to participate in this study.
- Echocardiography results with ejection fraction below 45%.
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- Advanced liver disease, 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 showed heavy iron overload and is judged by the investigator to be unable to participate in the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Clinical Study Evaluating the Safety and Efficacy of Ex-vivo tBE Edited Autologous Hematopoietic Stem Progenitor Cells (CS-101) in Treating Subjects With β-thalassemia
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating β-thalassemia.
Read the detailed description
CS-101 is an autologous CD34+ cell suspension, edited by ex vivo base editing technology, which modifies the BCL11A binding site in HBG 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 fetal hemoglobin(HbF) in the blood, compensating for the function of missing adult hemoglobin HbA 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
- Beta-Thalassemia
- Being tested
- CS-101
- 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-101Biological
Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique
| Study group | Role | What they receive |
|---|---|---|
| CS-101 | Experimental | CS-101: Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique Receives: Biological: CS-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency and severity of adverse events(AEs）as assessed by CTCAE v5.0
Measured: From signing informed consent to 12 months post-CS-101 infusion
Time to neutrophil and platelet 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； 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 in the past 7 days；
Measured: Days post-CS-101 infusion
Proportion of subjects with engraftment
Subjects with engraftment is defined as neutrophil engrafted
Measured: within 42 days post-CS-101infusion
Incidence of transplant-related mortality
Measured: From baseline to 100 days post-CS-101 infusion
All-cause mortality
Measured: From signing informed consent to 12 months post-CS-101 infusion
Proportion of subjects achieving transfusion independence for at least 6 consecutive months
Measured: From 3 months up to 12 months post-CS-101 infusion
Time to last red blood cell(RBC) transfusion
Measured: Days post-CS-101 infusion
Secondary outcomes (3)
Change in total hemoglobin(Hb) concentration over time
Total hemoglobin concentration change from baseline to 12 months post-CS-101 infusion
Measured: up to 12 months post-CS-101 infusion
Change in fetal hemoglobin(HbF) concentration over time
fetal hemoglobin concentration change from baseline to 12 months post-CS-101 infusion
Measured: up to 12 months post-CS-101 infusion
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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Sep 2023
- Study start26 Aug 2023
- Primary completion1 Jul 2025
- Study completion1 Jul 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Beta-ThalassemiaStudies 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- CorrectSequence Therapeutics Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- First Affiliated Hospital of Guangxi Medical University
- Responsible party
- Sponsor
- Investigators
- Yongrong Lai, M.D., Principal investigator, First Affiliated Hospital of Guangxi Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06024876
- Sponsor's protocol ID
- CS-101-06
- Status last verified
- Feb 2026
- First submitted
- 23 Aug 2023
- Last updated
- 12 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Lai Y, Liu R, Wang L, Ma XK, Li Y, Yang G, Shi L, Guo YL, Wei Z, Zhou X, Xu W, Hou Y, Miccio A, Yang B, Mou X, Yang L, Chen J. Clinical application of base editing for treating beta-thalassaemia. Nature. 2026 May;653(8115):923-932. doi: 10.1038/s41586-026-10342-9. Epub 2026 Apr 8.
PubMed 41951736
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Study Evaluating the Safety and Efficacy of Ex-vivo tBE Edited Autologous Hematopoietic Stem Progenitor Cells (CS-101) in Treating Subjects With β-thalassemia
- ClinicalTrials.gov ID
- NCT06024876
- Acronym / sponsor ID
- CS-101-06
- Conditions
- Beta-Thalassemia
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- CorrectSequence Therapeutics Co., LtdIndustry (pharma, biotech, devices)
- Collaborators
- First Affiliated Hospital of Guangxi Medical University
- Study start
- 26 Aug 2023 (actual)
- Primary completion
- 1 Jul 2025 (actual)
- Study completion
- 1 Jul 2025 (actual)
- First posted
- 6 Sep 2023 (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 β-thalassemia.
Detailed description
CS-101 is an autologous CD34+ cell suspension, edited by ex vivo base editing technology, which modifies the BCL11A binding site in HBG 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 fetal hemoglobin(HbF) in the blood, compensating for the function of missing adult hemoglobin HbA 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 | Experimental | Biological: CS-101CS-101: Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique |
Who may qualify
- Ages
- 6 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 6 to 35 years old(inclusive) male or female subjects at the time of informed consenting
- Diagnosis of β-thalassemia, genotypes include but are not limited to β+β0，βEβ0，β0β0, etc
- History of at least≥8 units/year of packed RBC transfusions in the prior 12 months prior to the screening period
- Generally in good condition, Karnofsky performance score≥60 points for subjects≥16 years old at the time of autologous hematopoietic stem cell collection, or Lansky Play-Performance score≥60 points for subjects under 16 years old, or equivalent clinical evaluation as the investigator site's common practice
Key exclusion criteria (10 listed)
- 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, when their drug-drug interaction on the efficacy and safety of CS-101 cannot be ruled out, unless at least there are 3 test results showing the total hemoglobin level before transfusion is below 9g/dL 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.
- Those with active infections, including but not limited to: HIV, hepatitis B, hepatitis C, cytomegalovirus, Epstein-Barr virus and treponema pallidum test positive, or known tuberculosis, parasitic infection, etc. who are judged by the investigator to be unsuitable to participate in this study.
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-101Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique | Biological | CS-101 |
Primary outcome
Frequency and severity of adverse events(AEs）as assessed by CTCAE v5.0
Time frame: From signing informed consent to 12 months post-CS-101 infusion
Time to neutrophil and platelet engraftment
Time frame: Days post-CS-101 infusion
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； 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 in the past 7 days；
Proportion of subjects with engraftment
Time frame: within 42 days post-CS-101infusion
Subjects with engraftment is defined as neutrophil engrafted
Incidence of transplant-related mortality
Time frame: From baseline to 100 days post-CS-101 infusion
All-cause mortality
Time frame: From signing informed consent to 12 months post-CS-101 infusion
Proportion of subjects achieving transfusion independence for at least 6 consecutive months
Time frame: From 3 months up to 12 months post-CS-101 infusion
Time to last red blood cell(RBC) transfusion
Time frame: Days post-CS-101 infusion
3 secondary outcomes
Change in total hemoglobin(Hb) concentration over time
Time frame: up to 12 months post-CS-101 infusion
Change in fetal hemoglobin(HbF) concentration over time
Time frame: up to 12 months post-CS-101 infusion
Chimerism level in Peripheral blood and bone marrow
Time frame: up to 12 months post-CS-101 infusion
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Feb 2026.
- Derived from this study: PubMed 41951736Lai Y, Liu R, Wang L, Ma XK, Li Y, Yang G, Shi L, Guo YL, Wei Z, Zhou X, Xu W, Hou Y, Miccio A, Yang B, Mou X, Yang L, Chen J. Clinical application of base editing for treating beta-thalassaemia. Nature. 2026 May;653(8115):923-932. doi: 10.1038/s41586-026-10342-9. Epub 2026 Apr 8.
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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. This study is not recruiting at the moment.
Yongrong Lai, M.D. principal investigator
First Affiliated Hospital of Guangxi Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing CS-101 in people with Beta-Thalassemia, run by CorrectSequence Therapeutics Co., Ltd at 1 site 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 4 inclusion and 10 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 12 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 1 site 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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?
Frequency and severity of adverse events(AEs）as assessed by CTCAE v5.0 (time frame: From signing informed consent to 12 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?
On the ClinicalTrials.gov record for NCT06024876, which also lists 1 linked publication.
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 12 Feb 2026.