Beta-ThalassemiaEarly Phase 1NCT06065189
Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major
A Early Phase 1 study testing CS-101 injection in people with Beta-Thalassemia, run by Children's Hospital of Fudan University 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 base-edited autologous hematopoietic stem cell transplantation(CS-101) in treating patients with β-thalassemia major. Read more about this study
- Who is it for?
- Ages 3 to 17 years
- Conditions the study is for
- Beta-Thalassemia
- 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
- 2 participants
- Where
- 1 siteChina
- Study dates
- 7 Nov 2023to 12 Aug 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 3 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 3 to 17 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
- 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
- For female subjects of childbearing potential: use effective contraceptive measures for at least 1 month prior to 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, 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.
- Subjects 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 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%
- Subjects who are febrile (temperature over 37.3° C) should be held back from enrolment.
- 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.
- 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
- 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
1 site in 1 country.
China1 site
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, 201102
About this study
Official title: Evaluation and Promotion of Key Technologies of Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of base-edited autologous hematopoietic stem cell transplantation(CS-101) in treating patients with β-thalassemia major.
Read the detailed description
CS-101 is an autologous CD34+ cell suspension modified by ex vivo base editing technology, removing the inhibitory effect of BCL11A on the γ-globin coding gene, inducing the production of γ-globin chains, increasing the concentration of fetal hemoglobin (HbF) in the blood, compensating for the loss of adult hemoglobin (HbA) to treat transfusion-dependent type/ Major β - thalassemia. The therapy addresses two major challenges in the treatment of the disease: lack of matching donors and graft-versus-host responses commonly seen in allogeneic hematopoietic stem cell transplantation.
The study consists of the following five phases:
Screening phase: Sign informed consent, complete screening assessments, and confirm the eligibility for enrollment; Baseline: check the subject's baseline status; Mobilization, collection and manufacturing phase: mobilize, collect autologous CD34+ cells and manufacture, release and transport CS-101 product; Conditioning and treatment phase: including myeloablation and CS-101 product infusion; Follow-up phase: 180 days post-infusion.
- Conditions
- Beta-Thalassemia
- 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 injectionBiological
Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique
| Study group | Role | What they receive |
|---|---|---|
| CS-101 injection | Experimental | Autologous CD34+(cluster of differentiation 34) hematopoietic stem cell suspension modified by in vitro base editing technique Receives: Biological: CS-101 injection |
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 180 days post-CS-101 infusion
Occurrence of engraftment
Subjects with engraftment is defined as neutrophil engrafted
Measured: within 42 days 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
Measured: Days post-CS-101 infusion
Occurrence of transplant-related death
Measured: baseline to 100 days post-CS-101 infusion
Occurrence of all-cause death
Measured: From signing informed consent to 180 days post-CS-101 infusion
Occurrence of achieving transfusion reduction for at least 3 consecutive months
Measured: From 3 months post -CS-101 infusion to 3 months post -CS-101 infusion
Secondary outcomes (5)
Occurrence of achieving transfusion independence for at least 3 consecutive months
Measured: From 3 months up to 180 days post-CS-101 infusion
Time to last red blood cell(RBC) transfusion
Measured: Days post-CS-101 infusion
Change in total hemoglobin(Hb) concentration over time
Measured: up to 180 days post-CS-101 infusion
Change in fetal hemoglobin(HbF) concentration over time
Measured: up to 180 days 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 180 days 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 posted3 Oct 2023
- Study start7 Nov 2023
- Primary completion12 Aug 2025
- Study completion12 Aug 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.
- More trials run by Children's Hospital of Fudan UniversityThe sponsor's other registered studies.
- 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
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Fudan University
- Sponsor type
- University, hospital or other
- Collaborators
- CorrectSequence Therapeutics Co., Ltd
- Responsible party
- Sponsor
- Investigators
- Xiaowen Zhai, M.D., Principal investigator, Children's Hospital of Fudan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06065189
- Sponsor's protocol ID
- CS-101-03
- Status last verified
- May 2026
- First submitted
- 27 Sep 2023
- Last updated
- 15 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Liu R, Lai Y, Wang L, Qian X, Ma XK, Li Y, Yang G, Shi L, Li Z, Wei J, Wei Z, Wang H, Zhou X, Yu L, Yang B, Zhai X, Mou X, Yang L, Chen J. Clinical base editing for beta-hemoglobinopathies across different genetic backgrounds. Cell Stem Cell. 2026 Sep 7:S1934-5909(26)00310-3. doi: 10.1016/j.stem.2026.08.009. Online ahead of print.
PubMed 42705233
Study at a glance
From the registry record, last updated by the sponsor on 15 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation and Promotion of Key Technologies of Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major
- ClinicalTrials.gov ID
- NCT06065189
- Acronym / sponsor ID
- CS-101-03
- Conditions
- Beta-Thalassemia
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Children's Hospital of Fudan UniversityUniversity, hospital or other
- Collaborators
- CorrectSequence Therapeutics Co., Ltd
- Study start
- 7 Nov 2023 (actual)
- Primary completion
- 12 Aug 2025 (actual)
- Study completion
- 12 Aug 2025 (actual)
- First posted
- 3 Oct 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 base-edited autologous hematopoietic stem cell transplantation(CS-101) in treating patients with β-thalassemia major.
Detailed description
CS-101 is an autologous CD34+ cell suspension modified by ex vivo base editing technology, removing the inhibitory effect of BCL11A on the γ-globin coding gene, inducing the production of γ-globin chains, increasing the concentration of fetal hemoglobin (HbF) in the blood, compensating for the loss of adult hemoglobin (HbA) to treat transfusion-dependent type/ Major β - thalassemia. The therapy addresses two major challenges in the treatment of the disease: lack of matching donors and graft-versus-host responses commonly seen in allogeneic hematopoietic stem cell transplantation.
The study consists of the following five phases:
Screening phase: Sign informed consent, complete screening assessments, and confirm the eligibility for enrollment; Baseline: check the subject's baseline status; Mobilization, collection and manufacturing phase: mobilize, collect autologous CD34+ cells and manufacture, release and transport CS-101 product; Conditioning and treatment phase: including myeloablation and CS-101 product infusion; Follow-up phase: 180 days post-infusion.
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 | Biological: CS-101 injectionAutologous CD34+(cluster of differentiation 34) hematopoietic stem cell suspension modified by in vitro base editing technique |
Who may qualify
- Ages
- 3 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 3 to 17 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
- 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
- For female subjects of childbearing potential: use effective contraceptive measures for at least 1 month prior to 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
Key exclusion criteria (16 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.
- Subjects with coexisting α-thalassemia and more than 2 deletions or non-deletional mutations in the α-globin chain coding genes.
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 in vitro base editing technique | Biological | CS-101 injection |
Primary outcome
Frequency and severity of adverse events(AEs) as assessed by CTCAE v5.0
Time frame: From signing informed consent to 180 days post-CS-101 infusion
Occurrence of engraftment
Time frame: within 42 days post-CS-101 infusion
Subjects with engraftment is defined as neutrophil engrafted
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
Occurrence of transplant-related death
Time frame: baseline to 100 days post-CS-101 infusion
Occurrence of all-cause death
Time frame: From signing informed consent to 180 days post-CS-101 infusion
Occurrence of achieving transfusion reduction for at least 3 consecutive months
Time frame: From 3 months post -CS-101 infusion to 3 months post -CS-101 infusion
5 secondary outcomes
Occurrence of achieving transfusion independence for at least 3 consecutive months
Time frame: From 3 months up to 180 days post-CS-101 infusion
Time to last red blood cell(RBC) transfusion
Time frame: Days post-CS-101 infusion
Change in total hemoglobin(Hb) concentration over time
Time frame: up to 180 days post-CS-101 infusion
Change in fetal hemoglobin(HbF) concentration over time
Time frame: up to 180 days post-CS-101 infusion
Chimerism level in Peripheral blood and bone marrow
Time frame: up to 180 days 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 15 May 2026.
- Derived from this study: PubMed 42705233Liu R, Lai Y, Wang L, Qian X, Ma XK, Li Y, Yang G, Shi L, Li Z, Wei J, Wei Z, Wang H, Zhou X, Yu L, Yang B, Zhai X, Mou X, Yang L, Chen J. Clinical base editing for beta-hemoglobinopathies across different genetic backgrounds. Cell Stem Cell. 2026 Sep 7:S1934-5909(26)00310-3. doi: 10.1016/j.stem.2026.08.009. Online ahead of print.
- 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. This study is not recruiting at the moment.
Xiaowen Zhai, M.D. principal investigator
Children's Hospital of Fudan 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 injection in people with Beta-Thalassemia, run by Children's Hospital of Fudan University 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 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 15 May 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 180 days post-CS-101 infusion). The study lists 6 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 NCT06065189, 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 15 May 2026.