Sickle Cell DiseaseEarly Phase 1NCT06565026
CS-206 in Patients With Sickle Cell Disease
A Early Phase 1 study testing CS-206 in people with Sickle Cell Disease, 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 injection in treating sickle cell disease. Read more about this study
- Who is it for?
- Ages 12 to 35 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- CS-206
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteChina
- Study dates
- 2 Sep 2024to 30 Jun 2027
- Phase
- Early Phase 1
- 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
- Participants must be between 12 to 35 years old (inclusive). Participants or their legal guardians (for participants below 18 years old) must provide written informed consent before any study-related procedures.
- Participants must have a Documented βS/βS, βS/β0 or βS/β+ genotype.
- Participants must have at least one of the following conditions
- At least 2 occurrences of any of the following events within 2 years prior to screening.
- Acute pain crisis: requiring a visit to a medical facility and administration of pain medications (opioids or intravenous NSAIDs) or red blood cell transfusions.
- Acute chest syndrome: defined by the presence of a new pulmonary infiltrate on a chest X-ray, associated with pneumonia-like symptoms, including chest pain, fever, or respiratory distress.
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- Priapism lasting more than 2 hours and necessitating a visit to a medical facility for intervention.
- Stroke or transient ischemic attack (TIA): confirmed by imaging studies (e.g., MRI or CT scan), including silent stroke, and overt stroke leading to neurological deficits lasting >24 hours.
- Presence of red cell alloimmunization (>2 antibodies) and the need for ongoing chronic transfusions.
- Participants who have failed, not tolerated, refused the standard of care for Sickle Cell Disease (SCD), or are unable to access the standard of care due to the availability
- Other situations deemed appropriate for hematopoietic stem cell transplantation according to the sickle cell anemia treatment guidelines, as determined by the investigator.
- Laboratory Parameters:
- Documented Hemoglobin S (HbS) level ≥30% of total hemoglobin (Hb) concentration prior to transfusion.
- HbF at screening < 20%
- Participants must have a Karnofsky Performance Status (KPS for participants above 16 years old, inclusive) or Lansky Play-Performance Scale (LPPS for participants below 16 years old) score of ≥70, indicating sufficient functional status to undergo the intervention.
- Willing to comply with the protocol requirements, use contraception as required, attend regular follow-up visits, and cooperate with examinations.
Who cannot join
- Female participants who are pregnant, breastfeeding, or planning pregnancy during the study period are excluded.
- Participation in another investigational drug trial within 30 days prior to screening or within 5 half-lives (whichever is longer).
- Subjects who have received or are receiving luspatercept treatment within 3 months prior to screening.
- Subjects who have previously received any gene therapy for the disease.
- Subjects with a fully matched related donor who are already scheduled for allogeneic hematopoietic stem cell transplantation.
- More than 10 unplanned hospitalizations or emergency visits within 12 months prior to screening, which the investigator believes are related to significant chronic pain rather than acute pain crisis (VOC).
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- Severe liver dysfunction:
- Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >3× the upper limit of normal (ULN) or:
- International Normalized Ratio (INR) >1.5× ULN
- Severe renal impairment (creatinine clearance <30 mL/min/1.73 m²) are excluded.
- Subjects with HIV, cytomegalovirus (CMV), Epstein-Barr virus (EBV), or Treponema pallidum infection during the screening period; those with active HBV or HCV infection; or known tuberculosis or parasitic infection, etc. Excludes subjects with stable hepatitis B (HBV-DNA negative) after treatment and those cured of hepatitis C (HCV-RNA negative). Known active bacterial, viral, or fungal infections.
- Deemed unsuitable for autologous hematopoietic stem cell transplantation procedures as determined by the investigator.
- Other situations deemed unsuitable for this study as determined by the investigator.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi
Yongrong Lai, M.D.
Yongrong Lai, M.D.
About this study
Official title: An Open-Label Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CD34+ Human Hematopoietic Stem Cells Modified Using Transformer Base Editor in Participants With Severe Sickle Cell Disease
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 injection in treating sickle cell disease.
Read the detailed description
CS-101 is an autologous CD34+ cell suspension, edited by in vitro 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
- Sickle Cell Disease
- Being tested
- CS-206
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CS-206Genetic
Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique
| Study group | Role | What they receive |
|---|---|---|
| CS-206 | Experimental | Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique Receives: Genetic: CS-206 |
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: From signing informed consent to 24 months post-CS-206 infusion
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 and 12 months post-CS-206 infusion
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 24 months post-CS-206 infusion
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 24 months post-CS-206 infusion
All-cause mortality
Measured: Up to 24 months post-CS-206 infusion
Free from severe VOCs for 12 consecutive months (VF12)
Free from severe vaso-occlusive crises (VOCs) for 12 consecutive months (VF12)
Measured: starting 60 days after the last red blood cell transfusion up to 24 months
Secondary outcomes (6)
Free from hospitalization due to severe vaso-occlusive crises for 12 consecutive months(HF12)
Measured: starting 60 days after the last red blood cell transfusion up to 24 months
Free from severe VOCs for 9 consecutive months (VF9)
Free from severe vaso-occlusive crises (VOCs) for 9 consecutive months (VF9)
Measured: starting 60 days after the last red blood cell transfusion up to 24 months
Annualized incidence of severe vaso-occlusive crises (VOC)
Measured: starting 60 days after the last red blood cell transfusion up to 24 months
Annualized incidence of hospitalization due to severe vaso-occlusive crises
Measured: starting 60 days after the last red blood cell transfusion
HbF (fetal hemoglobin) level in blood samples
Measured: up to 24 months post-CS-206 infusion
Proportion of edited alleles in peripheral blood leukocytes and bone marrow cells, and persistence and chimerism kinetics evaluation
Measured: up to 24 months post-CS-206 infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2024
- Study start2 Sep 2024
- Primary completion31 Dec 2026 (estimated)
- Study completion30 Jun 2027 (estimated)
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 Sickle Cell DiseaseStudies 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 (estimated)
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
- NCT06565026
- Sponsor's protocol ID
- CS-206-01
- Status last verified
- Feb 2026
- First submitted
- 13 Aug 2024
- Last updated
- 10 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- 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 10 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CD34+ Human Hematopoietic Stem Cells Modified Using Transformer Base Editor in Participants With Severe Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT06565026
- Acronym / sponsor ID
- CS-206-01
- Conditions
- Sickle Cell Disease
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- CorrectSequence Therapeutics Co., LtdIndustry (pharma, biotech, devices)
- Collaborators
- First Affiliated Hospital of Guangxi Medical University
- Study start
- 2 Sep 2024 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 21 Aug 2024 (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 injection in treating sickle cell disease.
Detailed description
CS-101 is an autologous CD34+ cell suspension, edited by in vitro 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-206 | Experimental | Genetic: CS-206Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique |
Who may qualify
- Ages
- 12 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (16 listed)
- Participants must be between 12 to 35 years old (inclusive). Participants or their legal guardians (for participants below 18 years old) must provide written informed consent before any study-related procedures.
- Participants must have a Documented βS/βS, βS/β0 or βS/β+ genotype.
- Participants must have at least one of the following conditions
- Laboratory Parameters:
- Participants must have a Karnofsky Performance Status (KPS for participants above 16 years old, inclusive) or Lansky Play-Performance Scale (LPPS for participants below 16 years old) score of ≥70, indicating sufficient functional status to undergo the intervention.
Key exclusion criteria (13 listed)
- Female participants who are pregnant, breastfeeding, or planning pregnancy during the study period are excluded.
- Participation in another investigational drug trial within 30 days prior to screening or within 5 half-lives (whichever is longer).
- Subjects who have received or are receiving luspatercept treatment within 3 months prior to screening.
- Subjects who have previously received any gene therapy for the disease.
- Subjects with a fully matched related donor who are already scheduled for allogeneic hematopoietic stem cell transplantation.
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-206Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique | Genetic | CS-206 |
Primary outcome
AEs(Adverse Events) and SAEs(Serious Adverse Events) after CS-101 infusion
Time frame: From signing informed consent to 24 months post-CS-206 infusion
Frequency and severity of adverse events(AEs）as assessed by CTCAE(Common Terminology Criteria for Adverse Events)v5.0
Incidence of transplant-related mortality
Time frame: From baseline to 100 days and 12 months post-CS-206 infusion
Incidence of transplant-related mortality(Transplant-related mortality events defined as deaths assessed by the investigator as potentially transplant-related)
Time to neutrophil engraftment
Time frame: Up to 24 months post-CS-206 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
Time frame: Up to 24 months post-CS-206 infusion
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.
All-cause mortality
Time frame: Up to 24 months post-CS-206 infusion
Free from severe VOCs for 12 consecutive months (VF12)
Time frame: starting 60 days after the last red blood cell transfusion up to 24 months
Free from severe vaso-occlusive crises (VOCs) for 12 consecutive months (VF12)
6 secondary outcomes
Free from hospitalization due to severe vaso-occlusive crises for 12 consecutive months(HF12)
Time frame: starting 60 days after the last red blood cell transfusion up to 24 months
Free from severe VOCs for 9 consecutive months (VF9)
Time frame: starting 60 days after the last red blood cell transfusion up to 24 months
Annualized incidence of severe vaso-occlusive crises (VOC)
Time frame: starting 60 days after the last red blood cell transfusion up to 24 months
Annualized incidence of hospitalization due to severe vaso-occlusive crises
Time frame: starting 60 days after the last red blood cell transfusion
HbF (fetal hemoglobin) level in blood samples
Time frame: up to 24 months post-CS-206 infusion
Proportion of edited alleles in peripheral blood leukocytes and bone marrow cells, and persistence and chimerism kinetics evaluation
Time frame: up to 24 months post-CS-206 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 10 Feb 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.
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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.
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-206 in people with Sickle Cell Disease, 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 16 inclusion and 13 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: looking for participants now, 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 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?
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 Yaliang Li (+ 86 186 2104 6122, CT@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: From signing informed consent to 24 months post-CS-206 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 NCT06565026, 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 10 Feb 2026.