Sickle Cell DiseaseEarly Phase 1NCT07000318
CS-206 in Patients With Sickle Cell Disease
A Early Phase 1 study testing CS-206 in people with Sickle Cell Disease, 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 CS-206 injection in treating sickle cell disease. Read more about this study
- Who is it for?
- Ages 12 to 18 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
- 30 Sep 2026to 31 Mar 2029
- 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 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participants must be between 12 to 18 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
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, 201102
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-206 injection in treating sickle cell disease.
Read the detailed description
CS-206 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
- 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 ex vivo base editing technique
| Study group | Role | What they receive |
|---|---|---|
| CS-206 | Experimental | Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo 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-206 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)
Whether the patient remained 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)
Whether the patient remained 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)
Whether the patient remained 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)
Severe vaso-occlusive crisis (VOC) annualized incidence rate
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
Annualized incidence rate of hospitalization due to severe vaso-occlusive crisis
Measured: starting 60 days after the last red blood cell transfusion
HbF (fetal hemoglobin) level in blood samples
Persistence of fetal hemoglobin (HbF) expression
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
Proportion of edited alleles in peripheral blood leukocytes and bone marrow cells, along with their persistence over time and the dynamics of chimerism rates.
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 posted2 Jun 2025
- Study start30 Sep 2026 (estimated)
- Primary completion30 Dec 2028 (estimated)
- Study completion31 Mar 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies 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
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Fudan University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Xiaowen Zhai, M.D., Principal investigator, Children's Hospital of Fudan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07000318
- Sponsor's protocol ID
- CS-206-02
- Status last verified
- May 2026
- First submitted
- 30 May 2025
- Last updated
- 15 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
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
- 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
- NCT07000318
- Acronym / sponsor ID
- CS-206-02
- Conditions
- Sickle Cell Disease
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Children's Hospital of Fudan UniversityUniversity, hospital or other
- Study start
- 30 Sep 2026 (estimated)
- Primary completion
- 30 Dec 2028 (estimated)
- Study completion
- 31 Mar 2029 (estimated)
- First posted
- 2 Jun 2025 (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-206 injection in treating sickle cell disease.
Detailed description
CS-206 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-206 | Experimental | Genetic: CS-206Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique |
Who may qualify
- Ages
- 12 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (16 listed)
- Participants must be between 12 to 18 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 ex vivo base editing technique | Genetic | CS-206 |
Primary outcome
AEs(Adverse Events) and SAEs(Serious Adverse Events) after CS-206 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
Whether the patient remained 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 15 May 2026.
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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.
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-206 in people with Sickle Cell Disease, 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 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: opening soon, 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?
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 Xiaowen Zhai, M.D. (+86-021-64931126, zhaixiaowendy@163.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-206 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?
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.