CS-206 in Patients With Sickle Cell Disease

February 8, 2026 updated by: CorrectSequence Therapeutics Co., Ltd

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
5

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Early Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • China
    • Guangxi
      • Nanning, Guangxi, China
        • Recruiting
        • The First Affiliated Hospital of Guangxi Medical University
        • Contact:
          • Yongrong Lai, M.D.
        • Principal Investigator:
          • Yongrong Lai, M.D.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • 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

    1. At least 2 occurrences of any of the following events within 2 years prior to screening.

      1. Acute pain crisis: requiring a visit to a medical facility and administration of pain medications (opioids or intravenous NSAIDs) or red blood cell transfusions.
      2. 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.
      3. Priapism lasting more than 2 hours and necessitating a visit to a medical facility for intervention.
      4. 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.
    2. Presence of red cell alloimmunization (>2 antibodies) and the need for ongoing chronic transfusions.
    3. 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
    4. Other situations deemed appropriate for hematopoietic stem cell transplantation according to the sickle cell anemia treatment guidelines, as determined by the investigator.

  • Laboratory Parameters:

    1. Documented Hemoglobin S (HbS) level ≥30% of total hemoglobin (Hb) concentration prior to transfusion.
    2. 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.

Exclusion Criteria:

  • 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).

  • Severe liver dysfunction:

    1. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >3× the upper limit of normal (ULN) or:
    2. 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: CS-206
Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique
Genetic: CS-206
Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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)
starting 60 days after the last red blood cell transfusion up to 24 months
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)
From baseline to 100 days and 12 months post-CS-206 infusion
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.
Up to 24 months post-CS-206 infusion
All-cause mortality
Time Frame: Up to 24 months post-CS-206 infusion
Up to 24 months post-CS-206 infusion
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
From signing informed consent to 24 months post-CS-206 infusion
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.
Up to 24 months post-CS-206 infusion

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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
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
Free from severe vaso-occlusive crises (VOCs) for 9 consecutive months (VF9)
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
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
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
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
up to 24 months post-CS-206 infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
CorrectSequence Therapeutics Co., Ltd

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
First Affiliated Hospital of Guangxi Medical University

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Yongrong Lai, M.D., First Affiliated Hospital of Guangxi Medical University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 2, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 31, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 30, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 13, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 19, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 21, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 10, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 8, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CS-206-01

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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