Loading clinical trials...

CS-101 in Patients With β-thalassemia | Clinical Trials | Clareo Health

ENROLLING_BY_INVITATIONEARLY_PHASE1

CS-101 in Patients With β-thalassemia

A Clinical Study Evaluating the Safety and Efficacy of In-vitro tBE Edited Autologous Hematopoietic Stem Progenitor Cells(CS-101) in Treating Subjects With β-thalassemia

NCT06328764•CorrectSequence Therapeutics Co., Ltd

View on ClinicalTrials.gov

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.

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.

Eligibility

Age

6 - 35 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•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

Exclusion Criteria

•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%. 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.

Locations (1)

The First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, China

Key Dates

Start Date

March 19, 2024

Primary Completion Date

July 31, 2026

Completion Date

July 31, 2026

Last Updated

February 10, 2026

Enrollment

ESTIMATED participants

Conditions

Beta-Thalassemia

Interventions

CS-101

GENETIC

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

NCT06647979

Sickle Cell DiseaseSickle Cell Anemia (HbSS, or HbSβ-thalassemia0)+2 more

View study

RECRUITINGPHASE1/PHASE2

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

NCT03653338

Sickle Cell AnemiaBeta-thalassemia Major+1 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 10, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

CS-101 in Patients With β-thalassemia

Inclusion Criteria

Exclusion Criteria

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia

Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia

A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]

Use of the Hemanext One® Hypoxic Red Blood Cell Storage System for Transfusion in Thalassemia Patients