Gene-edited stem cell test for beta-thalassemia
Tests CS-101, gene-edited patient stem cells, in people with beta-thalassemia.
Plain-language title generated from the registry record
Registry title: CS-101 in Patients With β-thalassemia
Key facts
- Where
- Nanning, ChinaSee the site
- Phase
- Early phase 1
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 6 and 35 years old?
Source: NCT06328764, who can take part
Where in China
- The First Affiliated Hospital of Guangxi Medical UniversityNanning, ChinaNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- CorrectSequence Therapeutics Co., Ltd
- Trial code
- NCT06328764
- Record updated
- 9 June 2026
- Original description
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.Read full original descriptionShow less
A Clinical Study Evaluating the Safety and Efficacy of Autologous Peripheral Blood Hematopoietic Stem and Progenitor Cells (CS-101) Modified by Ex Vivo Base Editing to Induce γ-Globin Production in Treating Patients With β-Thalassemia
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.
Inclusion: 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: 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.