NCT06024876CompletedEarly phase 1
A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia
A Clinical Study Evaluating the Safety and Efficacy of Ex-vivo tBE Edited Autologous Hematopoietic Stem Progenitor Cells (CS-101) in Treating Subjects With β-thalassemia
Source: ClinicalTrials.gov · Last updated 12 Feb 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Phase (registry)
- Early phase 1
- Study type
- Interventional
- Enrollment
- 5 (actual)
- First submitted
- 23 Aug 2023
- Start date
- 26 Aug 2023
- Primary completion date
- 1 Jul 2025
- Lead sponsor
- CorrectSequence Therapeutics Co., Ltd
- Diseases
- Thalassaemia
- NCT number
- NCT06024876
What you commit to
- Study type: Interventional · Early phase 1
- Participants enrolled: 5
- From 26 Aug 2023 to the primary completion date, 1 Jul 2025
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
- 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.
Other illness
- 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.
Lab values
- 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.
- 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.
Other
- MRI during the screening period showed heavy iron overload and is judged by the investigator to be unable to participate in the study.
Who the study is looking for
- 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
Age in the registry: 6 years to 35 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| The First Affiliated Hospital of Guangxi Medical University Coordinator of this site? Confirm its status | Nanning | China | Unknown status |
Related publications
- Clinical application of base editing for treating β-thalassaemia.Nature · 2026 · PMID 41951736
This page is information, not medical advice. Discuss any trial with your doctor.