Clinical trial
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
Name
CS-101-06
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.
Trial arms
Trial start
2023-08-26
Estimated PCD
2024-12-31
Trial end
2025-06-30
Status
Active (not recruiting)
Phase
Early phase I
Treatment
CS-101
Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique
Arms:
CS-101
Size
5
Primary endpoint
Frequency and severity of adverse events(AEs）as assessed by CTCAE v5.0
From signing informed consent to 12 months post-CS-101 infusion
Time to neutrophil and platelet engraftment
Days post-CS-101 infusion
Proportion of subjects with engraftment
within 42 days post-CS-101infusion
Incidence of transplant-related mortality
From baseline to 100 days post-CS-101 infusion
All-cause mortality
From signing informed consent to 12 months post-CS-101 infusion
Proportion of subjects achieving transfusion independence for at least 6 consecutive months
From 3 months up to 12 months post-CS-101 infusion
Time to last red blood cell(RBC) transfusion
Days post-CS-101 infusion
Eligibility criteria
Key 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
Key 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.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['EARLY_PHASE1'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 5, 'type': 'ACTUAL'}}
Updated at
2024-05-07
1 organization
1 product
1 indication
Organization
CorrectSequence TherapeuticsProduct
CS-101Indication
Beta Thalassemia